Archive for May, 2020

If we are looking for some entity to blame for the Coronavirus Pandemic, the obvious choice would be China. Scientists are fairly certain that is where the micro-organism began an assault on humanity.

The city of Wuhan is known as a center of bio-engineering experimentation. Whether or not the outbreak was an accident or part of, as some Russian military experts have claimed, a strategic effort to realign the political power structure on Earth, is a different question. If one wanted to put the question before a jury, as any prosecuting attorney would point out, China had the means, the opportunity, and the motive. Their economic light was dimming dramatically, and the recent surge of demands for democratic reforms, and the stiff tariffs imposed by Trump have shaken the Communist regime. With a population of nearly 1.4 billion, losing a few million citizens to disease would be a cheap price for them to pay to overcome Americas worldwide economic dominance.

What about Russia? Not a chance. They are super dependent on oil to prop up their weak economic infrastructure and they would never have contributed to crude prices falling into negative numbers. Europe? Not scientifically or politically motivated or capable.

That leaves the USA. The Chinese have suggested we started the outbreak, though they never outlined why we would want to destroy our own economy.

There is one group within the worldwide demographic that could qualify as beneficiaries of a complete economic and political realignment: That would be the post Baby Boomer generations of Millennials and X-Gens. We have surveys that claim most of them hate capitalism and economic inequality. If, as a theoretical political force, they wanted to clear the decks for their future, what would be the one thing that could redistribute economic wealth, restore the health of the environment, and expand the nanny state?

The answer is reducing or eliminating the drag that senior citizens put on civilization. Americas aging population is living longer, using more resources, drawing on retirement benefits and social services at higher rates than anyone ever predicted when statisticians did their amortization schedules back in the 70s and 80s. We old farts were supposed to have been gone by now. If there is any group that might see an economic upside to the pandemic it would be our kids and their kids.

Do I really think our descendants are anxious to kill us off? No, but the cause and effect are intriguing food for thought Do I really believe this Pandemic is manmade? Yes and no. I think science got burned playing with fire. In our never-ending search for the elixir of youth, science sometimes ventures into dangerous territory. So I think this epidemic was probably made worse by human experimentation, trying to understand more about how these molecular killers threaten our species. Were all living a nightmare resulting from good intentions gone horribly wrong.

Whether by accident or by design the Coovid19 pandemic is effectively culling the herd. Ranchers do this to improve the gene pool of their cattle, or to reduce their costs associated with illnesses. Mother Nature also has a way of draining the swamp just before it becomes too toxic, then replenishing it with freshwater and reviving the ecological balance.

Am I suggesting seniors are toxic and young people are willing to put us out to pasture? No, but the truth is life is terminal, and living in freedom is dangerous. No society can guarantee safety and security, under any political system. During this scary period, civilization should attempt to protect the most vulnerable, the aged, and the sick, and once again young people will bear the cost and lifestyle burden for them. Baby Boomers have made life extension our main goal, and now our offspring will have to pick up the tab.

Rick Elkin is an artist, author, and columnist. His most recent book, The Illusion of Knowledge: Why So Many Educated Americans Embrace Marxism, is available at most online booksellers. He resides in Escondido, California. You can follow him at RickElkin.com.

*Note: Opinions expressed by columnists and letter writers are those of the writers and not necessarily those of the newspaper.

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Picking Up the Tab - times-advocate.com

New Delhi: Eight workers of NLC India Ltd were injured and hospitalised in a boiler blast at NLC India Limiteds thermal power station (TPS) II in Neyveli in Tamil Nadu on Thursday.

The power stations of the integrated mining-cum-power generating company are located at Neyveli in Cuddalore.

Here is all you need to know about at NLC India Limiteds thermal power station (TPS) II in Neyveli, the company where the boiler blast occurred.

Neyveli Thermal Power Station-I

The first unit of the 600 MW Neyveli Thermal Power Station-I was synchronized in May 1962 and the last unit was synchronized in September 1970

The 600 MW Neyveli Thermal Power Station-I consists of six units of 50 MW each and three units of 100 MW each.

The Power generated from Thermal Power Station-I is supplied to TANGEDCO, Tamilnadu which is the sole beneficiary.

Due to the aging of the equipments / high pressure parts, the Life extension programme has been approved by the government in March 1992 and was successfully completed in March 1999 thus extending the life by 15 years, the company profile reads.

This power station is being operated after conducting Residual Life Assessment (RLA) study. Government has sanctioned a 2x500 MW Power Project (Neyveli New Thermal Power Plant NNTPS) in June 2011 as replacement for existing TPS-I, it adds.

More:
NLC India TPS II, all about the company where boiler blast occurred in Neyveli - Zee News

When looking at treatment outcomes for small, hormone-receptor-positive, HER2-positive breast cancers that have not spread to the lymph nodes, a study suggests that women diagnosed with cancers 810 mm (about 0.31 inches to 0.39 inches) in size had better survival when they were treated with chemotherapy after surgery compared to women diagnosed with smaller cancers.

The research was published on April 9, 2020, by the journal JAMA Network Open. Read the abstract of Association of Survival With Chemoendocrine Therapy in Women With Small, Hormone Receptor-Positive, ERBB2-Positive, Node-Negative Breast Cancer.

The HER2 gene is also called the ERBB2 (Erb-B2 receptor tyrosine kinase 2) gene.

Research shows that very small, node-negative, hormone-receptor-positive, HER2-positive breast cancers have a 5-year recurrence risk the risk of the cancer coming back within 5 years that ranges from 5% to 25%, with or without treatments after surgery. Doctors call treatments given after surgery adjuvant treatments.

Because HER2-positive cancer is considered more aggressive than HER2-negative breast cancer, it is usually treated with chemotherapy after surgery to reduce recurrence risk. Still, it has been unclear whether the benefits of treating very small, node-negative, hormone-receptor-positive, HER2-positive breast cancers with chemotherapy after surgery outweighed the risks.

It wasnt clear what to do with these patients, who make up a really small population of breast cancer patients, Anurag Singh, M.D., of Roswell Park Comprehensive Cancer Center, one of the researchers who did the study, said in an interview.

We know that if you have an ERBB2 tumor, it's worse, and the idea has been they should get chemotherapy,he continued. You have an otherwise healthy 60-year-old, mammographically screened and detected patient. When you give them the information, they're saying, 'OK, I have all of these good things, but I've got a 2-mm tumor and you want to give me 6 months of chemo. Are you serious? I'm going to lose my hair, I'm going to have this, I'm going to have that. It seems like an awful long way to go.' We didn't really have good evidence for them.

The researchers looked at the records of 10,065 women diagnosed with node-negative, hormone-receptor-positive, HER2-positive breast cancer that was 10 mm or smaller in size between 2010 and 2015.

All of the women were treated with hormonal therapy after surgery:

From 2013 to 2015, anti-HER2 therapy such as Herceptin (chemical name: trastuzumab) was coded differently than chemotherapy in patient records; 15% of the women treated during this time had either chemotherapy or anti-HER2 therapy alone.

Follow-up time ranged from about 2 years to about 5 years.

The researchers compared the outcomes of women treated with hormonal therapy and chemotherapy to women treated with hormonal therapy alone. They also looked to see if the size of the cancer affected any benefits from chemotherapy.

The analysis showed that chemotherapy was associated with better overall survival for women diagnosed with cancers that were 810 mm in size. Women diagnosed with cancers that were smaller did not seem to get any survival benefits from chemotherapy.

To our knowledge, this is the first report to suggest that there is an association between improved survival and adjuvant chemoendocrine therapy specifically for HR-positive, ERBB2-positive tumors 8 mm to 10 mm compared with those smaller than 8 mm, the researchers wrote. It is evident that tumors 10 mm and smaller represent a heterogeneous group whose treatment should be tailored to improve the risk-to-benefit ratio of systemic therapy.

The results of this study offer helpful information about which women may benefit from chemotherapy after surgery for very small, hormone-receptor-positive, HER2-positive breast cancer that hasnt spread to the lymph nodes.

Still, the results are based on extremely small differences in cancer size millimeters and require expert pathology review of the cancer. The researchers also didnt know the exact chemotherapy regimens the women had.

Also, anti-HER2 medicines, such as Herceptin, are often given along with chemotherapy. In this study, for 2 years of the 5-year study, anti-HER2 therapy and chemotherapy were not coded differently. So, a number of women may have been receiving both anti-HER2 therapy and chemotherapy during that time, which may have affected the study results.

If youve been diagnosed with a very small, hormone-receptor-positive, HER2-positive breast cancer that hasnt spread to the lymph nodes and are considering treatments after surgery, it makes sense to talk about this study with your doctor. The results offer more information about outcomes and can help you decide on the best treatments for your unique situation.

For more information on chemotherapy, including types of medicines and side effects, visit the Breastcancer.org Chemotherapy pages.

Written by: Jamie DePolo, senior editor

Published on May 7, 2020 at 12:02 PM

Excerpt from:
Chemotherapy for Certain Small, Hormone-Receptor-Positive, HER2-Positive Breast Cancers Seems to Improve Survival - Breastcancer.org

Updated: May 8, 2020 3:18:58 pm

By Dr Uma Vaidyanathan

Thalassaemia is a disorder of the haemoglobin structure. A tetramer molecule present in the RBCs, haemoglobin is responsible for supplying oxygen in the body and is a rich source of protein in the blood. The human Hb/HbA has two sets of globin chains. These are a-globin chain and -globin chain. Four genes (two inherited from the mother and two from the father) regulate the production of a-globin chain, while only two genes (each inherited from father and mother) control the production of -globin chain.

The thalassaemia syndromes are characterised by a basic defect in the synthesis of one type of globin chains. As a result, there is insufficient Hb content in the resultant red cells, leading to decreased haemoglobin concentration, anaemia and need for multiple blood transfusions in severe cases.

Each year, more than 70,000 babies are born with thalassaemia worldwide and this defect is very often seen in the Indian subcontinent. Haemoglobin electrophoresis is the gold standard in diagnosing this condition. Patients with thalassemia traits do not require long-term monitoring. They usually do not have iron deficiency, so iron supplements are unlikely to improve their anemia.

Read| Pregnant during the COVID-19 crisis? Heres how to take care of mental health

Alpha thalassaemia

In alpha thalassaemia, the hemoglobin does not produce enough alpha protein. To make alpha-globin protein chains we need four genes, two on each chromosome 16. We get two from each parent. If one or more of these genes is missing, it will result in alpha thalassemia. The severity of thalassemia depends on how many genes are faulty, or mutated.

One faulty gene: The patient has no symptoms. A healthy person who has a child with symptoms of thalassemia is a carrier. This type is known as alpha thalassemia minima.

Read| Covid-19 and pregnancy: What expectant mothers need to know

Two faulty genes: The patient has mild anemia. It is known as alpha thalassemia minor.

Three faulty genes: The patient has hemoglobin H disease. This is a type of chronic anemia. In this case, the patient needs regular blood transfusions throughout their life.

Four faulty genes: Alpha thalassaemia major is the most severe form of alpha thalassemia. It is known to cause hydrops fetalis, a serious condition in which fluid accumulates in parts of the fetus body. A fetus with four mutated genes cannot produce normal hemoglobin and is unlikely to survive, even with blood transfusions.

Beta Thalassemia

Two globin genes are required to make beta-globin chains: one gene from each parent. Beta thalassaemia is caused when one or both genes are faulty.

Severity depends on how many genes are mutated.

One faulty gene: This is called beta thalassaemia minor.

Two faulty genes: There may be moderate or severe symptoms. This is known as thalassaemia major (earlier known as Colleys anemia).

Medical management of thalassaemia major depending on severity would be:

Blood transfusions

Chelation therapy removal of excess iron overload in body

Bone marrow transplant

Gene therapy

Management during Pregnancy can be divided as follows:

Periconceptional care

Screening and counseling pre-pregnancy: Screening can identify couples having 25 per cent risk or more of having a pregnancy with signi?cant haemoglobinopathy. If a pregnant woman is found to be a carrier of haemoglobinopathy, the partner needs to be screened as soon as possible. If a risk of the fetus having major haemoglobinopathy is detected, urgent expert counselling is provided to the couple so that they can make an informed choice regarding the prenatal diagnosis and the possible termination of pregnancy.

Folic acid supplementation: Beginning in the preconceptual period, at least three months prior to conception, folic acid in the dosage of 5 mg/day helps in preventing neural tube defects.

Some additional tests also might be needed. Doctor might ask the patient to take blood sugar and thyroid function tests. Patients could be asked for cardiovascular assessment. Ultrasound of liver and the gallbladder (and spleen, if present) should be performed. This helps in detecting gallstones and evidence of liver cirrhosis due to iron overload or transfusion-related viral hepatitis.

Antenatal care

Women with thalassaemia should be reviewed on a monthly basis until 28 weeks of gestation and fortnightly thereafter. Women with thalassaemia are best treated in a multidisciplinary team setting, including an obstetrician with expertise in managing high-risk pregnancies and a hematologist.

Ultrasound scanning

An early scan after 7 to 9 weeks of gestation is needed to determine viability as well as the presence of a multiple pregnancy. A detailed anomaly scan must also be done after 11-12 weeks and 18 to 20 weeks of gestation. Later, ultrasounds may be needed to assess foetal growth restriction.

Care during labour and delivery

Thalassaemia is not an indication for cesarean section. Patients may need blood transfusion in case of excessive blood loss during the delivery.

Postpartum care

Women with thalassemia are at high risk for venous thromboembolism due to the presence of abnormal red blood cells in the circulation.

Breastfeeding is safe and should be encouraged. In addition, there is no contraindication to the use of hormonal methods of contraception, such as the combined oral contraceptive pill, the progestogen-only pill, hormonal implants, and the Mirena intrauterine system in women with thalassaemia.

(The writer is Senior Consultant, Obstetrics and Gynecology Fortis Hospital, Shalimar Bagh.)

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World Thalassaemia Day: What does the condition mean for pregnancy? - The Indian Express

According to the World Health Organization (WHO), cardiovascular disease, specifically ischemic heart disease, is one of the leading causes of death worldwide. Cardiovascular diseases result in an estimated 17.9 million deaths each year. This is about 31% of all deaths worldwide (1). Medical researchers are continually working on ways to reduce those numbers, including the development of new technologies to combat premature deaths from cardiovascular diseases. This article will focus, in particular, on the value of induced pluripotent stem cells (iPSCs) in cardiac research.

iPSCs are a type of pluripotent stem cell. These are master cells that can differentiate into any cell or tissue the body needs. They are generated directly from somatic cells through ectopic expression of various transcription factors, such as

Theyve become key tools to model biological processes, particularly in cell types that are difficult to access from living donors. Many research laboratories are working to enhance reprogramming efficiency by testing different cocktails of transcription factors.

iPSCs have become essential in a number of different research fields, including cardiac research.

They are a valuable and advantageous technologic development for two main reasons:

Most people have heard of embryonic stem cells, which are one variation of pluripotent cells. Like iPSCs, they can be used to replace or restore tissues that have been damaged.

The problem is that embryonic stem cells are only found in preimplantation stage embryos (3). Whereas iPSCs are adult cells that have been genetically modified to work like embryonic stem cells. Thus, the term, inducedpluripotent stem cells.

The development of iPSCs was helpful because embryos are not needed. This reduces the controversy surrounding the creation and use of stem cells. Further, iPSCs from human donors are also more compatible with patients than animal iPSCs, making them even closer to their embryonic cousins.

The Japanese inventor of iPSCs, Professor Shinya Yamanaka earned a Nobel Prize in 2012 for the discovery that mature cells can be reprogrammed to become pluripotent. (4) The Prize was awarded to Dr. Yamanaka because of the significant medical and research implications this technology holds.

iPSCs hold a lot of promise for transplantation medicine. Further, they are highly useful in drug development and modeling of diseases.

iPSCs may become important in transplantation medicine because the tissues developed from them are a nearly identical match to the cell donors. This can potentially reduce the chances of rejection by the immune system (5).

In the future, and with enough research, it is highly possible that researchers may be able to perfect the iPSC technology so that it can efficiently reprogram cells and repair damaged tissues throughout the body.

iPSCs forgo the need for embryos and can be made to match specific patients. This makes them extremely useful in both research and medicine.

Every individual with damaged or diseased tissues could have their own pluripotent stem cells created to replace or repair them. Of course, more research is needed before that becomes a reality. To date, the use of iPSCs in therapeutic transplants has been very limited.

One of the most significant areas where iPSCs are currently being used is in cardiac research. With appropriate nutrients and inducers, iPSC can be programmed to differentiate into any cell type of the body, including cardiomyocyte. This heart-specific cell can then serve as a great model for therapeutic drug screening or assay development.

Another notable application of iPSCs in cardiac research is optical mapping technology. Optical mapping technology employs high-speed cameras and fluorescence microscopy to examines the etiology and therapy of cardiac arrhythmias in a patient-like environment. This is typically done by looking into electrical properties of multicellular cardiac preparations., e.g. action potential or calcium transient, at high spatiotemporal resolution (6).

Optical mapping technology can correctly record or acquire data from iPSCs. iPSCs are also useful in mimicking a patients cardiomyocytes with their specific behaviors, resulting in more reliable and quality data of cardiac diseases.

iPSCs are vital tools in cardiac research for the following reasons:

iPSCs are patient-specific because they are 100% genetically identical with their donors. This genomic make-up allows researchers to study patients pathology further and develop therapeutic agents for treating their cardiac diseases.

