Archive for February, 2021

Andrew Brooks, a research professor at Rutgers University who developed the first saliva test for the coronavirus, died on Jan. 23 in Manhattan. He was 51.

The cause was a heart attack, his sister, Janet Green, said.

In April 2020, when coronavirus tests were scarce and lines to get them long, Dr. Brooks made worldwide news when the Food and Drug Administration gave emergency approval to his technique, which promised to radically increase the speed and safety of the testing process.

Instead of having a naso- or oropharyngeal swab thats placed in your nose or the back of your throat, you simply have to spit in a tube, he told Bill Hemmer of Fox News, adding, It doesnt require a health care worker to collect it, six inches away from an infected person.

In the 10 months since Dr. Brooks received approval, health care workers have performed more than four million tests using his approach, and it remains one of the most reliable means of determining whether someone has the coronavirus.

In a statement, Gov. Phil Murphy of New Jersey called Dr. Brooks one of the states unsung heroes who had undoubtedly saved lives.

Andrew Ira Brooks was born on Feb. 10, 1969, in Bronxville, N.Y. His father, Perry H. Brooks, was a diamond setter. His mother, Phyllis (Heitner) Brooks, was a schoolteacher.

In addition to his sister, he is survived by his mother; his wife, Jil (Larsen) Brooks; and three daughters, Lauren, Hannah and Danielle. His first two marriages ended in divorce.

Dr. Brooks grew up in Old Bridge, N.J., where he earned spending money by performing magic shows at birthday parties. Though he was adept at tricks involving doves and rabbits, his real forte was close-up handwork, especially card tricks.

He played varsity golf at Cornell, where he majored in animal sciences, having initially planned to become a veterinarian. But after a summer internship at the Memorial Sloan Kettering Cancer Center in Manhattan, he grew fascinated with the study of human disease. He received a doctorate in microbiology and immunology from the University of Rochester in 2000.

After working at the universitys medical center for four years, he returned to New Jersey to take a job at Rutgers, and in 2009 joined its Cell and DNA Repository, a university-owned company that provides data management and analysis for biological research.

Feb. 6, 2021, 6:56 p.m. ET

Dr. Brooks became the companys chief operating officer, discovering that he had a flair for the business side of science. He expanded the company from just a few dozen employees to nearly 250, working with nearly every major pharmaceutical company.

Most scientists I meet are not interested, or are incidentally interested, in the commercialization of what they do, said Dr. Jay Tischfield, a Rutgers professor and chief executive of the repository. Andy understood that if you want something to get out and be used, you have to be a player. You cant rely on other people.

In 2018 the company, by then called the Rutgers University Cell and DNA Repository Infinite Biologics, decided to go private; Dr. Brooks was named chief executive. The university agreed with the move but kept a significant stake in the new company, now called Infinity Biologix.

The resources and experience he accrued at the repository made it relatively easy for Dr. Brooks to develop the coronavirus spit test, which he did in partnership with two other companies, Spectrum Solutions and Accurate Diagnostics Labs.

He was used to doing genetic testing through saliva, and it wasnt rocket science, Dr. Tischfield said, to adapt those techniques to extract RNA from the coronavirus. The company even had thousands of tubes on hand that it could use to collect samples.

After the F.D.A. granted approval, Dr. Brooks faced a different challenge: scale. He needed significantly more equipment and staff, immediately, to produce the tests and process the results. But a propitious call from the White House offering help, and a multimillion-dollar loan arranged by Dr. Tischfield allowed the company to add additional analytic equipment rapidly and to nearly double its work force almost overnight.

As important as the actual saliva test was, it was Dr. Brookss ability to rapidly scale up the operation in the middle of taking it private that most impressed Dr. Tischfield.

Ive been doing this for 50 years, and Ive met all kinds of people, he said. But Andy, he was a force of nature.

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Andrew Brooks, Who Developed a Coronavirus Spit Test, Dies at 51 - The New York Times

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Preimplantation genetic testing (PGT) (PGS/PGD) market garnered a revenue of USD 98.23 million in the year 2019 globally and has been foreseen to yield USD 201.32 million by the year 2027 at a compound annual growth (CAGR) of 5.1% over the forecast period.

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Market Segments by Major Manufacturers:

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Preimplantation Genetic Testing (PGT) (PGS/PGD) Market, By Application (2016-2027)

Preimplantation Genetic Testing (PGT) (PGS/PGD) Market, By PGD/PGS (2016-2027)

Preimplantation Genetic Testing (PGT) (PGS/PGD) Market, By Type (2016-2027)

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North America (USA, Canada)Latin America (Chile, Brazil, Argentina, rest of Latin America)Europe (UK, Italy, Germany, France, rest of the EU)Asia Pacific (India, Japan, China, South Korea, Australia, rest of APAC)Middle East and Africa (Saudi Arabia, United Arab Emirates, South Africa, rest of MEA)

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Preimplantation Genetic Testing (PGT) (PGS/PGD) Market Size, Top Key Players, Applications, Business Statistics, Trends and Forecast 2021-2027 ...