Induced pluripotent stem cell-derived cardiomyocytes (iPSC-CMs), help researchers predict the cardiotoxicity of drugs like with widely used chemotherapy reagents (10). Predictions like this were close to impossible before iPSC technology entered the research game.

iPSCs really come into play with their ability to model diseases. Because iPSCs are genetic matches to their living donors, they are uniquely useful for the study of genetic cardiac diseases like monogenic disorders. iPSCs help researchers understand how disease genotypes at the genetic level manifest as phenotypes at the cellular level (5).

Long QT syndrome, a condition that affects the repolarization of a patients heart after a heartbeat, is a notable example of iPSC-based disease modeling (7). This syndrome has been successfully modeled using iPSCs and is an excellent model for other promising target diseases (7).

Long QT syndrome is not the only disease that has been modeled by iPSCs. Other cardiac diseases like Barth syndrome-associated cardiomyopathy and drug-induced kidney glomerular injuries have been modeled as well (8).

The advent of iPSC technology has created a wealth of new opportunities and applications in cardiovascular research and treatments. In the near future, researchers hope that iPSC-derived therapies will be an option for thousands, if not millions of patients worldwide.

More from this author: The Promising Future of Nanomedicine and Nanoparticles

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What is the Value of iPSC Technology in Cardiac... - The Doctor Weighs In

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The U.S. Food and Drug Administration (FDA) announced on Tuesday that it has approved dapagliflozin, also known under the brand name Farxiga, for the treatment of heart failure in adults with reduced ejection fraction. The drug can potentially reduce the risk of cardiovascular death and hospitalization for heart failure.

AstraZenecas Farxiga is now the first in its drug class of sodium-glucose co-transporter 2 (SGLT2) inhibitors to be approved to treat adults with the New York Heart Associations functional class II-IV heart failure with reduced ejection fraction. AstraZeneca was granted with the approval of Farxiga related to heart failure by the FDA.

In a clinical trial, Farxiga appeared to improve survival and reduce the need for hospitalization in adults with heart failure and reduced ejection fraction.

To determine the efficacy of the drug, researchers looked at the number of instances of cardiovascular death, hospitalization for heart failure and urgent heart failure visits. Some trial participants were given a once-daily dose of 10mg of Farxiga, while others were given a placebo. After approximately 18 months, those who were given Farxiga had fewer cardiovascular deaths, hospitalizations for heart failure and urgent heart failure visits compared to their counterparts.

Heart failure is a serious health condition that contributes to one in eight deaths in the U.S. and impacts nearly 6.5 million Americans, said Norman Stockbridge, M.D., Ph.D., director of the Division of Cardiology and Nephrology in the FDAs Center for Drug Evaluation and Research. This approval provides patients with heart failure with reduced ejection fraction an additional treatment option that can improve survival and reduce the need for hospitalization.

Farxiga can cause side effects including dehydration, urinary tract infections and genetical yeast infections. It can also potentially result in serious cases of necrotizing fasciitis of the perineum in people with diabetes and low blood sugar when combined with insulin.

On Tuesday, BioCardia, Inc. also announced positive preclinical data supporting its new drug application for anti-inflammatory cell therapy for heart failure. BioCardias allogenic neurokinin 1 receptor positive mesenchymal stem cell (NK1R+ MSC) therapy appeared to improve heart function in a study. NK1R+ MSC is being marketed under the name CardiALLO.

Researchers looked at 26 animals treated with both low dose and high dose CardiALLO in their study. Echocardiographic measures of cardiac ejection fraction, fractional shortening and cardiac outflow all notably improved in the animals.

In light of these positive data on our allogenic NK1R+ MSC therapy, we expect to meet our internal timeline to complete our submission to the FDA for our first indication for CardiALLO, and potentially receive IND acceptance by the end of the second quarter, said BioCardia Chief Scientific Officer Ian McNiece, PhD. The MSCs that were studied are subtypes of MSC that we have delivered previously in our co-sponsored trials, which we believe have enhanced potency over MSC generated from unselected bone marrow cells. We look forward to seeing additional data from this animal study that are currently being analyzed, including histology and pathology of the heart and lungs.

BioCardia also intends to submit an IND for the use of NK1R+ MSC delivered via intravenous infusion for the treatment of Acute Respiratory Distress Syndrome caused by COVID-19.

Approximately 6.5 million adults in the U.S. are living with heart failure, according to the Centers for Disease Control and Protection. In 2017, it was a contributing cause of death in one out of eight people.

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FDA Approves AstraZeneca's Farxiga for Heart Failure in Adults with Reduced Ejection Fraction - BioSpace

(MENAFN - iCrowdNewsWire) May 8, 2020

According to the new market research report " Cell Therapy Technologies Market by Product (Consumables, Equipment, Software), Cell Type (Human Stem & Differentiated, Animal), Process Stages (Cell Processing, Distribution, Handling, QC), End User, and Region - Global Forecast to 2023, , published by MarketsandMarkets, The global cell therapy technologies market is projected to reach USD 19.9 billion by 2023 from USD 10.2 billion in 2018, at a CAGR of 14.4% during the forecast period.

Browse in-depth TOC on 'Cell Therapy Technologies Market" 75 - Table30 Figures116 Pages

Download PDF Brochure: https://www.marketsandmarkets.com/pdfdownloadNew.asp?id=213334978

Rising government investments for cell-based research, the increasing number of GMP-certified production facilities, and the large number of oncology-oriented cell-based therapy clinical trials are the key factors driving the growth of this market. China, India, Japan, Korea, and Brazil are emerging markets for cell therapy instruments. These markets boast comparatively lenient standards and government regulations as opposed to developed markets in North America and the EU, and thus offer significant growth potential for providers. However, the high cost of cell-based research and the low success rate is expected to restrain market growth to some extent during the forecast period.

Consumables are expected to account for the largest cell therapy technologies market share in 2018 : By product, the cell therapy technologies market is segmented into consumables, equipment, and systems & software. The consumables segment is expected to account for the largest share of the market in 2018. Factors such as increasing investments by companies to develop advanced products as well as government initiatives for enhancing cell-based research are contributing to the growth of the cell therapy consumables market.

Cell processing segment to witness the highest growth during the forecast period :

Based on process, the cell therapy technologies market is segmented into cell processing; cell preservation, distribution, and handling; and process monitoring and quality control. The cell processing segment is expected to account for the largest market share in 2018 and is projected to witness the highest CAGR during the forecasted period.

Human cells segment accounts for the large share of the cell therapy instruments market, by cell type :

Based on cell type, the market is segmented into human cells and animal cells. In 2018, the human cells segment is expected to account for the largest share of the cell therapy technologies market. The rising adoption of human cells over animal cells for cell therapeutics research, technological advancements, and the rising incidence of diseases such as cancer and cardiac abnormalities are the key factors driving the growth of this segment.

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North America to dominate the cell therapy technologies market during the forecast period : The market is segmented into four major regions, namely, North America, Europe, Asia Pacific, and the Rest of the World (RoW). North America is expected to dominate the market in 2018 owing to the high burden of chronic diseases and increasing R & D activities in the pharmaceutical and biotechnology industries. The Asia Pacific region is expected to register the highest CAGR during the forecast period.

The major players in the western blotting market are Beckman Coulter (US), Becton, Dickinson and Company (US), GE Healthcare (US), Lonza (Switzerland), Merck KGaA (Germany), Miltenyi Biotec (Germany), STEMCELL Technologies, Inc. (Canada), Terumo BCT (US), and Thermo Fisher Scientific (US).

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Cell Therapy Technologies Market to Receive Overwhelming Hike in Revenues by 2023 - MENAFN.COM

Pratteln, Switzerland, May06, 2020 Santhera Pharmaceuticals (SIX: SANN) announces the signing of two agreements with Rutgers, The State University of New Jersey as part of its program to advance gene therapy research for the treatment of LAMA2-deficient congenital muscular dystrophy (LAMA2MD or MDC1A). Under the agreements, Santhera gains rights to intellectual property developed at Rutgers on certain gene constructs that will be further studied under a collaboration agreement.

Santhera has entered into a license agreement with Rutgers, The State University of New Jersey and a collaboration with Prof. Peter Yurchenco, a pioneer in a novel gene therapy approach for the treatment of LAMA2MD. These agreements complement the ongoing collaboration of Santhera with Prof. Markus Regg from the Biozentrum of the University of Basel [1]. Previous collaborative work by Prof. Regg and Prof. Yurchenco has established the potential of this approach in animal models.

The novel gene therapy strategy developed by these leading experts uses two linker proteins that are composed of domains derived from extracellular matrix proteins agrin, laminin and nidogen [2-5]. In animal models for LAMA2MD, this approach has led to restoration of muscle fiber basement membranes, recovery of muscle force and size, increased overall body weight and markedly prolonged survival thus demonstrating strong evidence for disease modifying potential [2].

The coordinated work of both collaborations will further advance Santheras effort to bring this innovative gene therapy approach to patients with LAMA2MD.

Gene replacement is a promising therapeutic option for the treatment of LAMA2MD, said Peter D. Yurchenco, MD, PhD, Professor at Rutgers Robert Wood Johnson Medical School, USA. We have been working on continuously optimizing linker proteins engineered from extracellular matrix proteins which will aid in advancing such gene therapy approach towards clinical use.

Santhera is excited to extend its collaborative network for this therapeutic approach, now including experts from Rutgers University, added Kristina Sjblom Nygren, MD, Chief Medical Officer and Head of Development of Santhera. This will add value to our gene therapy program for LAMA2MD and complements the work already under way with the Biozentrum at the University of Basel, which was awarded a grant by Innosuisse in 2019. Both of our collaboration partners have pioneered this field and will work closely with Santhera, clinical experts and the patient community to establish the best way to bring this approach to clinical use.

About LAMA2MD (CMD Type 1A or MDC1A) and Emerging Therapy ApproachesCongenital muscular dystrophies (CMDs) are inherited neuromuscular diseases characterized by early-onset weakness and hypotonia alongside associated dystrophic findings in muscle biopsy. Progressive muscle weakness, joint contractures and respiratory insufficiency characterize most CMDs. Laminins are proteins of the extracellular matrix that help maintain muscle fiber stability by binding to other proteins. LAMA2-related muscular dystrophy (LAMA2MD, also called MDC1A), is one of the most common forms of CMD. It is caused by mutations in the LAMA2 gene encoding the alpha2 subunit of laminin-211. Most LAMA2MD patients show complete absence of laminin-alpha 2, are hypotonic (floppy) at birth, fail to ambulate, and succumb to respiratory complications.

Previous work has demonstrated that two linker proteins, engineered with domains derived from the extracellular matrix proteins agrin, laminin and nidogen, could compensate for the lack of laminin-alpha2 and restore the muscle basement membrane [2-5]. Through simultaneous expression of artificial linkers (SEAL), this gene therapy approach aims to overcome the genetic defect by substituting laminin-alpha2 deficiency with small linker proteins containing necessary binding domains to re-establish muscle fiber integrity. In a transgenic mouse model, the linker expression increased the lifespan of LAMA2-deficient mice 5-fold to a median of 81 weeks compared to 15.5 weeks in the disease model without the therapeutic linker expression [2]. Recently, it was demonstrated that such linker constructs could be applied by standard adeno-associated virus (AAV) vectors [6, 7]. First results using the AAV technology have been presented by Prof Regg [8].

References [1] Santhera press release on gene collaboration with Biozentrum Basel (May 21, 2019), accessible here

[2] Reinhard et al. (2017). Sci Transl Med 9, eaal4649[3] Moll et al. (2001). Nature 413, 302-307.

[4] Meinen et al. (2007) J. Cell Biol. 176, 979-993.[5] McKee et al. (2017) J. Clin. Invest. 127, 1075-1089.[6] Qiao et al. (2018) Mol Ther Methods Clin Dev 9, 47-56.

[7] Qiao et al. (2005) Proc. Natl. Acad. Sci. U. S. A. 102, 11999-12004.[8] Reinhard, J. et al. (2019) Neuromuscular Disorders, Volume 29, S164

About Rutgers, The State University of New JerseyRutgers, The State University of New Jersey, is a leading national research university and the state of New Jerseys preeminent, comprehensive public institution of higher education. Established in 1766, the university is the eighth-oldest higher education institution in the United States. More than 71,000 students and 23,000 faculty and staff learn, work and serve the public at Rutgers University-New Brunswick, Rutgers University-Newark, Rutgers University-Camden, and Rutgers Biomedical and Health Sciences.

About Santhera Santhera Pharmaceuticals (SIX: SANN) is a Swiss specialty pharmaceutical company focused on the development and commercialization of innovative medicines for rare neuromuscular and pulmonary diseases with high unmet medical need. Santhera is building a Duchenne muscular dystrophy (DMD) product portfolio to treat patients irrespective of causative mutations, disease stage or age. A marketing authorization application for Puldysa (idebenone) is currently under review by the European Medicines Agency. Santhera has an option to license vamorolone, a first-in-class anti-inflammatory drug candidate with novel mode of action, currently investigated in a pivotal study in patients with DMD to replace standard corticosteroids. The clinical stage pipeline also includes lonodelestat (POL6014) to treat cystic fibrosis (CF) and other neutrophilic pulmonary diseases, as well as omigapil and an exploratory gene therapy approach targeting congenital muscular dystrophies. Santhera out-licensed ex-North American rights to its first approved product, Raxone (idebenone), for the treatment of Leber's hereditary optic neuropathy (LHON) to Chiesi Group. For further information, please visit http://www.santhera.com.

Raxone and Puldysa are trademarks of Santhera Pharmaceuticals.

For further information please contact: public-relations@santhera.com orEva Kalias, Head External CommunicationsPhone: +41 79 875 27 80eva.kalias@santhera.com

Disclaimer / Forward-looking statements This communication does not constitute an offer or invitation to subscribe for or purchase any securities of Santhera Pharmaceuticals Holding AG. This publication may contain certain forward-looking statements concerning the Company and its business. Such statements involve certain risks, uncertainties and other factors which could cause the actual results, financial condition, performance or achievements of the Company to be materially different from those expressed or implied by such statements. Readers should therefore not place undue reliance on these statements, particularly not in connection with any contract or investment decision. The Company disclaims any obligation to update these forward-looking statements.# # #

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Santhera Signs Agreements in Gene Therapy Research for Congenital Muscular Dystrophy with Rutgers University - GlobeNewswire

For Immediate Release: May 08, 2020

Today, the U.S. Food and Drug Administration approved Retevmo (selpercatinib) capsules to treat three types of tumors non-small cell lung cancer, medullary thyroid cancer and other types of thyroid cancers in patients whose tumors have an alteration (mutation or fusion) in a specific gene (RET or rearranged during transfection). Retevmo is the first therapy approved specifically for cancer patients with the RET gene alterations.

Innovations in gene-specific therapies continue to advance the practice of medicine at a rapid pace and offer options to patients who previously had few, said Richard Pazdur, M.D., director of the FDAs Oncology Center of Excellence and acting director of the Office of Oncologic Diseases in the FDAs Center for Drug Evaluation and Research. The FDA is committed to reviewing treatments like Retevmo that are targeted to specific subsets of patients with cancer.

Specifically, the cancers that Retevmo is approved to treat include:

Retevmo is a kinase inhibitor, meaning it blocks a type of enzyme (kinase) and helps prevent the cancer cells from growing. Before beginning treatment, the identification of a RET gene alteration must be determined using laboratory testing.

The FDA approved Retevmo on the results of a clinical trial involving patients with each of the three types of tumors. During the clinical trial, patients received 160 mg Retevmo orally twice daily until disease progression or unacceptable toxicity. The major efficacy outcome measures were overall response rate (ORR), which reflects the percentage of patients that had a certain amount of tumor shrinkage, and duration of response (DOR).

Efficacy for NSCLC was evaluated in 105 adult patients with RET fusion-positive NSCLC who were previously treated with platinum chemotherapy. The ORR for the 105 patients was 64%. For 81% of patients who had a response to the treatment, their response lasted at least six months. Efficacy was also evaluated in 39 patients with RET fusion-positive NSCLC who had never undergone treatment. The ORR for these patients was 84%. For 58% of patients who had a response to the treatment, their response lasted at least six months.

Efficacy for MTC in adults and pediatric patients was evaluated in those 12 years of age and older with RET-mutant MTC. The study enrolled 143 patients with advanced or metastatic RET-mutant MTC who had been previously treated with cabozantinib, vandetanib or both (types of chemotherapy), and patients with advanced or metastatic RET-mutant MTC who had not received prior treatment with cabozantinib or vandetanib. The ORR for the 55 previously treated patients was 69%. For 76% of patients who had a response to the treatment, their response lasted at least six months. Efficacy was also evaluated in 88 patients who had not been previously treated with an approved therapy for MTC. The ORR for these patients was 73%. For 61% of patients who had a response to the treatment, their response lasted at least six months.

Efficacy for RET fusion-positive thyroid cancer was evaluated in adults and pediatric patients 12 years of age and older. The study enrolled 19 patients with RET fusion-positive thyroid cancer who were radioactive iodine-refractory (RAI, if an appropriate treatment option) and had received another prior systemic treatment, and eight patients with RET fusion-positive thyroid cancer who were RAI-refractory and had not received any additional therapy. The ORR for the 19 previously treated patients was 79%. For 87% of patients who had a response to the treatment, their response lasted at least six months. Efficacy was also evaluated in eight patients who had not received therapy other than RAI. The ORR for these patients was 100%. For 75% of patients who had a response to the treatment, their response lasted at least six months.

The most common side effects with Retevmo were increased aspartate aminotransferase (AST) and alanine aminotransferase (ALT) enzymes in the liver, increased blood sugar, decreased white blood cell count, decreased albumin in the blood, decreased calcium in the blood, dry mouth, diarrhea, increased creatinine (which can measure kidney function), increased alkaline phosphatase (an enzyme found in the liver and bones), hypertension, fatigue, swelling in the body or limbs, low blood platelet count, increased cholesterol, rash, constipation and decreased sodium in the blood.

Retevmo can cause serious side effects including hepatotoxicity (liver damage or injury), elevated blood pressure, QT prolongation (the heart muscle takes longer than normal to recharge between beats), bleeding and allergic reactions. If a patient experiences hepatotoxicity, Retevmo should be withheld, dose reduced or permanently discontinued. Patients undergoing surgery should tell their doctor as drugs similar to Retevmo have caused problems with wound healing.

Retevmo may cause harm to a developing fetus or a newborn baby. Health care professionals should advise pregnant women of this risk and should advise both females of reproductive potential and males patients with female partners of reproductive potential to use effective contraception during treatment with Retevmo and for one week after the last dose. Additionally, women should not breastfeed while on Retevmo.

Retevmo was approved under the Accelerated Approval pathway, which provides for the approval of drugs that treat serious or life-threatening diseases and generally provide a meaningful advantage over existing treatments. The FDA also granted this application Priority Review and Breakthrough Therapy designation, which expedites the development and review of drugs that are intended to treat a serious condition, when preliminary clinical evidence indicates that the drug may demonstrate substantial improvement over available therapies. Additionally, Retevmo received Orphan Drug designation, which provides incentives to assist and encourage the development of drugs for rare diseases.

The FDA granted approval of Retevmo to Loxo Oncology, Inc., a subsidiary of Eli Lilly and Company.

The FDA, an agency within the U.S. Department of Health and Human Services, protects the public health by assuring the safety, effectiveness, and security of human and veterinary drugs, vaccines and other biological products for human use, and medical devices. The agency also is responsible for the safety and security of our nations food supply, cosmetics, dietary supplements, products that give off electronic radiation, and for regulating tobacco products.

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FDA Approves First Therapy for Patients with Lung and Thyroid Cancers with a Certain Genetic Mutation or Fusion - FDA.gov

It works like a very fine "molecular knob" able to modulate the electrical activity of the neurons of our cerebral cortex, crucial to the functioning of our brain. Its name is Foxg1, it is a gene, and its unprecedented role is the protagonist of the discovery just published on the journal Cerebral Cortex. Foxg1 was already known for being a "master gene" able to coordinate the action of hundreds of other genes necessary for the development of our anterior central nervous system. As this new study reports, the "excitability" of neurons, namely their ability to respond to stimuli, communicating between each other and carrying out all their tasks, also depends on this gene. To discover this, the researchers developed and studied animal and cellular models in which Foxg1 has an artificially altered activity: a lack of activity, as it happens in patients affected by a rare variant of Rett Syndrome, which leads to clinical manifestations of the autistic realm; or an excessive action, as in a specific variant of the West Syndrome, with neurological symptoms such as serious epilepsy and severe cognitive impairment. As deduced by the scientists in the research, the flaw in the "knob" lies in an altered electrical activity in the brain with important consequences for the entire system, similar to what happens in the two syndromes mentioned.

Shedding light on this mechanism, say the researchers, allows to understand more deeply the functioning of our central nervous system in sickness and in health, a fundamental step to assess possible future therapeutic interventions for these pathologies. What has just been published is the latest in a series of three studies on the Foxg1 gene, recently published by the researchers of SISSA on Cerebral Cortex. It is the result of a project begun more than five years ago, which saw the team of Professor Antonello Mallamaci of SISSA in the front line with researchers of the University of Trento and the Neuroscience Institute of Pisa, with the support of the Telethon Foundation, of the Fondation Jerome Lejeune and of the FOXG1 Research Foundation.

The many abilities of the "master gene"

"We knew that this gene is important for the development of the anterior central nervous system" explains the Professor Antonello Mallamaci of SISSA, who has coordinated the research. "In previous studies we had already highlighted how it was involved in the development of particular brain cells, the astrocytes, as well as the neuronal dendrites, which are part of the nerve cells that transport the incoming electrical signal to the cell. The fact that it had mutated in patients affected by specific variants of the Rett and West Syndromes in which we see, respectively, an insufficient and excessive activity of this gene, made us explore the possibility that its role was also another. And, from what has emerged, it would appear that way".

The research findings

According to the study, the activation of the electrical activity of Foxg1 follows a positive circuit. Professor Mallamaci explains: "If the gene is very active there is increased electrical activity in the cerebral cortex. In addition, the neurons, when active, tend to make it work even harder. One process, in short, feeds the other. Obviously, in normal conditions, the system is slowed down at a certain point. "If, however, the gene functions abnormally, or it is found in a number of copies other than two, as it happens in the two syndromes above, the point of balance changes and the electrical activity is altered. All this, in addition to making us understand the mechanisms of the pathology, tells us that Foxg1 functions precisely as a key regulator of the electrical activity in the cerebral cortex".

The next step, explains the professor, will be to understand the role of the mediating genes, namely of some of the many genes whose action is regulated by the master gene Foxg1. This analysis is important to understand in more detail how this gene works under normal and pathological conditions.

How the master gene produces the pathological effects, when and how to intervene

Understanding the molecular mechanisms that Foxg1 controls is also important to study what could be the targets on which to intervene for possible therapeutic approaches. "Given that finding a therapy for these illnesses is very difficult, working so in depth you might find, for example, that most problems are caused precisely by some of the "operators" that Foxg1 regulates. And that we should therefore focus our attention on these goals, rather than on the master gene, maybe using drugs that already exist and have been seen to be useful in remedying those specific flaws". In the case of a future approach that would instead correct the anomalies of the FOXG1 gene with the gene therapy, explains Professor Mallamaci, "it is necessary to understand when to intervene, namely from what moment on the pathological effects due to the mutation of this gene become irreversible. To replace the flawed copy with the correct one, it is necessary to intervene before that moment, which might suppose you would have to make a prenatal gene diagnosis and treatment". "The next steps we will take", concludes Professor Mallamaci "will be directed precisely in the direction of a deeper understanding of all these aspects".

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Like a molecular knob: That is how a gene controls the electrical activity of the brain - Science Codex

This is an excerpt fromSecond Opinion, aweeklyroundup of eclectic and under-the-radar health and medical science news emailed to subscribers every Saturday morning.If you haven't subscribed yet, you can do that byclicking here.

As researchers around the world race under immense pressure to develop a COVID-19 vaccine, a controversial approach could potentially help get them there faster but it's incredibly risky.

The process is known as a human challenge study, and it involves intentionally infecting willing volunteers with the coronavirus that causes COVID-19 in order to test the effectiveness of potential vaccines and treatments against it.

"These are very powerful studies that could make a difference, especially since we don't know a lot about the novel coronavirus," said Seema Shah, a medical ethicist at Northwestern University and Lurie Children's Hospital of Chicago.

"They could clarify what's happening in infections with people who are not symptomatic and people who have more severe disease even if they don't have underlying conditions that put them at higher risk."

Shah said COVID-19 human challenge studies could also help identify people who have developed immunity to the coronavirus, while also helping to narrow down the growing list of potential vaccines and treatments for patients.

"If there were a couple of vaccine candidates that had gotten through safety testing and there was a question about which one of those vaccines was more likely to work, a human challenge study could be a quick way to pick the best vaccine of the candidates," she said.

"It could also be useful to study whether the vaccine itself causes different kinds of harm."

The WHOsays any potential vaccine is still at least a year away, but human challenge trials could accelerate theprocess because of the time they save in the clinical trial phase.

Typically, researchers inject thousands of study participants with a vaccine or placebo and wait for symptoms to develop an approach that can take months or years after vaccine development.

Human challenge studies instead vaccinatea small group of people and then intentionally infectthem with the virus, saving critical research time, especially during a global pandemic.

But despite the potential benefits of a controlled human challenge study on COVID-19 patients, experts say the controversial approach is an ethical minefield that could have disastrous consequences if not handled carefully.

Human challenge studies typically recruit young, healthy volunteers in an effort to keep the risk of severe medical complications low.

Thousands of potential volunteers have already pledged to participate in human challenge trials on a website called 1DaySooner, but no such studies are yet underway.

Yet given what we know and don't know about the different ways in which COVID-19 attacks the human body even in young, healthy people, how do we effectively inform participants who may be unknowingly putting themselves at severe risk?

"We know that younger people tend to tolerate COVID-19 as an illness better, but what would worry me about that is there's a lot that is still unknown," said Kerry Bowman, a bioethicist and professor of global health at the University of Toronto.

He said the potential for COVID-19 patients of all ages to face long-term health implications and even death from a virus we still know so little aboutcalls into question how truly informed participants could be on the risks of a human challenge study.

"Do you truly have an informed decision?" he said. "You have consent, but is itreally well-informed? Do people fully understand? Because if we don't understand the virus itself, I wonder about the quality of informed consent that you can ask of people."

In a new paper published in the journal Science Thursday, Shah and a team of international researchers outline an ethical framework for how human challenge studies could be effectively used to combat COVID-19.

The researchers supportdeveloping a "challenge strain" of the coronavirus a stabilizedversion of the one thatis circulating worldwide to potentially infect participants, but stopped short of advocating for the work to proceed.

"The pandemic has affected just about everyone in the world in various ways, so the potential amount of social value is unprecedented here," Shah said.

"That's why our group concluded it's really important to give challenge studies a hard look and potentially invest in laying the groundwork for doing them.

"But then make that judgment call about whether and how to do them at a later date when they're ready to go."

The World Health Organization released specific criteria this week outlining its recommended approach to conducting human challenge studies, without advocating for or against them.

Among those recommendations is a need for "strong scientific justification" for the studies, ensuring that the potential benefits outweigh risksand that the selection of participants should be done with "rigorous" informed consent.

"The overarching, really important one is to minimize risk to participants as much as possible," said bioethicist Dr. Ross Upshur, of the University of Toronto's Dalla Lana School of Public Health, who helped work on the WHO guidelines.

"You need to make sure that people understand what's being proposed:what they're going to be doing;how they're going to be managed in this situation;how their safety and their well-being is going to be protected.

"But it's also incumbent on the researchers to outline all of the uncertainties,because we may not be able to actually quantify some of those risks."

Timothy Caulfield, a Canada Research Chair in health law and policy at the University of Alberta who has researched human challenge studies, said those ethical dilemmas have historically plagued this approach.

"I understand the desire to use human challenge trials, especially in this context, because people are desperate to get a vaccine quickly, not just for clinical reasons, but also for economic and social reasons," he said.

"So the pressure is intense, but the exact reason that we have research ethics guidelines is to protect research participants."

Caulfield said the damage that could be done if a human challenge trial were to go awry would be devastating.

He points to the ethics scandal involvingJesse Gelsinger, a teenager who died in a clinical trial for gene therapy in 1999, as an example of a failed human study that set the research ethics field back immensely.

Gelsingerhad a genetic disease called ornithine transcarbamylase (OTC) that he controlled through diet and medication, but after enrolling in the trialhe was injected with an experimental therapy and died a few days later.

"Just imagine the impact that it could have on vaccine research, especially in this environment where there's so much uncertainty," Caulfieldsaid.

"If it doesn't go well and if we cut corners on research ethics standards, it could end up backfiring and being really problematic."

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Could intentionally infecting volunteers with COVID-19 help find a cure sooner? - CBC.ca

Global Gene Therapy for Inherited Genetic Disorder Market statistical report provides a wide-ranging research on the key players and in-depth insights which includes the competitiveness of the trending players. This Market research report that evaluates its current value, size, performance and statistics. The report is an important dynamic of the market and gives an idea of the types, the process, and value chain that has been included in the report.

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BioMarin Pharmaceutical Inc., bluebird bio Inc., Novartis AG, Orchard Therapeutics Plc, and Spark Therapeutics.

Key questions answered in this report:

Geographically, regions such as North America, Europe, Asia-Pacific (APAC), Middle East & Africa and Latin America can be segmented on the basis of the global Gene Therapy for Inherited Genetic Disorder Market. Market drivers, restraints, and opportunities have been evaluated to explain the anticipated nature of investments and its impact on the global market in terms of future prospects. The report provides qualitative as well as quantitative researched data of the global Market.

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In the last sections of the report, the recent innovations along with its impact on innovative growth that is expected to be introduced by the significant players form a core part of the report. This study is done by considering the demographics, regional trends, product demand evaluation in a forward-looking perspective on different factors driving or restraining market growth.

Table of Contents

Global Gene Therapy for Inherited Genetic Disorder Market Research Report

Chapter 1 Gene Therapy for Inherited Genetic Disorder Market Overview

Chapter 2 Global Economic Impact on Industry

Chapter 3 Global Market Competition by Manufacturers

Chapter 4 Global Production, Revenue (Value) by Region

Chapter 5 Global Supply (Production), Consumption, Export, Import by Regions

Chapter 6 Global Production, Revenue (Value), Price Trend by Type

Chapter 7 Global Market Analysis by Application

Chapter 8 Manufacturing Cost Analysis

Chapter 9 Industrial Chain, Sourcing Strategy and Downstream Buyers

Chapter 10 Marketing Strategy Analysis, Distributors/Traders

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How COVID-19 Impacting On Gene Therapy for Inherited Genetic Disorder Market Trends, Drivers, Strategies, Segmentation Application with Top Key...

BURLINGTON, Mass., May 05, 2020 (GLOBE NEWSWIRE) -- Flexion Therapeutics, Inc. (Nasdaq:FLXN) will present positive data from two studies of FX201, an investigational gene therapy for osteoarthritis (OA), at the American Society of Gene and Cell Therapy (ASGCT) Annual Meeting taking place virtually May 12-15, 2020. The abstracts were published in the May supplement of Molecular Therapy.

The data from the rodent model served as a basis for the FX201 IND and reinforce our belief that FX201 holds the potential to become a transformative therapy for OA, saidMichael Clayman, M.D., President and Chief Executive Officer of Flexion. Further, we are delighted to be presenting manufacturing studies at ASGCT which demonstrate a capable Good Manufacturing Process (GMP) to support the production of FX201 for our clinical trials.

Establishing the Efficacy, Safety, and Biodistribution of FX201, a Helper-Dependent Adenoviral Gene Therapy for the Treatment of Osteoarthritis, in a Rat Model (Abstract 747)

Flexion assessed the efficacy of HDAd-rat-IL-1Ra, the rat surrogate of FX201, in a rodent surgical model of OA. Efficacy was assessed after a single intra-articular administration one week post-surgery via histopathology at Week 12 using a semi-quantitative microscopic grading system (Osteoarthritis Research Society International [OARSI] score) for OA-related cartilage, synovium, and bone changes. In addition, two Good Laboratory Practice (GLP) studies were performed in a rodent model of OA to evaluate the safety and biodistribution of FX201 and the rat surrogate (a helper-dependent adenovirus vector with a transgene encoding rat variant of IL-1Ra) administered four weeks post-surgery. Key study findings include:

Development of a Highly Productive and Reproducible Manufacturing Process for FX201, a Novel Helper-Dependent Adenovirus-Based Gene Therapy for Osteoarthritis (Abstract 1273)

Using a fit-for-purpose manufacturing process suitable for early development, Flexion successfully produced four batches of drug substance, which will enable GLP toxicology, pharmacology, and GMP clinical studies. Key findings include:

About FX201FX201 is a locally administered gene therapy product candidate which utilizes a helper-dependent adenovirus (HDAd) vector, designed to stimulate the production of an anti-inflammatory protein, interleukin-1 receptor antagonist (IL-1Ra), whenever inflammation is present within the joint. Inflammation is a known cause of pain, and chronic inflammation is thought to play a major role in the progression of osteoarthritis (OA). By persistently suppressing inflammation, Flexion believes FX201 holds the potential to both reduce OA pain and modify the disease.

About Osteoarthritis (OA) of the KneeOA, also known as degenerative joint disease, affects more than 30 million Americans and accounts for more than $185 billion in annual expenditures. In 2017, approximately 15 million Americans were diagnosed with OA of the knee and the average age of physician-diagnosed knee OA has fallen by 16 years, from 72 in the 1990s to 56 in the 2010s. The prevalence of OA is expected to continue to increase as a result of aging, obesity and sports injuries. Each year, approximately five million OA patients receive either a corticosteroid (immediate-release or extended-release) or hyaluronic acid intra-articular injection to manage their knee pain.

About Flexion TherapeuticsFlexion Therapeutics(Nasdaq:FLXN) is a biopharmaceutical company focused on the development and commercialization of novel, local therapies for the treatment of patients with musculoskeletal conditions, beginning with osteoarthritis, the most common form of arthritis. The company's core values are focus, ingenuity, tenacity, transparency and fun. Visitflexiontherapeutics.com.

Forward-Looking Statements This release contains forward-looking statements that are based on the current expectations and beliefs of Flexion. Statements in this press release regarding matters that are not historical facts, including, but not limited to, statements relating to the future of Flexion; expected increases in the rate of individuals with OA of the knee; and the potential therapeutic and other benefits of FX201, are forward looking statements. These forward-looking statements are based on management's expectations and assumptions as of the date of this press release and are subject to numerous risks and uncertainties, which could cause actual results to differ materially from those expressed or implied by such statements. These risks and uncertainties include, without limitation, risks related to clinical trials, including potential delays, safety issues or negative results; risks related to key employees, markets, economic conditions, and health care reform; and other risks and uncertainties described in our filings with theSecurities and Exchange Commission(SEC), including under the heading "Risk Factors" in our Annual Report on Form 10-Kfiled with theSEConMarch 12, 2020and subsequent filings with theSEC. The forward-looking statements in this press release speak only as of the date of this press release, and we undertake no obligation to update or revise any of the statements. We caution investors not to place considerable reliance on the forward-looking statements contained in this press release.

Contacts:

Scott YoungVice President, Corporate Communications & Investor RelationsFlexion Therapeutics, Inc.T: 781-305-7194syoung@flexiontherapeutics.com

Julie DownsAssociate Director, Corporate Communications & Investor Relations Flexion Therapeutics, Inc.T: 781-305-7137jdowns@flexiontherapeutics.com

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Flexion Therapeutics Announces Virtual Poster Presentations for FX201, an Intra-Articular Gene Therapy Candidate for the Treatment of Osteoarthritis,...

SDMR International recently published a report on the Global Viral Vectors and Plasmid DNA Manufacturing Market, which offers an in-depth overview of the factors that are impacting the state and progress of the worldwide business. The research report incorporates all the vital data that will help you updated with the latest market trends and get ahead in the competition in the various market segments and the leading geographies studied in the report. The study gives valuable insights into the future progress of the market and all essential aspects of the Viral Vectors and Plasmid DNA Manufacturing market for the forecast period from 2020 to 2026.

The Viral Vectors and Plasmid DNA Manufacturing Market research report gives the latest market information, highlighting the product range and services offered in the market. The report provides key facts and figures relating to the market status, size, share, and growth aspects of the Viral Vectors and Plasmid DNA Manufacturing industry. The research comprises of elaborate profiles of the leading companies operating in the global sector by taking into account their gross revenue, total sales, market share, and competitive landscape.

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Covid-19 impact on Viral Vectors and Plasmid DNA Manufacturing Market Research Report Analysis and Forecast till 2026 | Cell and Gene Therapy...

Shares of Orchard Therapeutics fell more than 13% in premarket trading after the company announced a new strategic plan that includes the shifting of its clinical focus, the shuttering of a proposed manufacturing facility in California and the termination of 25% of company staff.

On Thursday, London-based Orchard announced the new plan that will shift the companys clinical priorities away from some of its long-time focuses, including a stem cell gene therapy for the treatment of severe combined immune deficiency due to adenosine deaminase deficiency (ADA-SCID) and other ultra-rare conditions. Instead, Orchard said it will now focus its development priorities on more common conditions that include Crohns disease, a type of dementia and some neurometabolic disorders. Among those are the development of a treatment for metachromatic leukodystrophy (MLD), a rare and life-threatening inherited metabolic disease. Another priority will be its treatment for Wiskott-Aldrich syndrome (WAS), a life-threatening immune disorder. Orchard will also continue to develop its treatments for mucopolysaccharidosis types I and IIIa.

The shifts in Orchards plans were announced a couple of months following a management change. In March, company founder and Chief Scientific Officer Bobby Gaspar took over the helm of the company from Mark Rothera, who led Orchard since 2017.

I feel privileged to lead Orchard as we embark on this new chapter, which is rooted in fulfilling the powerful possibilities for HSC gene therapies beyond ultra-rare diseases, Gaspar said in a statement. Moving forward, we are focusing on advancing therapies for high need and high-value diseases, and our work in neurometabolic disorders is a clear example of this. Were also excited to announce new research programs which we believe will demonstrate the breadth of the HSC platform approach.

Orchard said it intends to adjust its manufacturing plans to focus on the expected regulatory approvals of OTL-200, the MLD treatment and its WAS drug, OTL-103. The company hopes to obtain approval in the European Union for OTL-200 for the treatment of MLD in the second half of 2020 and launch in the first half of 2021. Orchard is also preparing a Biologics License Application for its WAS treatment in both the U.S. and EU in 2021.

Orchard said it also intends to stop its plans to build a gene therapy manufacturing facility in Fremont, Calif. That project was first announced in 2018.

The culling of one-fourth of its employees is expected to save the company approximately $125 million through the end of 2021. That will extend the companys existing cash runway into 2022, Orchard said. Cash, cash equivalents and investments as of March 31, were $263.9 million compared to $325.0 million as of Dec. 31, 2019.

Frank Thomas, Orchards chief operations officer, called the strategic shift and layoffs necessary steps for the company to achieve its objectives by matching our attention and resources to a set of core imperatives for the business.

I believe that these are necessary steps, especially in light of the current environment in which we are operating, with focused investments in areas such as commercial and manufacturing operations supporting the needs we have now without a near-term dependence on the capital markets, Thomas said in a statement.

See the rest here:
Orchard Therapeutics Cuts 25% of Staff as it Shifts Clinical Focus - BioSpace

CAMBRIDGE, Mass.--(BUSINESS WIRE)--AVROBIO, Inc. (Nasdaq: AVRO) and Magenta Therapeutics (Nasdaq: MGTA) today announced a research and clinical collaboration agreement to evaluate the potential utility of MGTA-117, Magentas novel targeted antibody-drug conjugate (ADC) for conditioning patients before they receive one of AVROBIOs investigational lentiviral gene therapies.

The collaboration will combine AVROBIOs leadership in lentiviral gene therapies with Magentas expertise in ADC-based conditioning and is expected to further the two companies shared mission to enable patients to live free from disease. Under the collaboration, AVROBIO and Magenta will jointly evaluate MGTA-117 in conjunction with one or more of AVROBIOs investigational gene therapies. Magenta will retain all commercial rights to MGTA-117. AVROBIO will retain all commercial rights to its gene therapies and will be responsible for the clinical trial costs related to the evaluation of MGTA-117 with AVROBIOs gene therapies.

This agreement with Magenta springs from our strategic focus on maintaining technology leadership in gene therapy, said Geoff MacKay, AVROBIOs president and CEO. AVROBIO has always led by investing early in technological innovations that further the field of lentiviral gene therapy, such as plato, our proprietary platform designed to optimize the safety, potency and durability of our investigational lentiviral gene therapies. Were continually assessing new technologies that could be complementary to our plato platform to sustain our cutting-edge advantage and continue to evolve platos capabilities.

We believe targeted ADCs represent the next generation of medicines to prepare patients for gene therapy or transplant in a targeted, precise way. AVROBIOs investigational gene therapies complement our platform as well as our focus and commitment to patients. This partnership will allow Magenta to validate our conditioning platform in lentiviral gene therapy applications, said Jason Gardner, D.Phil., president and chief executive officer, Magenta Therapeutics. Weve selected ADCs as the preferred modality for our conditioning programs, as we believe they offer the most promising option for more patients. We have optimized our ADCs for gene therapy and transplant settings and look forward to collaborating with AVROBIO to evaluate MGTA-117 in specific gene therapy settings. Magenta will continue to develop MGTA-117 in other diseases, including blood cancers and genetic diseases.

MGTA-117, Magentas most advanced conditioning program, is a CD117-targeted antibody engineered for the transplant setting and conjugated to amanitin, a toxin in-licensed from Heidelberg Pharma. It is designed to precisely deplete only hematopoietic stem and progenitor cells and has shown high selectivity, potent efficacy, wide safety margins and broad tolerability in non-human primate models, suggesting that it may be capable of clearing space in bone marrow to support long-term engraftment and rapid recovery in humans. Magenta plans to complete IND-enabling studies this year.

AVROBIO currently uses a personalized conditioning regimen with precision dosing of busulfan, an extensively validated conditioning agent generally considered to be the gold standard for ex vivo lentiviral gene therapy, based on decades of general use and administration to hundreds of patients treated with lentiviral gene therapy candidates. The treating clinician uses therapeutic drug monitoring (TDM) to evaluate how quickly the patient metabolizes busulfan and adjusts the dose regimen accordingly with the goal of achieving the optimum result. AVROBIO has reported early clinical data with this precision conditioning regimen with TDM in its own clinical trials, adding to a body of data that suggest busulfan can effectively clear space in the patients bone marrow, where stem cells engraft, produce generations of daughter cells carrying the therapeutic gene and make the functional protein the patient needs to maintain cellular health.

About AVROBIO

Our mission is to free people from a lifetime of genetic disease with a single dose of gene therapy. We aim to halt or reverse disease throughout the body by driving durable expression of functional protein, even in hard-to-reach tissues and organs including the brain, muscle and bone. Our clinical-stage programs include Fabry disease, Gaucher disease and cystinosis and we also are advancing a program in Pompe disease. AVROBIO is powered by the plato gene therapy platform, our foundation designed to scale gene therapy worldwide. We are headquartered in Cambridge, Mass., with an office in Toronto, Ontario. For additional information, visit avrobio.com, and follow us on Twitter and LinkedIn.

About Magenta Therapeutics

Magenta Therapeutics is a clinical-stage biotechnology company developing medicines to bring the curative power of immune system reset through stem cell transplant to more patients with autoimmune diseases, genetic diseases and blood cancers. Magenta is combining leadership in stem cell biology and biotherapeutics development with clinical and regulatory expertise, a unique business model and broad networks in the stem cell transplant world to revolutionize immune reset for more patients. Magenta is based in Cambridge, Mass. For more information, please visit http://www.magentatx.com. Follow Magenta on Twitter: @magentatx.

AVROBIO Forward-Looking Statements

This press release contains forward-looking statements, including statements made pursuant to the safe harbor provisions of the Private Securities Litigation Reform Act of 1995. These statements may be identified by words and phrases such as aims, anticipates, believes, could, designed to, estimates, expects, forecasts, goal, intends, may, plans, possible, potential, seeks, will, and variations of these words and phrases or similar expressions that are intended to identify forward-looking statements. These forward-looking statements include, without limitation, statements regarding our business strategy for and the potential therapeutic benefits of our prospective product candidates, the design, commencement, enrollment and timing of ongoing or planned clinical trials, clinical trial results, product approvals and regulatory pathways, anticipated benefits of our gene therapy platform including potential impact on our commercialization activities, timing and likelihood of success, the expected benefits and results of our implementation of the plato platform in our clinical trials and gene therapy programs, the expected safety profile of our investigational gene therapies, and the potential and expected benefits of MGTA-117, Magentas investigational antibody-drug conjugate, including the ability of MGTA-117 to deplete hematopoietic stem and progenitor cells in order to clear space in bone marrow to support long-term engraftment in humans, as well as MGTA-117s potential application to AVROBIOs investigational gene therapies. Any such statements in this press release that are not statements of historical fact may be deemed to be forward-looking statements. Results in preclinical or early-stage clinical trials may not be indicative of results from later stage or larger scale clinical trials and do not ensure regulatory approval. You should not place undue reliance on these statements, or the scientific data presented.

Any forward-looking statements in this press release are based on AVROBIOs current expectations, estimates and projections about our industry as well as managements current beliefs and expectations of future events only as of today and are subject to a number of risks and uncertainties that could cause actual results to differ materially and adversely from those set forth in or implied by such forward-looking statements. These risks and uncertainties include, but are not limited to, the risk that any one or more of AVROBIOs product candidates will not be successfully developed or commercialized, the risk of cessation or delay of any ongoing or planned clinical trials of AVROBIO or our collaborators, the risk that AVROBIO may not successfully recruit or enroll a sufficient number of patients for our clinical trials, the risk that AVROBIO may not realize the intended benefits of our gene therapy platform, including the features of our plato platform, the risk that AVROBIO may not realize the intended benefit of MGTA-117 with respect to AVROBIOs investigational gene therapies, the risk that our product candidates or procedures in connection with the administration thereof will not have the safety or efficacy profile that we anticipate, the risk that prior results, such as signals of safety, activity or durability of effect, observed from preclinical or clinical trials, will not be replicated or will not continue in ongoing or future studies or trials involving AVROBIOs product candidates, the risk that we will be unable to obtain and maintain regulatory approval for our product candidates, the risk that the size and growth potential of the market for our product candidates will not materialize as expected, risks associated with our dependence on third-party suppliers and manufacturers, risks regarding the accuracy of our estimates of expenses and future revenue, risks relating to our capital requirements and needs for additional financing, risks relating to clinical trial and business interruptions resulting from the COVID-19 outbreak or similar public health crises, including that such interruptions may materially delay our development timeline and/or increase our development costs or that data collection efforts may be impaired or otherwise impacted by such crises, and risks relating to our ability to obtain and maintain intellectual property protection for our product candidates. For a discussion of these and other risks and uncertainties, and other important factors, any of which could cause AVROBIOs actual results to differ materially and adversely from those contained in the forward-looking statements, see the section entitled Risk Factors in AVROBIOs most recent Annual or Quarterly Report, as well as discussions of potential risks, uncertainties and other important factors in AVROBIOs subsequent filings with the Securities and Exchange Commission. AVROBIO explicitly disclaims any obligation to update any forward-looking statements except to the extent required by law.

Magenta Therapeutics Forward Looking Statements

This press release may contain forward-looking statements and information within the meaning of The Private Securities Litigation Reform Act of 1995 and other federal securities laws, including, without limitation, statements regarding the research and clinical collaboration agreement between Magenta and AVROBIO, including the timing, progress and success of the collaboration contemplated under the agreement, the successful evaluation MGTA-117 in conjunction with one or more of AVROBIOs investigational gene therapies under the agreement, the anticipated cost allocation and other commercial terms under the agreement, Magentas strategy and business plan, as well as the future development, manufacture and commercialization between AVROBIO and Magenta. The use of words such as may, will, could, should, expects, intends, plans, anticipates, believes, estimates, predicts, projects, seeks, endeavor, potential, continue or the negative of such words or other similar expressions can be used to identify forward-looking statements. The express or implied forward-looking statements included in this press release are only predictions and are subject to a number of risks, uncertainties and assumptions, including, without limitation, risks set forth under the caption Risk Factors in Magentas most recent Annual Report on Form 10-K, as updated by Magentas most recent Quarterly Report on Form 10-Q and its other filings with the Securities and Exchange Commission, as well as risks, uncertainties and assumptions regarding the impact of the COVID-19 pandemic to Magentas business, operations, strategy, goals and anticipated timelines, including, without limitation, Magentas ongoing and planned preclinical activities, ability to initiate, enroll, conduct or complete ongoing and planned clinical trials, timelines for regulatory submissions and financial position. In light of these risks, uncertainties and assumptions, the forward-looking events and circumstances discussed in this press release may not occur and actual results could differ materially and adversely from those anticipated or implied in the forward-looking statements. You should not rely upon forward-looking statements as predictions of future events. Although Magenta believes that the expectations reflected in the forward-looking statements are reasonable, it cannot guarantee that the future results, levels of activity, performance or events and circumstances reflected in the forward-looking statements will be achieved or occur. Moreover, except as required by law, neither Magenta nor any other person assumes responsibility for the accuracy and completeness of the forward-looking statements included in this press release. Any forward-looking statement included in this press release speaks only as of the date on which it was made. We undertake no obligation to publicly update or revise any forward-looking statement, whether as a result of new information, future events or otherwise, except as required by law.

View source version on businesswire.com: https://www.businesswire.com/news/home/20200506005265/en/

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AVROBIO and Magenta Therapeutics Announce Collaboration to Evaluate Targeted Antibody-Drug Conjugate as a Potential Conditioning Regimen for...

Rockville May 9, 2020 (Thomson StreetEvents) -- Edited Transcript of Regenxbio Inc earnings conference call or presentation Thursday, May 7, 2020 at 8:30:00pm GMT

* Kenneth T. Mills

REGENXBIO Inc. - CEO, President & Director

* Patrick J. Christmas

REGENXBIO Inc. - Senior VP & Chief Legal Officer

REGENXBIO Inc. - Senior VP & Chief Medical Officer

* Vittal K. Vasista

REGENXBIO Inc. - Senior VP & CFO

Chardan Capital Markets, LLC, Research Division - Director of Research & Head of Healthcare Research

SVB Leerink LLC, Research Division - MD of Genetic Medicines & Senior Research Analyst

Good afternoon, and welcome to the REGENXBIO First Quarter 2020 Earnings Conference Call. (Operator Instructions) As a reminder, this conference call is being recorded. I would now like to turn the call over to Mr. Patrick Christmas, Senior Vice President and General Counsel for REGENXBIO. You may begin.

Patrick J. Christmas, REGENXBIO Inc. - Senior VP & Chief Legal Officer [2]

Good afternoon, and thank you for joining us today. With us are Ken Mills, REGENXBIO's President and Chief Executive Officer; Dr. Steve Pakola, our Chief Medical Officer; and Vit Vasista, our Chief Financial Officer.

Earlier this afternoon, REGENXBIO released financial and operating results for the first quarter ended March 31, 2020. The press release reporting our financial results is available on our website at http://www.regenxbio.com.

Today's conference call will include forward-looking statements regarding our financial outlook in addition to regulatory and product development plans. These forward-looking statements are subject to risks and uncertainties that may cause actual results to differ from those forecasted and can be identified by words such as expect, plan, will, may, anticipate, believe, should, intend and other words of similar meaning. Any such forward-looking statements are not guarantees of future performance and involve certain risks and uncertainties. These risks are described in the Risk Factors and the Management's Discussion and Analysis sections of REGENXBIO's annual report on Form 10-K for the full year ended December 31, 2019, and comparable sections of REGENXBIO's other filings, which are on file with the Securities and Exchange Commission and available on the SEC's website.

Any information we provide on this conference call is provided only as of the date of this call, May 7, 2020. And we undertake no obligation to update any forward-looking statements we may make on this call on account of new information, future events or otherwise. Please be advised that today's call is being recorded and webcast. In addition, any unaudited or pro forma financial information that may be provided is preliminary and does not purport to project financial positions or operating results of the company. Actual results may differ materially.

I would now like to turn the call over to Ken.

Kenneth T. Mills, REGENXBIO Inc. - CEO, President & Director [3]

Thank you, Patrick. Good afternoon, everyone, and thanks for joining us. On today's conference call, we'll provide a recap of our recent progress, advancing and expanding the NAV Technology Platform as well as expected future milestones. Steve will provide an update on our clinical programs, and Vit will provide an update on the financial results for the first quarter of 2020. Then we'll open the call for questions.

First, I want to take a moment to say that I hope everyone is healthy, staying safe during the COVID-19 pandemic. At REGENXBIO, we've made some changes to our business operations in order to support the health and safety of our employees and the community, and we're fortunate that we've been able to successfully advance our business during this time. As always, and especially throughout the past few months, our overall focus remains on the important goal of improving lives through the curative potential of gene therapy, and I'm grateful that our team is dedicated to this pursuit even in these challenging times. Along these lines, we've made important progress in recent months at REGENXBIO as we continue to see the remarkably consistent and durable effects of gene therapy treatment, patients with severe wet AMD.

If you haven't done so already, I highly recommend referring back to our webcast that we hosted on April 22, in which several leading retina specialists joined us to provide their perspectives on our recently announced data. Our RGX-314 gene therapy is designed to enable sustained production of an anti-VEGF antibody fragment in the eye, and we've now demonstrated stable and consistent results out to 2 years in the third dose cohort. We believe this is the longest time line of continuous therapeutic effects demonstrated in wet AMD patients from a single administration of an anti-VEGF treatment.

We also provided additional data from the fifth cohort, which received a higher dose of RGX-314 and in which 73% of patients remain anti-VEGF injection-free 9 months after a onetime administration of RGX-314. In our program, we're thinking carefully of all aspects of clinical management, not just anti-VEGF injections, are cognizant of the variables that might impact patient care and vision. And Steve will provide more details on these results and next steps for the program in his remarks.

Beyond RGX-314, we've continued to drive our internal gene therapy pipeline forward. As we previously announced in February, we presented encouraging initial data at the WORLDSymposium from Cohort 1, the Phase I/II trial for MPS II, and we look forward to providing additional data from these patients in mid-2020. We've begun enrolling patients in Cohort 2, where they're receiving a higher dose of RGX-121 and look forward to providing interim data on Cohort 2 in the second half of 2020.

We anticipate several other important updates this year including from our Phase I/II trial of RGX-501 for the treatment of HoFH, our Phase I/II trial of RGX-111 for the treatment of MPS-I and our RGX-181 program for the treatment of CLN2 disease, as well as our research programs in hereditary angioedema, neurodegenerative and neuromuscular diseases.

Lastly, construction of our GMP production facility here in Rockville continues, and the facility is expected to be operational in 2021. We expect the facility to enable us to strategically scale production while continuing to ensure high quality for patients. So with that, I'll turn the call over to Steve for a clinical and regulatory update.

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Stephen Pakola, REGENXBIO Inc. - Senior VP & Chief Medical Officer [4]

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Thanks, Ken. As you've mentioned, last month, we announced updated data from our Phase I/IIA study of RGX-314 for the treatment of wet AMD. We reported that the gene therapy continued to be well tolerated at all dose levels. And there were no reports of ocular inflammation beyond what is expected following routine vitrectomy. The latest efficacy update was focused on the 2-year data from Cohort 3 of the study. Patients in this cohort received [6 C] 10 genome copies per eye. And now at 2 years after administration of RGX-314, they have demonstrated markedly improved visual acuity and stable retinal thickness as well as significantly reduced need for anti-VEGF intraocular injections and stable protein expression.

50% of patients within this cohort did not receive any anti-VEGF injections over the full 2 years of the study. And one additional subject did not receive injections starting 9 months after the administration of RGX-314. We saw an impressive improvement in visual acuity with an increase of 14 letters in both the full cohort as well as the 4 patients who did not receive anti-VEGF injections in the second year of the study. This improved vision and durability of anti-VEGF activity is particularly meaningful, as real-world evidence has shown us that patients commonly lose vision over time, even with current standard of care.

And finally, Cohort 3 showed consistent protein production over 2 years, giving us confidence that the transduced cells in the retina have been producing the RGX-314 protein at a steady rate throughout the study. We also provided an interim update from Cohort 5. 73% of patients were anti-VEGF injection free over 9 months. We are very pleased with these results and look forward to additional efficacy data at the 1-year time point. We will use this data to then finalize the design of the pivotal program for RGX-314, which we expect to initiate in the second half of 2020. We are also planning to start trials of RGX-314 using the in-office suprachoroidal delivery approach in 2020 and for both wet AMD and diabetic retinopathy. We look forward to providing additional information about these trials over the coming months.

Turning to our rare disease portfolio. Data thus far from our Phase I/II trial of RGX-121 has been encouraging as patients in the first cohort demonstrated consistent and sustained reduction in heparan sulfate in the CSF, and available data support early signs of neurocognitive stability. We look forward to providing additional data from these 3 patients in mid-2020. Meanwhile, enrollment in Cohort 2 at a higher dose level continues and is expected to be complete in the first half of 2020, with interim data expected in the second half of 2020.

Recruitment screening and additional site activations are ongoing in our Phase I/II clinical trial evaluating RGX-111 for the treatment of MPS I. Recruitment in this trial had been previously focused on an initial patient over the age of 18, but the protocol was recently amended to allow enrollment of patients as young as 4 months of age.

We expect to provide a program update in the second half of 2020. We also anticipate updates from our Phase I/II trial of RGX-501 for the treatment of HoFH in the first half of this year. We have several other study programs headed towards the clinic, including the HAE and neuromuscular programs, and I look forward to providing additional details in the coming months. With that, I turn the call back over to Ken. Ken?

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Kenneth T. Mills, REGENXBIO Inc. - CEO, President & Director [5]

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Thanks for that summary, Steve. REGENX has an extensive footprint in the gene therapy space, and we're always purposeful in developing partnerships with key players in the space to continue to realize the potential of NAV Technology. Our NAV Technology is currently being applied in 1 marketed product and more than 20 additional partnered product candidates.

We continue to track the positive progress of Novartis' ZOLGENSMA, which uses the NAV AAV9 vector. Novartis has stated that they're treating about 100 patients per quarter in the U.S. based on their current launch. We're encouraged by the success that the Novartis team in reaching patients. I believe that this is among the most successful launches of gene therapy so far and believe that it demonstrates the transformational impact that NAV Technology can have on the treatment of genetic diseases with significant unmet needs.

We were also pleased to see the positive regulatory developments in Japan and Europe this quarter, which signal additional validation of the technology across the globe. And importantly, for the entire gene therapy field, Novartis recently published additional detailed data for the intrathecal delivery of ZOLGENSMA, which has shown improvement in motor function and achievement of motor skills following treatment. I'm excited for the additional progress to come from this program.

In addition, we recently announced another exclusive worldwide license agreement with Ultragenyx, extending our companies' existing gene therapy partnership. This agreement will enable the Ultragenyx team to apply our NAV technology, AAV8 and AAV9 vectors to the development of a new gene therapy for a rare metabolic disorder, and provides further validation of the breadth and depth of our intellectual property portfolio.

Throughout the remainder of this year, we also anticipate regulatory updates from our partners like Audentes Therapeutics, now part of Astellas, for their gene therapy candidate for x-linked myotubular myopathy, which uses our NAV AAV8 vector. The promising milestones and achievements from our partners, as well as the progress in our own internal pipeline, provide additional validation the proprietary NAV Technology platform and further demonstrate the transformational impact that can come from a onetime administration of gene therapy.

With that summary, I want to turn the call over to Vit for a review of our financials.

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Vittal K. Vasista, REGENXBIO Inc. - Senior VP & CFO [6]

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Thank you, Ken. REGENXBIO ended the quarter on March 31, 2020 with cash, cash equivalents and marketable securities totaling $356.6 million compared to $400 million as of December 31, 2019. The decrease was primarily attributable to net cash used in operating activities of $35.6 million and cash used to purchase property and equipment of $4.6 million.

Revenues were $17.6 million for the 3 months ended March 31, 2020, compared to $900,000 for the same period in 2019. The increase was primarily attributable to $10 million of royalty revenue recognized on net sales of ZOLGENSMA in the first quarter of 2020 (inaudible) $7.2 million of license revenue recognized from new licensing granted to Ultragenyx during the period. Commercial sales of ZOLGENSMA commenced in the second quarter of 2019. The REGENXBIO is eligible to receive a milestone payment of $80 million from AveXis upon the achievement of $1 billion in cumulative net sales of ZOLGENSMA. As of the end of the first quarter of 2020, they have reported over $530 million in net sales, so we are more than halfway to that milestone.

Research and development expenses were $37 million for the 3 months ended March 31, 2020, compared to $25.2 million for the same period in 2019. The increase was primarily attributable to personnel-related costs as a result of increased headcount, laboratory and facility costs, expenses associated with conducting clinical trials for our lead product candidates, and externally sourced services for preclinical, regulatory and manufacturing-related activities.

General and administrative expenses were $14.8 million for the 3 months ended March 31, 2020, compared to $11.6 million for the same period in 2019. The increase was primarily attributable to personnel-related costs as a result of increased headcount, and professional fees for advisory and other services.

Net loss was $40 million or $1.08 basic and diluted net loss per share for the 3 months of March 31, 2020, compared to a net loss of $32.2 million or $0.89 basic and diluted net loss per share for the same period in 2019. As of March 31, 2020, we had approximately 37.2 million common shares outstanding.

Based on our current operating plan, we expect the balance in cash, cash equivalents and marketable securities of $356.6 million to fund the completion of our internal manufacturing capabilities and clinical advancement of our product candidates into 2022. With that, I will turn the call back to Ken to provide final thoughts.

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Kenneth T. Mills, REGENXBIO Inc. - CEO, President & Director [7]

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Thanks for that update, Vit. So next week is the American Society of Gene and Cell Therapy Conference. And we've announced a number of scientific posters and presentations that will be shared. Our research and development team continues to demonstrate a deep and impressive knowledge in AAV discovery, characterization, delivery and significant experience and expertise in process development, production at large scale. So we look forward to participating with our industry partners, friends and colleagues, highlighting some of the work next week as well as continuing to share new data from our ongoing research throughout 2020.

Finally, as I've worked with our team to continue to pursue our mission for patients, balance the challenges and dealing with the COVID-19 pandemic, I've also reflected more on how the potential gene therapy treatments to help ease the burden on the medical community while also protecting patients, families, caregivers, even larger communities from certain risks involved current treatment options during events like what we're experiencing with this pandemic. There are many patient populations that rely on traveling, regular access (inaudible) medical care facilities for injections or infusions that are important, aesthetically necessary, sight-saving or life-saving medicines. Single administration gene therapy treatments can deliver important alternatives in moments where travel and access is restricted. Health care resources are limited, and product supplies may be threatened. Against the background of a pandemic that's taxing the global health care resources at unprecedented levels, it's even more apparent to me that our single-administration treatments have the potential to also create safer and more robust system for care.

The medical community and the biotech financial community should be doing everything possible to support and expedite single administration treatments, especially for large and at-risk populations. We continue to make strong progress in advancing key programs, broadening our internal care pipeline. After more than a decade of steadfast effort and focus, we remain dedicated and committed to improving lives through the curative potential of gene therapy.

With that, we're happy to turn the call over for questions. Operator?

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Questions and Answers

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Operator [1]

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(Operator Instructions) And your first question comes from Gena Wang with Barclays.

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Huidong Wang, Barclays Bank PLC, Research Division - Research Analyst [2]

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First related to resolution on the latest update in data was really impressive, especially after the update from other competitors. So another thing I wanted to ask you is, regarding the suprachoroidal data and the initiation first half this year, and also the clinical data, first cohort data by the end of this year. Just wondering what kind of data should we expect will you be presenting at the end of this year? Regarding the patient numbers, if we understand correctly, it's 6 patients? And what other data will you be able to share with us?

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Stephen Pakola, REGENXBIO Inc. - Senior VP & Chief Medical Officer [3]

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Gena, Steve here. Thanks for the questions. Yes, as you mentioned, our guidance is we continue to target starting our suprachoroidal delivery development program, first with a wet AMD and to kick that study off by the end of the first half of this year. And we still are guiding towards having interim initial data at the end of the year. We haven't actually disclosed how many patients we have in the study and other details, and we look forward to providing that later. But certainly, we stand by our guidance of giving an update based on some of the data that we'll have as of the end of the year. One of the nice things of the wet AMD indication is we already have a good handle on wet AMD in terms of looking at the appropriate types of endpoints, both anatomic, functional and treatment burden endpoints that we've talked about in the past and very recently with our recent data update in our subretinal delivery program. So we have a good handle on those types of endpoints. So I think you can envision similar types of data readouts.

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Operator [4]

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Your next question comes from Gbola Amusa with Chardan.

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Gbolahan Amusa, Chardan Capital Markets, LLC, Research Division - Director of Research & Head of Healthcare Research [5]

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Congrats as well on the 2-year durability data for 314. I had a couple of questions on the potential for inflammation for your in-office suprachoroidal approach. Obviously, there's a lot of benefit in going in the office, but some would argue there's greater potential for inflammation with that approach. So 1 -- 2 questions is, do you agree with that assessment? And then second, given that we've seen very recent and very early results with intravitreal gene therapy using expanded courses of prophylactic steroids addressing issues, maybe addressing thoroughly issues of inflammation with the (inaudible) capsid and wet AMD and also RP. Do those results go early? Do they motivate you to use an extended course of prophylactic steroids for your SCS microinjector programs? So those 2 questions then very quickly. Novartis just said that there's a multibillion-dollar potential for ZOLGENSMA, again, and this has updated guidance, and you got obviously up to 10% of that. What -- could you cover what they said [invites public forms] on the timing of the ramp towards that guidance?

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Kenneth T. Mills, REGENXBIO Inc. - CEO, President & Director [6]

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Steve, maybe I'll let you start with the 314 questions.

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Stephen Pakola, REGENXBIO Inc. - Senior VP & Chief Medical Officer [7]

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Okay. That makes sense. Gbola, that's a great question. Suprachoroidal delivery is a different route of administration than subretinal. Subretinal is the gold standard. That's where we have the most efficacy and safety and clear demonstration that there isn't inflammation associated with subretinal gene therapy. And that's obviously very excited about for our lead program with subretinal delivery. Suprachoroidal, the in-office potential delivery there, where we still are anatomically delivering very close to the target issue of the RPE and the photoreceptors, but you raise a legitimate question. What do we know about immune privilege or lack thereof and the potential for inflammation? Historically, there's been inflammation seen, an immune response with suprachoroidal delivery of earlier generation AAV vectors such as AAV2 and AAV5. One of the things that got us very excited about suprachoroidal delivery with our proprietary NAV technology is actual preclinical data in both small and large animal models, where we've seen no inflammation with suprachoroidal delivery of AAV8 vector including RGX-314 in multiple studies. So that gives us (inaudible) that perhaps there is less of a risk of inflammation with [suprachoroidal] delivery than exists with intravitreal, for example.

So with intravitreal, we know historically -- universally really, with preclinical and clinical experiments, that at the doses that you have to give with either AAV2 or other vectors, that you have to give a high enough dose to have fusion to get to the back of the eye, the target tissue and through the internal limiting membrane barrier, that invariably at doses where you get good transduction, you also see immune-mediated inflammation. And we continue to see that, frankly, validated in any preclinical experiments or clinical data that's come out with intravitreal administration.

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Kenneth T. Mills, REGENXBIO Inc. - CEO, President & Director [8]

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Gbola, just turning to the question about ZOLGENSMA. I think what we've seen most recently between Novartis' update at their last earnings call, and then they had an update around new data that was presented at MDA, is that for the IV route of administration, that's, of course, currently approved and being marketed, facilitating the sales numbers that we're seeing based on the U.S. approval, there's been some regulatory events in Japan and Europe. Specifically, they pointed to that indicate that reimbursement is coming in other jurisdictions as early as the end of the first half of this year. In addition, with respect to the new intrathecal data that we mentioned earlier in the call, that there was an update on the strong study showing improved outcomes as described, and there's been guidance from Novartis that a BLA could be filed or equivalents, I guess, worldwide, as early as the second half of 2020 or into 2021. So we continue to be really encouraged by the data, by what we're seeing in terms of the uptake of the use of gene therapy. Again, I think we're looking at what must mean among, if not the, most successful launch for gene therapy to date and are liking to see the regulatory and commercial milestones that are emerging.

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Operator [9]

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Your next question comes from Mani Foroohar with SVB Leerink.

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Mani Foroohar, SVB Leerink LLC, Research Division - MD of Genetic Medicines & Senior Research Analyst [10]

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Edited Transcript of RGNX earnings conference call or presentation 7-May-20 8:30pm GMT - Yahoo Finance

ReportsnReports always aims at offering their clients an in-depth analysis and the best research material of the various market. This report on the global Regenerative Medicine Market is committed fulfilling the requirements of the clients by giving them thorough insights into the market. An exclusive data offered in this report is collected by research and industry experts team. Regenerative Medicine Market market spread across 137 Pages, profiling 10 companies and supported with tables and figures.

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#Top Key Players in the Regenerative Medicine Market include are Organogenesis Inc. (U.S.), Osiris Therapeutics, Inc. (U.S.), Vericel Corporation (U.S.), Stryker Corporation (U.S.), and NuVasive, Inc. (U.S.). The key players in the acellular products segment are Medtronic (Ireland), Acelity (KCI Concepts) (U.S.), Integra Life Sciences (U.S.), Cook Biotech Inc. (U.S.), and C.R. Bard (U.S.).

On the basis of applications, the segments are orthopedic & musculoskeletal disorders, dermatology, cardiology, diabetes, central nervous system diseases, and other applications. Oncology is the fastest growing application market. Increasing cancer incidence, rich product pipeline and increasing demand for cancer treatments fuelling the growth of the market.

Geographically, the Regenerative medicine market is dominated by North America, followed by Europe, Asia-Pacific, and the Rest of the World (RoW). Growth in the North American segment is primarily driven by rapidly increasing aging population and increase in chronic diseases are the major drivers for developed economies like the U.S.

#Target Audience for Regenerative Medicine Market: Healthcare providers & clinical experts,Service providers,Therapeutic companies,Contract manufacturers,Academic research institutes.

A Discount (20% or More) can be asked before order a copy of Regenerative Medicine Market report at https://www.reportsnreports.com/contacts/discount.aspx?name=858290

List of Tables:

Table 1 Few Important Clinical Studies in the Regenerative Medicine: Market

Table 2 Cancer Incidence and Mortality, By Region, 20122035

Table 3 Funding for Regenerative Medicine Research By the Nih Under the 21st Century Cures Act

Table 4 Nih Funding for Regenerative Medicine Research in the Us, 20142016 (USD Million)

Table 5 California Institute of Regenerative Medicine Funding for Regenerative Medicine Research

Table 6 Number of Companies Operating in the Regenerative Medicine: Market, By Region (20142018)

Table 7 Market, By Type, 20172024 (USD Billion)

Table 8 Few of the Commercialized Cell-Based Immunotherapy & Cell Therapy Products

Table 9 Cell-Based Immunotherapy & Cell Therapy Products Market, By Region, 20172024 (USD Billion)

Table 10 Cell-Based Immunotherapy & Cell Therapy Products Market, By Type, 20172024 (USD Billion)

Table 11 Cell-Based Allogeneic Regenerative Medicine

Table 12 Allogeneic Products Market, By Region, 20172024 (USD Billion)

Table 13 Cell-Based Autologous Regenerative Medicine

Table 14 Autologous Products Market, By Region, 20172024 (USD Billion)

Table 15 Few of the Commercialized Tissue-Engineered Products

.and more

The rest is here:
Regenerative Medicine Market by Type [Cell-Based Immunotherapy & Cell Therapy (Allogeneic & Autologous Products), Tissue Engineering, Gene...

As the number of thalassemia patients is increasing in India, need for a prevention and control program is felt. With preventive health checks not being the norm in India, people suffering from thalassemia are unknowingly passing on this genetic disorder to their children. The need of the hour is to have a national policy on thalassemia which will help in not just creating awareness about the disease but also ensure strategies to prevent its spread, diagnosis, and treatment for all. Even though we have the highest number of thalassemia patients in South Asia, the promising treatment modalities like bone marrow transplant (BMT) and gene therapy have their own share of risks and shortcomings due to a lack of incentives and support for the research studies.

To save valuable lives, setting up of small or micro blood donation camps or mobile vans can be done following the social distancing norms in the areas of green zones across the country with the help of various welfare organizations. Making appeals through social media platforms to healthy people to come out in numbers and donate blood is one way to create an informed society.

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How COVID-19 is endangering the lives of Thalassemia patients - National Herald

Lately, there has been remarkable shift in the therapeutics method. People are inclining towards personalized medication rather than pharmaceutical treatment methods. This is result of advancements in biological therapies. As a result, advanced therapy medicinal products market (ATMP) is emerging. Such products provide solution for conditions with no therapeutic alternatives. This is a key factor driving growth of the advanced therapy medicinal products market across the globe.

At present, regulations for ATMPs is at its budding stage. Extensive research activities has been going on, which is resulting in Investigational New Drug (IND) applications.

The upcoming advanced therapy medicinal products market analysis report provides insight about the upcoming trends and restraining factors likely to shape growth of the market during forecast period (2019-2029). The report also provides a comprehensive analysis of the key companies of the market and offers details about the capacities and competencies of these companies. The market report also focusses on the markets competitive landscape and provides detail of the product portfolio of various companies.

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Advanced Therapy Medicinal Products Market: Competitive Analysis

At present, the advanced therapy medicinal products market is growing at a lucrative rate. This growth rate is attributed to recent approval of various advanced therapy medicinal products. Post success of approved products, stakeholders are investing at enormously in clinical trials of advanced therapy medicinal products.

On the other hand, companies operating in the market are adopting various strategies to accelerate the product manufacturing rate. While most of the companies are relying on in-house production of therapies, few players such as Contract Manufacturing Organizations (CMOs) are opting for third-party service providers.

Upsurge in demand for gene therapy has resulted in widening of drug development landscape and rise in the number of new entrants. However, there is lack of production capabilities.

Also, several companies are strengthening their foothold in the global market by strategic alliances and acquiring small CAR T-cell therapy developers.

Some of the key players operating in the advanced therapy medicinal products market are-

Advanced Therapy Medicinal Products Market: Key Trends

Despite high cost of the products, the market is expanding at lucrative rate. The growth rate is attributed to various health benefits provided by new classes of therapies.

Registering clinical benefits and efficiency of the products, stakeholders in the market are developing new strategies to overcome the challenged and boost application of advanced therapy medicinal products

Advanced Therapy Medicinal Products Market: Regional Outlook

Availability of significant number of FDA approved advanced therapy medicinal products in the U.S. has accounted for the prominent share of North America region, in terms of revenue. Recently, approval of products such as Yescarta, Zolgensma, and Kymriah has led to prominent investment in the U.S. advanced therapy medicinal products market.

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Europe hold second-largest pharmaceutical market space across the globe. In coming years, cell therapy developers are anticipated to hold prominent share in the Europes drug revenue. Moreover, several academic institutes in Europe are conducting extensive research in early-stage cell therapy. This factor is likely to fuel the regional revenue contribution.

Meanwhile, global manufacturing companies operating in the market are enhancing their reach across Europe. This, in turn, may drive growth in the regional market.

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Advanced Therapy Medicinal Products Market: Key Challenges and Winning Imperatives - BioSpace

BURLINGTON, Mass., May 07, 2020 (GLOBE NEWSWIRE) -- Flexion Therapeutics, Inc. (Nasdaq:FLXN) today reported financial results and recent business highlights for the quarter ended March 31, 2020.

We were very pleased with ZILRETTAs trajectory earlier in the first quarter of 2020; however, in March we began to experience impacts from the COVID-19 pandemic, said Michael Clayman, M.D., President and Chief Executive Officer of Flexion Therapeutics. The pandemic negatively affected our customers ability to treat patients as well as our first-quarter sales, and we believe that COVID-19 will adversely impact revenue for the remainder of the year. That said, our field teams have been utilizing various technologies to engage in conversations with prescribing physicians, and our confidence in ZILRETTAs long-term potential is undiminished. In fact, given the cancellations and postponements of total knee replacement surgeries, we believe ZILRETTA has the potential to play an increasingly prominent role in a post-pandemic environment. However, since that time horizon is uncertain, we have taken meaningful actions to reduce our operating expenses.

Dr. Clayman added, We recently made the strategic decision to discontinue our Phase 2 trial investigating ZILRETTA in shoulder OA and adhesive capsulitis. Given the small number of patients enrolled in that trial, the uncertainty around when we will be able to restart it, as well as the costs required to maintain the study in an inactive status, we believe terminating the trial is the most responsible action at this time. Moving forward, we will look to leverage our learnings from the study and potentially incorporate them into a new trial design to advance ZILRETTA in these indications. We continue to plan to reinitiate the FX201 single ascending dose trial as soon as feasible. Ultimately, we are committed to ensuring Flexion emerges from the crisis in a position of strength and that we are ready to quickly reaccelerate our commercial and R&D activities.

First-Quarter Results & Financial HighlightsThe Company reported a net loss of $36.8 million for the first quarter of 2020, compared to a net loss of $41.5 million for the same period of 2019. Net sales of ZILRETTA were $20.1 million and $10.6 million for the three months ended March 31, 2020 and 2019, respectively. Cost of sales was $2.3 million and $1.8 million for the three months ended March 31, 2020 and 2019, respectively.

Research and development expenses were $21.1 million and $15.4 million for the three months ended March 31, 2020 and 2019, respectively. The increase in research and development expenses of $5.7 million was primarily due to an increase of $2.2 million in salary and other employee-related costs for additional headcount and stock compensation expense, an increase in expenses related to FX201, including the payment of $2.5 million to GeneQuine for dosing the first human patient in the Phase I clinical trial, and an increase in other portfolio expenses, primarily related to the $0.5 million milestone to Xenon Pharmaceuticals, offset by a decrease of $0.5 million in development expenses for ZILRETTA due to lower clinical trial expenses during the period.

Selling, general and administrative expenses were $29.3 million and $32.2 million for the three months ended March 31, 2020 and 2019, respectively. Selling expenses were $20.5 million and $23.8 million for the three months ended March 31, 2020 and 2019, respectively. The year-over-year decrease in selling expenses of $3.3 million was primarily due to a reduction in physician and patient marketing activities. General and administrative expenses were $8.8 million and $8.4 million for the three months ended March 31, 2020 and 2019, respectively, which represents an increase of $0.4 million.

Interest income was $0.4 million and $1.0 million for the three months ended March 31, 2020 and 2019, respectively. Interest expense was $4.7 and $3.9 million for the three months ended March 31, 2020 and 2019, respectively.

As of March 31, 2020, the Company had approximately $125.2 million in cash, cash equivalents and marketable securities compared with $136.7 million as of December 31, 2019.

ZILRETTA Commercial MetricsSince the launch of ZILRETTA in November 2017 through March 31, 2020:

Reductions in Operating ExpensesIn response to the economic and business disruption caused by COVID-19, Flexion undertook prudent and disciplined steps to reduce expenses across the organization. The Company believes these steps will enhance financial flexibility and liquidity and estimates they will deliver between $43 million and $53 million in savings this year.As a result, Flexion expects full-year 2020 operating expenses (including cost of sales, research and development, and selling, general and administrative) will be in the range of $167 million to $177 million.The Company expects to achieve these cost savings through:

ZILRETTA Supply ChainThe Company remains confident in its ability to maintain adequate commercial supply of ZILRETTA and expects its current finished goods inventory will be sufficient to meet demand for at least the remainder of 2020. To avoid excess levels of inventory, Flexion is temporarily suspending manufacturing activities for ZILRETTA. Since Flexion employs a condominium model at Patheons manufacturing site, the Company has the ability to reinitiate manufacturing following three months notice to Patheon once additional supply is needed.

Recent News & Business Updates

Conference CallFlexions management will host a conference call today at 4:30 p.m. ET. A live webcast of the conference call can be accessed through the Investors tab on the Flexion Therapeutics website, and a replay will be available online after the call. For those planning to ask a question, the dial-in number for the conference call is 855-770-0022 for domestic participants and 908-982-4677 for international participants, with Conference ID # 4498458. Please dial in at least 15 minutes in advance to ensure a timely connection to the call.

Indication and Select Important Safety Information for ZILRETTA

Indication:ZILRETTA is indicated as an intra-articular injection for the management of osteoarthritis pain of the knee.

Limitation of Use: The efficacy and safety of repeat administration of ZILRETTA have not been demonstrated.

Contraindication:ZILRETTA is contraindicated in patients who are hypersensitive to triamcinolone acetonide, corticosteroids or any components of the product.

Warnings and Precautions:

Adverse Reactions:The most commonly reported adverse reactions (incidence 1%) in clinical studies included sinusitis, cough, and contusions.

Please seeZilrettaLabel.comfor full Prescribing Information.

About ZILRETTAOn October 6, 2017, ZILRETTA was approved by the U.S. FDA as the first and only extended-release intra-articular therapy for patients confronting osteoarthritis-related knee pain. ZILRETTA employs proprietary microsphere technology combining triamcinolone acetonide a commonly administered, short-acting corticosteroid with a poly lactic-co-glycolic acid (PLGA) matrix to provide extended pain relief. The pivotal Phase 3 trial on which the approval of ZILRETTA was based showed that ZILRETTA significantly reduced knee pain for 12 weeks, with some people experiencing pain relief through Week 16. Learn more at http://www.zilretta.com.

About Osteoarthritis (OA) of the KneeOA, also known as degenerative joint disease, affects more than 30 million Americans and accounts for more than $185 billion in annual expenditures. In 2018, more than 15 million Americans were diagnosed with OA of the knee and the average age of physician-diagnosed knee OA has fallen by 16 years, from 72 in the 1990s to 56 in the 2010s. The prevalence of OA is expected to continue to increase as a result of aging, obesity and sports injuries. Each year, approximately five million OA patients receive either a corticosteroid (immediate-release or extended-release) or hyaluronic acid intra-articular injection to manage their knee pain.

About FX201FX201 is a locally administered gene therapy product candidate which utilizes a helper-dependent adenovirus (HDAd) vector, designed to stimulate the production of an anti-inflammatory protein, interleukin-1 receptor antagonist (IL-1Ra), whenever inflammation is present within the joint. Inflammation is a known cause of pain, and chronic inflammation is thought to play a major role in the progression of osteoarthritis (OA). By persistently suppressing inflammation, Flexion believes FX201 holds the potential to both reduce OA pain and modify disease progression.

About FX301FX301 is a locally administered NaV1.7 inhibitor product candidate, known as funapide formulated for extended release in a thermosensitive hydrogel. The initial development of FX301 is intended to support administration as a peripheral nerve block for control of post-operative pain. Flexion believes FX301 has the potential to provide effective and durable pain relief while preserving motor function and anticipates initiating clinical trials in 2021.

AboutFlexion TherapeuticsFlexion Therapeutics(Nasdaq:FLXN) is a biopharmaceutical company focused on the development and commercialization of novel, local therapies for the treatment of people with musculoskeletal conditions, beginning with osteoarthritis, the most common form of arthritis. The Company's core values are focus, ingenuity, tenacity, transparency and fun. Visitflexiontherapeutics.com.

Forward-Looking StatementsThis release contains forward-looking statements that are based on the current expectations and beliefs of Flexion. Statements in this press release regarding matters that are not historical facts, including, but not limited to, statements relating to the future of Flexion; our expectations regarding expenses for the year endedDecember 31, 2020; expected savings from actions to reduce operating expenses; expected impacts from COVID-19 and the timing and duration of such impacts; Flexions ability to maintain commercial supply of ZILRETTA; long-term potential of ZILRETTA, including following the COVID-19 pandemic; expected clinical developments and clinical trial timelines; expected increases in the rate of individuals with OA of the knee; and the potential therapeutic and other benefits of ZILRETTA and Flexions product pipeline, are forward looking statements. These forward-looking statements are based on management's expectations and assumptions as of the date of this press release and are subject to numerous risks and uncertainties, which could cause actual results to differ materially from those expressed or implied by such statements. These risks and uncertainties include, without limitation, risk that we may not achieve expense expectations for 2020; the fact that the impacts and expected duration of the COVID-19 pandemic are uncertain and rapidly changing; the risk that we may not be able to successfully maintain an effective sales force to commercialize ZILRETTA; competition from alternative therapies; the risk that we may not be able to maintain and enforce our intellectual property, including intellectual property related to ZILRETTA; the risk that ZILRETTA may not be successfully commercialized or adopted; risks regarding our ability to obtain adequate reimbursement from payers for ZILRETTA; risks related to the manufacture and distribution of ZILRETTA, including our reliance on sole sources of supply and distribution; risks related to clinical trials, including potential delays, safety issues or negative results; risks related to key employees, markets, economic conditions, health care reform, prices and reimbursement rates; and other risks and uncertainties described in our filings with theSecurities and Exchange Commission(SEC), including under the heading "Risk Factors" in our Annual Report on Form 10-K for the year endedDecember 31, 2019filed with theSEConMarch 12, 2020and subsequent filings with theSEC. The forward-looking statements in this press release speak only as of the date of this press release, and we undertake no obligation to update or revise any of the statements. We caution investors not to place considerable reliance on the forward-looking statements contained in this press release.

Contact:

Scott YoungVice President, Corporate Communications & Investor RelationsT: 781-305-7194syoung@flexiontherapeutics.com

Julie DownsAssociate Director, Corporate Communications & Investor RelationsT: 781-305-7137jdowns@flexiontherapeutics.com

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Flexion Therapeutics Reports First-Quarter 2020 Financial Results and Recent Business Highlights - GlobeNewswire

Oncogenuity, Inc., a Fortress partner company, enters into an agreement with Columbia University to develop a broad platform technology using oligonucleotides

Initial target is KRAS-driven cancers, often considered un-druggable

Platform being explored as a treatment for coronaviruses, including COVID-19

NEW YORK, May 08, 2020 (GLOBE NEWSWIRE) -- Fortress Biotech, Inc. (Nasdaq: FBIO) (Fortress), an innovative biopharmaceutical company, today announced that Oncogenuity, Inc. (Oncogenuity), a new Fortress partner company, has entered into an exclusive worldwide licensing agreement with Columbia University to develop novel oligonucleotides for the treatment of genetically driven cancers. The proprietary platform produces oligomers, now known as ONCOlogues, that are capable of binding gene sequences 1,000 times more effectively than complementary native DNA. The technology comes from the labs of Gary Schwartz, M.D., Division Chief, Hematology/Oncology, and Jeffrey Rothman, M.D., Ph.D., Assistant Professor of Medicine.

ONCOlogues are sensitive to a single base pair mismatch, resistant to degradation and use a proprietary delivery sequence to enter cells. ONCOlogues selectivity enables Oncogenuity to target genetically driven cancers caused by mutations without impacting wild-type (WT) DNA sequences, potentially limiting off-target toxicity. In addition, this allows ONCOlogues to target mutations that have historically been considered un-druggable.

Oncogenuity has established proof-of-concept in a pre-clinical setting for various cancer types. The companys most advanced program is targeting the KRAS mutation G12D, which was previously considered un-druggable and plays a significant role in various cancer types with substantial unmet need, including pancreatic and colorectal. Given the platforms ability to target any mutation, Oncogenuity will continue to evaluate other mutations simultaneously. The company anticipates additional data publications in the coming 12 months.

Additionally, Oncogenuity is exploring the platforms potential to treat coronaviruses. Coronaviruses have single-stranded RNA genomes, making them strong targets for ONCOlogues. The company is studying replacement sequences, which could help combat COVID-19 and provide proof-of-concept as a treatment for coronaviruses. These ongoing experiments would validate ONCOlogues as a possible treatment for COVID-19, as well as potentially expedite the discovery of treatments for future coronavirus outbreaks.

Lindsay A. Rosenwald, M.D., Fortress Chairman, President and Chief Executive Officer, said, We are excited to work with the excellent scientists and physicians at Columbia University again. Our last joint effort with Columbia University led to the formation of our partner company, Caelum Biosciences, Inc. (Caelum). Since formation, Caelum has raised approximately $60 million in development funding from a number of sources, with additional amounts available upon the satisfaction of certain milestones and will be initiating two registration clinical trials in the next several weeks. Building upon our success with Caelum, we are grateful to Columbia University for entrusting us to develop this highly innovative technology using oligonucleotides to target genetically driven cancers and coronaviruses. Using a targeted genetic approach to treat cancer has become essential to limiting toxicity and treating patients effectively. This technology has the potential to target mutations that have previously been considered un-druggable. Oncogenuity will aggressively pursue the development of ONCOlogues to ultimately provide patients with new, safe and effective treatment options.

Scientific Co-Founder Jeffrey Rothman, M.D., Ph.D., said, Through rigorous statistical, mechanical and molecular modeling, combined with gene sequence data, we are able to create sequence-specific, targeted therapeutics against oncogenes, which are the cause of and specific to tumor cells. Until now, achieving this goal had been considered nearly impossible. However, with these novel design features, we now have the ability to target cancer while potentially avoiding side effects, which are the main cause of dose-limitation, by design. There is much potential because we are able to target multiple genes and therefore, multiple cancers. Moreover, due to their single-strand format, application toward viral targets such as in COVID are even more facile given their easier accessibility. We are excited and determined to pursue this endeavor with Fortress Biotech and very much welcome their continued support.

About Oncogenuity, Inc.Oncogenuity, Inc. is a biopharmaceutical company focused on the development and commercialization of ONCOlogues for the treatment of genetically driven cancers and coronaviruses. Oncogenuitys lead asset targets a KRAS mutation, G12D. Oncogenuity is located in New York City and was founded by Fortress Biotech, Inc. (Nasdaq: FBIO).

About Fortress Biotech Fortress Biotech, Inc. (Fortress) is an innovative biopharmaceutical company that was recently ranked number 10 in Deloittes 2019 Technology Fast 500, an annual ranking of the fastest-growing North American companies in the technology, media, telecommunications, life sciences and energy tech sectors, based on percentage of fiscal year revenue growth over a three-year period. Fortress is focused on acquiring, developing and commercializing high-potential marketed pharmaceutical products and development-stage pharmaceutical product candidates. The company has five marketed prescription pharmaceutical products and over 25 programs in development at Fortress, at its majority-owned and majority-controlled partners and at partners it founded and in which it holds significant minority ownership positions. Such product candidates span six large-market areas, including oncology, rare diseases and gene therapy, which allow it to create value while mitigating risk for shareholders. Fortress advances its diversified pipeline through a streamlined operating structure that fosters efficient drug development. The Fortress model is driven by a world-class business development team that is focused on leveraging its significant biopharmaceutical industry expertise to further expand the companys portfolio of product opportunities. Fortress has established partnerships with some of the worlds leading academic research institutions and biopharmaceutical companies to maximize each opportunity to its full potential, including Alexion Pharmaceuticals, Inc., AstraZeneca, City of Hope, Fred Hutchinson Cancer Research Center, InvaGen Pharmaceuticals Inc. (a subsidiary of Cipla Limited), St. Jude Childrens Research Hospital and Nationwide Childrens Hospital. For more information, visit http://www.fortressbiotech.com.

Forward-Looking StatementsThis press release may contain forward-looking statements within the meaning of Section 27A of the Securities Act of 1933 and Section 21E of the Securities Exchange Act of 1934, as amended. As used below and throughout this press release, the words we, us and our may refer to Fortress individually or together with one or more partner companies, as dictated by context. Such statements include, but are not limited to, any statements relating to our growth strategy and product development programs and any other statements that are not historical facts. Forward-looking statements are based on managements current expectations and are subject to risks and uncertainties that could negatively affect our business, operating results, financial condition and stock price. Factors that could cause actual results to differ materially from those currently anticipated include: risks relating to our growth strategy; our ability to obtain, perform under and maintain financing and strategic agreements and relationships; risks relating to the results of research and development activities; uncertainties relating to preclinical and clinical testing; risks relating to the timing of starting and completing clinical trials; our dependence on third-party suppliers; risks relating to the COVID-19 outbreak and its potential impact on our employees and consultants ability to complete work in a timely manner and on our ability to obtain additional financing on favorable terms or at all; our ability to attract, integrate and retain key personnel; the early stage of products under development; our need for substantial additional funds; government regulation; patent and intellectual property matters; competition; as well as other risks described in our SEC filings. We expressly disclaim any obligation or undertaking to release publicly any updates or revisions to any forward-looking statements contained herein to reflect any change in our expectations or any changes in events, conditions or circumstances on which any such statement is based, except as may be required by law. The information contained herein is intended to be reviewed in its totality, and any stipulations, conditions or provisos that apply to a given piece of information in one part of this press release should be read as applying mutatis mutandis to every other instance of such information appearing herein.

Company Contacts:Jaclyn Jaffe and William BegienFortress Biotech, Inc.(781) 652-4500ir@fortressbiotech.com

Investor Relations Contact:Daniel FerryLifeSci Advisors, LLC(617) 430-7576daniel@lifesciadvisors.com

Media Relations Contact:Tony Plohoros6 Degrees(908) 591-2839tplohoros@6degreespr.com

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Fortress Biotech Announces Exclusive Worldwide License Agreement With Columbia University to Develop Novel Oligonucleotide Platform for the Treatment...

Orchard Therapeutics plc (NASDAQ:ORTX) Q1 2020 Earnings Conference Call May 7, 2020 8:00 AM ET

Company Participants

Renee Leck Director-Investor Relations

Bobby Gaspar Chief Executive Officer

Frank Thomas Chief Operating Officer

Conference Call Participants

Whitney Ijem Guggenheim

Esther Rajavelu Oppenheimer

Yaron Werber Cowen

Peter Kim Barclays

Operator

Ladies and gentlemen, thank you for standing by, and welcome to the Orchard Therapeutics First Quarter 2020 Investor Conference Call. [Operator Instructions] I would now like to hand off the conference over to your speaker today, Renee Leck, Director of Investor Relations. Please go ahead, maam.

Renee Leck

Thanks, Sonia. Good morning, everyone, and welcome to Orchards first quarter 2020 investor call. You can access the slides for todays call by going to the Investors section of our website, orchardtx.com.

Before we get started, Id like to remind everyone that statements we make on this call will include forward-looking statements. Actual events and results could differ materially from those expressed or implied by any forward-looking statements as a result of various risk factors and uncertainties, and including those set forth in our annual 10-K filed with the SEC and any other filings we may make. In addition, any forward-looking statements made on this call represent our views only as of today and should not be relied upon as representing our views as of any subsequent date. We specifically disclaim any obligation to update or revise any forward-looking statements.

And with that, Ill turn the call over to our CEO, Bobby Gaspar.

Bobby Gaspar

Thanks, Renee. Hello, everyone, and welcome. Id like to start by first acknowledging the tremendous efforts of our organization and our partners in the health care field to ensure patients in need continue to receive care during this difficult time. Thank you, everyone.

The last few weeks have been an important period for Orchard. Since taking on the leadership, Frank and I, together with the executive team, have thought very carefully about what the new Orchard can become, how we can ensure that Orchard can fulfill its true potential and what we need to do to make that happen. When we think about our strategic vision as a company, its really all based on the potential of the hematopoietic stem cell gene therapy platform, where it can take us and the benefit it can provide for many patient populations even beyond our current portfolio of ultra-rare diseases. We have taken some bold and decisive actions that we believe will allow Orchard to achieve long-term growth and focus the company on sustainable value creation. This vision is supported by a new strategic plan that we have developed and which is built around four pillars. Each of these forms a chapter in our remarks this morning.

First, operating efficiencies. We have made a series of important changes to our operations that will enable us to sharpen our focus and more efficiently execute our strategy, which I will detail in a moment. Second is our commercial build. We are focused on establishing the right model for the diagnosis and treatment of patients undergoing HSC gene therapy, and see the true value of this approach over a series of ultra-rare products.

Third, one of the most exciting areas in gene therapy right now is the innovation taking place in manufacturing technologies that have the potential to deliver economies of scale. We want to be leaders and invest in this space, knowing that our near-term capacity needs are covered by our experienced CDMO network.

Finally, central to this strategy is prioritizing our portfolio to enable the expansion of Orchards pipeline beyond ultra-rare to less-rare indications. We are disclosing two new research programs for the first time today, and these are a genetic subset of frontotemporal dementia or FTD, and a genetic subset of Crohns disease. We believe that the biological and clinical validation that has already been shown in our ultra-rare indications allow us to expand with confidence to these larger indications.

Turning to the first chapter in our new strategic plan. We are focused on improving the operational efficiency throughout the organization. This started with an extensive evaluation over the past six weeks of each program in our portfolio using several criteria that are shown here on the left-hand side of Slide 5. We undertook an objective analysis that involved both financial metrics and strategic considerations in identifying those programs where there was high need for patients and high-value creation for shareholders. As you can imagine, these were difficult decisions given the potentially transformative nature of many of these programs. Each has value, and we intend to realize that in different ways and over different time horizons.

Today, however, we believe our resources are best focused on Metachromatic Leukodystrophy, Wiskott-Aldrich syndrome, the MPS programs and our research programs. This also means that we have a balanced portfolio with late, mid and early stage programs. The programs I havent mentioned such as OTL-101 and ADA-SCID and the transfusion-dependent, beta-thalassemia program, OTL-300, will have a reduced investment moving forward. We will look for alternative ways to realize value with those programs, including through partnerships.

So Slide 6 brings together a summary of the operational changes that weve announced today. We believe these changes were important and necessary to enable Orchard to execute its mission and objectives at the highest level by matching our attention and resources to a set of core imperatives for the business. As summarized here, we expect to realize cash savings of approximately $15 million from the prioritization of our portfolio. Another $60 million in savings results from the decision to consolidate our R&D teams to one site and defer the investment in the manufacturing facility.

Finally, the more staged approach to the commercial build-out and 25% reduction to our existing workforce and future headcount planning will each yield another $25 million in savings. All of these cash savings are expected to be realized over 2020 and 2021, and result in total expected savings of $125 million over that period. With the revised plan, we now have cash runway into 2022 and no near term need to finance.

Its worth briefly mentioning that this $125 million savings is after making investments in the following key areas to support our new strategy, shown on Slide 7. In commercial, diagnostic and screening initiatives, including no-charge testing programs to help identify patients with MLD and other neurodegenerative conditions in time for treatment. In manufacturing, the technology, process innovations and efficiencies to drive scalability.

In R&D, initiatives in less-rare diseases that have the potential to fuel the companys future growth in a substantial way. This wasnt just an exercise to reduce expenses, but important decision-making to ensure our capital is deployed in a disciplined manner, while building a pipeline that can leverage our success across all phases of our business.

Now let me turn the call over to Frank to discuss additional key elements of the new plan.

Frank Thomas

Thanks, Bobby, and good morning, everyone. As you can tell from this mornings press release, we have carefully examined each aspect of our business. You heard that a moment ago from Bobby, with the way we are creating operational efficiencies, and I think you will see additional evidence in the next two sections as we summarize our latest thinking around commercial deployment and manufacturing.

Starting with commercial. We understand the importance of developing a commercial model that will demonstrate our ability to execute and bring these therapies to the market successfully. This model and the infrastructure that we build will also be leveraged for any future product launches.

As youll note from the bottom of Slide 9, each rare disease has certain dynamics that will impact the launch trajectory and speed with which we can penetrate the market. In fact, we anticipate our first two potential launches in WAS and MLD having distinct but complementary launch curves, as you can see from the illustrative diagrams.

Let me start with MLD on the left, where we expect to launch first in the EU, followed by the U.S. and then other countries around the world. We think an important inflection point on the revenue curve with MLD will come later when newborn screening is established, providing an opportunity for an acceleration in growth rate. Disease progression is a second important dynamic that will affect market penetration. Because MLD advances so rapidly, it will be important to diagnose patients early and get them treated.

For Wiskott-Aldrich syndrome, the dynamics are very different, and its reflected in the shape of the curve on the right. Unlike MLD, this disease is slower progressing and more readily diagnosed. We believe that WAS will provide an opportunity to treat a number of prevalent patients from the outset and also give us additional long-term revenue stream. This program, the BLA and MAA filings are on track for 2021.

Turning back to MLD for an update on the regulatory time line. We are on track to get a decision from the European Medicines Agency later this year, and if approved, launch in the EU in the first half of 2021. In the U.S., we recently engaged with the FDA on our planned BLA submission of OTL-200 for the treatment of MLD. The FDA has provided written feedback on the sufficiency of the companys data package, including the clinical endpoint, the natural history comparator and the CMC data package.

As a result of this feedback, we intend to file an IND later this year and also seek RMAT designation, both of which we believe will facilitate a more comprehensive dialogue to discuss the data more fully and resolve the open matters before submitting a BLA. We are committed to working closely with the agency, and well provide updated guidance on the new filing time lines for the BLA after further regulatory interaction.

On Slide 10, you can see that were tracking nicely for the launch of OTL-200 in the EU in the first half of 2021, if approved, with Germany being the first country where we expect to treat commercial patients. Many of the prelaunch activities are underway, and the team has been able to keep up momentum during the pandemic to work with key centers and progress with site qualifications. We intend to set up a network of treatment centers where MLD patients are often referred and who also have transplant expertise. These same centers can be leveraged in future launches, especially for programs in the neurometabolic franchise.

I previously mentioned the importance of diagnosis in MLD to identify patients at early stages of disease, and we are taking the necessary steps to achieve long-term success. Beyond typical disease awareness efforts, we are also looking at initiatives such as no-charge diagnostic testing with partners such as Invitae, and we are looking to facilitate newborn screening for MLD with funding of upcoming pilots in New York State and Italy that are designed to validate the assay and provide the data for wider implementation. Success in these key initiatives will support early MLD patient identification.

Coming up quickly behind MLD and the neurometabolic franchise, our two proof-of-concept programs in MPS disorders, where we have made recent progress even during this challenging period with COVID-19. For MPS-I, over the past year, weve shown promising preliminary proof-of-concept data with positive engraftment, biomarker correction and encouraging early clinical outcomes, and we are excited to announce our plan to begin a registrational trial next year, bringing this program one step closer to commercialization.

For MPS-IIIA, we announced late last month that the first patient was treated in a proof-of-concept trial at Royal Manchester Childrens Hospital, with enrollment planned to continue this year and interim data to be released in 2021.

You can see graphically on Slide 12 how the aggregation of these commercial markets lead to sustainable revenue growth. In addition, the infrastructure build is designed to provide the necessary commercial capabilities to realize the potential of the portfolio. On this slide, weve included the incidence figures for MLD and the incidence and prevalence figures for WAS to help you understand each opportunity as we see it today.

Given the dynamics at play for MLD that I described on Slide 9, we believe this opportunity should largely be tied to the incident patient population, which we believe ranges from 200 to 600 patients per year in countries where rare diseases are often reimbursed. Weve taken a more conservative view than previously on the addressable patient and market opportunity in countries such as those in the Middle East and Turkey, where the literature has a wide range of differing incident figures. Also, over time, with improved disease awareness, there may be prevalent patients identified who also could benefit from therapy. Our commercial strategy has always been and continues to be based not only on 1 product, but rather the aggregation of multiple potential products launching off one HSC gene therapy platform and infrastructure.

Turning to manufacturing. Weve also made some key changes to our approach in manufacturing and how we allocate capital in the short and mid-term. On Slide 14, youll see the main tenets of our new manufacturing strategy. First, in the near term, we plan to focus on innovative technologies to enable commercial scalability.

Second, to ensure the appropriate focus on those technologies, weve made a decision to consolidate R&D to a single site in London, which brings together our organization in a more efficient way. This will allow efforts made to improve our manufacturing processes to be quickly and easily shared and then scaled commercially to transfer to our third party manufacturers, all of whom are currently located in Europe. As part of this consolidation, we will close our California site, including the termination of the Fremont project and associated capital spend.

Third, we have strong relationships with CDMOs that will ensure supply of clinical and commercial product to satisfy near-term requirements. And longer term, we intend to identify a new site in the U.S. to eventually bring manufacturing capabilities in-house with a facility that is appropriately sized and fitted for future techniques and operations.

Slide 15 shows the three phases of our approach in manufacturing: invest, partner and build. Today, we are investing, and well continue to invest in technologies such as transduction enhancers, stable producer cell line and closed automated processing of the drug product. This will potentially reduce the amount of vector needed, drive down COGS and potentially change the way products are manufactured, making it less labor-intensive, less expensive and more consistent. In the near and mid-term, we will continue to rely on our manufacturing partners for the early planned launches in MLD and WAS. For example, MolMed has been with these programs since the beginning, and theyve been a reliable commercial partner with Strimvelis.

In addition to our existing CDMO network, we have begun to search for a drug product partner in the U.S. to complete a tech transfer and serve the U.S. market, thereby reducing scheduling challenges and creating some redundancy. And finally, over time, we plan to build in-house manufacturing capabilities closer to when there is a need for additional capacity. This enables us to explore options that are more aligned with our business in terms of scale and timing.

And with that, Ill turn the call back over to Bobby.

Bobby Gaspar

Thanks, Frank. In this section, Im going to briefly highlight the potential of HSC gene therapy to correct not only blood lineage cells, but also how through natural mechanisms, specific cell types may allow correction of disease in specific organ systems and enable expansion of our portfolio into new research indications.

As many of you know, and as shown on Slide 17, through HSC gene therapy, we are able to insert a working copy of the gene permanently into the genome of HSCs, and these genetically modified cells can lead to multiple corrected cell types in the bloodstream, including immune cells, red blood cells and platelets. In addition, HSCs can differentiate into cells of the monocyte macrophage lineage that naturally migrate into various organ systems, and thus gives us an opportunity to deliver genes and proteins directly to those organs, including the brain and the GI tract.

Within the neurometabolic space, in particular, we have understood through our preclinical and clinical programs in MLD, MPS-I and MPS-IIIA how HSC gene therapy can deliver genes and proteins to the CNS to correct neurodegeneration. Here is an example of this natural mechanism at work in Slide 18.

Data shows that there are a population of gene-modified HSCs that can naturally cross the blood-brain barrier, distribute throughout the brain, engraft as microglia and express enzyme that is taken up by neurons. We have seen this approach results in clinical benefits for patients with MLD, and we are also using the same approach for MPS-I and MPS-IIIA. Beyond this, we see that the HSC gene therapy approach could be used to deliver specific genes and proteins for other larger neurodegenerative conditions which have high unmet need.

One of the conditions we are disclosing today, and shown on Slide 19, is a specific genetic subset of frontotemporal dementia, where the underlying pathogenesis has a number of parallels with the neurometabolic conditions that we are already addressing. This program involves a broad strategic alliance with Dr. Alessandra Biffi, Boston Childrens Hospital and Padua University in Italy, to further explore the potential of ex vivo HSC gene therapy in neurometabolic and neurodegenerative conditions.

In other organ systems, such as the GI tract, there are similar mechanisms at work which are illustrated on Slide 20. Tissue resident macrophages in the gut wall are required to respond to bacterial invasion from the gut lumen and prevent infection. In certain disorders, such as X-linked chronic granulomatous disease or XCGD, defects in macrophage function results in an abnormal immune response and severe colitis.

Moving on to Slide 21. We have already seen in our XCGD program the modification of HSCs and migration of gene-modified cells into the gut can lead to resolution of colitis through presumed reconstitution of the immune response. Certain subsets of Crohns disease are also associated with mutations in genes that affect the response of macrophages to infection, and so our clinical observations that HSC gene therapy for XCGD suggest that the same approach may be applicable to this genetic subset of Crohns disease. This preclinical work is ongoing in our Orchard research laboratories.

As we advance our work in FTD and Crohns disease, and assuming we show preclinical proof-of-concept, these will become exciting opportunities for us to expand and address larger patient populations, either alone or in partnership. We believe we have truly just begun to explore the potential for HSC gene therapy in diseases such as these and others, and are excited to share more about the preclinical development of these programs later this year.

So to summarize our path forward on Slide 22, the next 12 to 18 months offers many important milestones as we continue our evolution to a commercial stage company and advance our next wave of clinical stage therapies. We anticipate approval and launch of OTL-200 for MLD in the EU, additional regulatory filings in Wiskott-Aldrich syndrome and MLD, a new registrational study next year in MPS-I, multiple clinical data readouts from our neurometabolic franchise and further detail and progress on our research programs in FTD and Crohns disease.

To wrap up our prepared remarks, we are confident that our new strategic plan and operational decisions announced today will set us on the right path to achieve long-term growth, build sustainable value and serve an even larger number of patients who could benefit from hematopoietic stem cell gene therapy.

Thank you very much. And now well use the rest of the time to answer your questions. So lets have the operator open up the line.

Question-and-Answer Session

Operator

Thank you. [Operator Instructions] And our first question comes from Whitney Ijem from Guggenheim. Your line is now open. Please go ahead.

Whitney Ijem

Hi, guys. Thanks for the question. So first, just wondering, can you give us some more color maybe on the discussions youre having with the FDA in MLD? Kind of what are they looking for? And I guess is the IND just sort of a tool to get RMAT? Or is there additional kind of clinical work you plan you think youll need to do?

Bobby Gaspar

Whitney, Bobby here. Thanks for that. In general, we cant go into all of the details, obviously, of the discussions with the FDA. But I think in the release and in the script, weve talked about the fact that theyve commented on certain endpoints, the natural history, the CMC package, et cetera. Now I think Id just like to say this is a and obviously, a very complex disease, a very ultra-rare population, we have extensive data set, and we have already filed with the EMA. Now for historical reasons, there hasnt been an IND in the U.S., and so we havent had the opportunity to discuss that data in full with the FDA.

What I can say is that we do have an extensive body of data. We want to be able to talk to the FDA and have a comprehensive dialogue to be able to explain that full data set. We feel confident that we have the endpoints that they are looking for and the data that they are asking for. But we need to have that conversation with them in order to be explain to be able to do that fully. So thats why were filing an IND filing, filing the RMAT, so we can have that dialogue. And once we can clarify those issues, then we can go ahead with submission of the BLA.

Whitney Ijem

Okay. Got it. And then just one quick follow-up on MLD. Can you remind us where you are with newborn screening, I guess, both in Europe and then in the U.S.?

Bobby Gaspar

Yes, sure. So newborn screening for MLD, I think, is an important, a very important issue, because, obviously, that means that well be able to get earlier diagnosis and have more patients be able to access therapy. So its a very important part of our kind of diagnostic initiatives in this disease.

What we have so far is that we have worked with a key scientist, where an assay has been developed, thats been published to show that there is an assay that weve done on a dry blood spot to understand the decrease in the enzyme activity and also the increase in the sulfur-type levels. And that assay is now going to be put into pilots, and we are funding a pilot in New York State, and that will start later this year. And were also looking at pilots in other states as well. Were also transferring that assay to Italy and that and were funding a program in Tuscany and in Italy where that will be rolled out. And were also looking for opportunities in other EU states as well. So Id say, there are already two that are going to start, we are looking to fund other pilots as well.

And together, that data will allow us to validate the assay but also allow wider implementation of newborn screening, and also for nomination, for example, onto the WAS panel for implementation in states in the U.S. So I say theres a lot of work going on in order to make sure that happens.

Whitney Ijem

Great. Thanks.

Operator

Thank you. And your next question comes from Esther Rajavelu from Oppenheimer. Your line is now open. Please go ahead.

Esther Rajavelu

Hey, guys. Congrats on all the changes. I guess, my first question again on MLD is Im trying to understand the duration between EU approval and NBS. I dont know if that math or if that graph was drawn to scale, but it looks like its almost a four-year lag from first approval to newborn screening. Can you help us understand the time line there?

Frank Thomas

You mean between EU and U.S. or around newborn screening or both?

Esther Rajavelu

Around newborn screening, generally, between EU approval and newborn screening.

Frank Thomas

Yes, sure. As Bobby mentioned, theres a pretty active program planned around newborn screening that I think we will expect will come over time in order to even apply for the Ross Panel, there are certain requirements that need to be met in terms of the number of patients or a number of children that have to be screened, identifying the positive patients and then you can apply on the Ross Panel.

And then from there, theres a process that you go through in the U.S., at least, on a state-by-state basis to get it added. So I think there are a number of steps along the way. We havent guided specifically on the time line, but I think there are other precedents out there that suggest that this could take years. Once we screen the once we apply for the Ross Panel to get sort of full reimbursement, but obviously, well focus on states initially after that approval that have the largest populations.

Esther Rajavelu

And my Yes, go ahead.

Bobby Gaspar

Esther for I was just going to say for the EU, obviously, were looking for approval for MLD later this year. As far as people screening in the EU is concerned, thats on a country-by-country basis, and sometimes its even certain states. But Ive worked on newborn screening for SCID, for example, in the EU. And now there are numerous countries in the EU that are screening for SCID with a number of pilots also in the pipeline as well. And so with that kind of experience, and we would be looking to kind of really facilitate that uptake in the EU and as in and in the U.S., as Frank has already mentioned.

Esther Rajavelu

Understood. And then the decision to defer CapEx, is that related to some of the time lines for U.S. versus EU approvals and the newborn screening? Or what really kind of went into that delay, given you already have some cost into that facility?

Frank Thomas

Yes. I can start, and Bobby can add on that again. I think, obviously, we continue to believe in-house manufacturing is an important capability that were going to want to have over some period of time. It really comes down to sort of when is the need for that capacity and capability relative to the various programs we have. Working with the CDMOs that we have today, we know that we have capacity for the MLD and WAS launches and for a period beyond the launch. So theres not an imminent need to secure the capacity today, and we think that deferring it makes the most sense. Well continue to work with CDMOs on those launches. We will look at bringing on a U.S. supplier for drug product to be able to more easily service the U.S. market.

And then longer term, look at, potentially, in-house manufacturing at a site and location that we think is more fitted to what the capacity needs will be. So I wouldnt say its tied to any sort of launch time lines because the plan always was to utilize CDMOs for WAS and MLD. But certainly, as those launches roll out and demand grows, our capacity needs will grow and that will be the appropriate time, we think, to make the investment.

Esther Rajavelu

Understood. Thank you very much.

Operator

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Orchard Therapeutics' (ORTX) CEO Bobby Gaspar on Q1 2020 Results - Earnings Call Transcript - Seeking Alpha

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Gene Editing Technologies Market What Factors will drive the Gene Editing Technologies Market in Upcoming Years and How it is Going to Impact on...

A high-dose chemotherapy combination given to wipe out the immune system before its rescue withautologous hematopoietic stem cell transplant (aHSCT)can cause transient damage to neurons and supporting cells of the central nervous system in people with aggressive multiple sclerosis (MS), a Canadian study reports.

Nonetheless, its researchers believe that the risk of such neuronal toxicity is offset by the benefits of the aHSCT procedure in aggressive MS cases.

The study, Neurotoxicity after hematopoietic stem cell transplant in multiple sclerosis, was published in the journal Annals of Clinical and Translational Neurology.

aHSCT is an intensive yet effective treatment, particularly used to rebuild the immune system of people with aggressive MS.

The procedure begins with collecting a patients own (meaning autologous) healthy hematopoieticstem cellsfrom the bone marrow, followed by immunoablation the wiping out of the immune system with a high-dose chemotherapy combination that kills the patients immune cells. The hematopoieticstem cellsare then infused back to the person to generate a new and healthy immune system.

Intensity and duration of the chemotherapy given varies among centers.

In Canada, the chemotherapy regimen used in clinical practice is particularly intense and involves high doses of two agents, cyclophosphamide and busulfan. This combination is known to penetrate the central nervous system (CNS, brain and spinal cord), and to kill the CNS immune cells (called microglia) that go awry in MS.

Previously, researchers in Canada noted faster brain volume loss in MS patients over the six months following aHSCT. This was interpreted as a false (pseudo) shrinkage of brain volume due to lesser inflammation.They also thought that the toxicity related to the chemotherapy regimen itself could be a contributing factor, as chemotherapy can affect cognitive and memory abilities what patients refer to as chemo brain and chemo fog.

To test these interpretations, a team led by researchers at the University of Ottawameasured the levels of two markers serum neurofilament light chain (sNfL)and serum glial fibrillary acidic protein (sGFAP) in the blood (serum) of MS patients before and after the chemotherapy regimen in aHSCT.

sNfL has been proposed as a marker of neuron degeneration and damage, and sGFAP is a marker of astrocytes, cells involved in the provision of nutrients to neurons, the repair of nervous tissue following injury, and in facilitating neurotransmission (essential in nerve cell communication).

Researchers measured the levels of these two markers in blood samples from 22 MS patients with aggressive disease, all enrolled in the Phase 2 BMT trial (NCT01099930) in Canada. Blood samples were collected at specific times (three, six, nine, and 12 months) before and after aHSCT. Aggressive disease for this study was defined as having a poor predicted 10-year prognosis [likely course].

Blood samples from another 28 people of similar ages and with non-inflammatory diseases (including migraine, fibromyalgia, anxiety, Bells palsy, labyrinthitis, and depression) were used as controls.

sNfL levels prior to aHSCT were seen to be significantly elevated in the blood of MS patients (mean of 21.8 pg/mL) compared with controls (6.4 pg/mL). So too were blood levels of sGFAP: 107.4 pg/mL in MS patients versus 50.7 pg/mL in controls.

Three months after aHSCT, levels of sNfL significantly increased by 32.1% (from 21.8 to 28.8 pg/mL), and sGFAP levels by 74.8% (from 107.4 to 187.7 pg/mL) in 15 patients with available three-month data, compared to their pre-treatment levels. In two of these 15 people, both sNfL and sGFAP levels were lower three months after treatment.

Further analysis looked at changes from baseline (study start) and three-month post-aHSCT measures of sNfL levels and grey matter volume loss. This was done to look for correlations between early serum biomarker changes with clinical and MRI markers of possible neurotoxicity, the researchers wrote.

Their analysis found correlations at all early time points months one, two, four and six but not at nine or 12months post-treatment, highlighting that associated increases in both sNfL and MRI grey matter volume loss are temporary phenomena in the first year following IHASCT, they wrote.

Patients given the highest dose of busulfan (more than 700 mg) had the highest levels of sNfL and a greater extend of grey matter loss, shown by MRI, at three months post-treatment.

The chemotherapy regimen used increases sNfL and sGFAP levels in the first few months after treatment, the study noted, with sNfL increases correlated with the total busulfan dose given. The result is a transient worsening of posttreatment EDSS score as well as MRI volume loss, especially in the grey matter, the researchers wrote.

This study is the first to correlate these increases [rises in brain atrophy and serum markers of neurotoxicity] with doses of chemotherapy administered in aHSCT, they added.

Although we see transient neurotoxicity likely related to busulfan in this study, this has to put in the context of their highly active refractory disease, the team concluded. While the long-term effects of chemotherapeutic neurotoxicity are not known, in cases of aggressive MS, short-term chemotherapy-related neuronal toxicity may be a risk that is greatly offset by the benefit of long-term freedom from disease-related inflammation.

Patricia holds her Ph.D. in Cell Biology from University Nova de Lisboa, and has served as an author on several research projects and fellowships, as well as major grant applications for European Agencies. She also served as a PhD student research assistant in the Laboratory of Doctor David A. Fidock, Department of Microbiology & Immunology, Columbia University, New York.

Total Posts: 1,053

Patrcia holds her PhD in Medical Microbiology and Infectious Diseases from the Leiden University Medical Center in Leiden, The Netherlands. She has studied Applied Biology at Universidade do Minho and was a postdoctoral research fellow at Instituto de Medicina Molecular in Lisbon, Portugal. Her work has been focused on molecular genetic traits of infectious agents such as viruses and parasites.

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'Transcient' Damage to CNS Seen with Chemotherapy Used in Stem... - Multiple Sclerosis News Today