A new market study is released on Global Animal Genetic Market with data Tables for historical and forecast years represented with Chats & Graphs spread through Pages with easy to understand detailed analysis. This report is delivered as the most relevant, unique, fair, and creditable global market research report to valuable customers and clients depending upon their specific business needs. It facilitates in adjusting the production depending on the conditions of demand which avoids wastage of goods. Market research reports like this Global Animal Genetic Market surely helps to reduce business risk and failure. Major competitor strategies include but are not limited to new product launches, expansions, agreements, joint ventures, partnerships, and acquisitions. Research and analysis is carried out with one step or the combination of several steps depending upon the client need and the business requirements.

Global Animal Genetic Market Statistical Overview Report 2021 gives an outstanding tool for market Survey, openings, and vital key and strategic basic leadership. This report perceives that in this quickly advancing and competitive scenario by up-coming data on the basis of research execution and settled on basic choices for development and benefit. It gives data on Latest trends and advancements and sheds light on various sectors, limitations and advancements, and on the evolving structure of the market.

Global Animal Genetic Marketis expected to rise from its estimated value of USD 4.39 billion in 2018 to an estimated value of USD 8.68 billion by 2026 registering a CAGR of 8.9% in the forecast period of 2019-2026.The upcoming market report contains data for historic years 2017, the base year of calculation is 2018 and the forecast period is 2019 to 2026.

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Few of the major market competitors currently working in the animal genetics market areNEOGEN CORPORATION, Zoetis, Envigo, Animal Genetics Inc., VetGen, Groupe Grimaud, Hendrix Genetics BV, EW Nutrition GmbH, Alta Genetics Inc., Genus, Topigs Norsvin, CRV Holding B.V., URUS, Trans Ova Genetics., Inguran LLC dba STgenetics., Semex, Beacon Automation Pty Ltd., Cogent, EVOLUTION International , Genex Services, LLC, Rockway, Inc., River Valley Dairy., ABS GLOBAL, INC., Anicam Enterprises Inc., Milk Source. among others

Competitive Analysis: Global Animal Genetic Market

The global animal genetics market is highly fragmented and the major players have used various strategies such as new product launches, expansions, agreements, joint ventures, partnerships, acquisitions, and others to increase their footprints in this market. The report includes market shares of global animal genetics market for Global, Europe, North America, Asia Pacific, South America and Middle East & Africa.

Global Animal Genetic Market By Product (Live Animals (Poultry, Porcine, Bovines, Canine, Others), Genetic Material (Semens, Embroys)), Genetic Testing Services (DNA typing, genetic trait tests, genetic disease tests, and others), Geography (North America, South America, Europe, Asia-Pacific and Middle East and Africa) Industry Trends & Forecast to 2026;

Market Definition: Global Animal Genetic Market

Animal genetics is the branch of science which deals with the study of inheritance and gene variation in domestic and wild animals. Animal genetics are mostly used for genetic trait testing, DNA testing, and genetic disease traiting. The animal genetics market is expected to increase due to the fast demand, ingesting of animal proteins, and surge in urban population, which shows demand for meat products across the globe during the forecast period.

Market Drivers

Market Restraints

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Segmentation: Global Animal Genetic Market

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Sana Biotechnology led the way with a very large initial public offering (IPO) that could bring the companys market value to $4.6 billion or more. But its not the only IPO this week. Heres a look.

Sana Biotechnology Sana announced plans to raise $517 million with 22 million shares at a price range of $23 to $24 per share. The company previously filed to offer 15 million shares at a range of $20 to $23. At the midpoint of the revised range, the company plans to raise 60% more in proceeds than they had previously expected.

The company is working to develop in vivo and ex vivo cell engineering platforms for oncology, diabetes, central nervous system disorders, cardiovascular diseases and genetic disorders.

On October 30, 2020, Sana acquired Oscine Corp., which focuses on developing cell therapies for brain and CNS disorders. Oscine offers up a glial progenitor cell program and various underlying technologies. Glial progenitor cells play a role in supporting the brain.

Sana has a broad preclinical pipeline with 11 programs ranging from non-Hodgkin lymphoma (NHL), acute lymphoblastic leukemia (ALL), chronic lymphocytic leukemia (CLL) and multiple myeloma to sickle cell disease, beta-thalassemia, type 1 diabetes and others. The company raised more than $700 million in private funding and will have a market value of about $4.6 billion. Theyre also offering an additional 30-day option for another 3.525 million shares to underwriters, which if they are fully exercised, could bring the IPO raised to $675.6 million.

Sensei Biotherapeutics Sensei announced the pricing of its upsized IPO of 7,000,052 shares of common stock at $19 per share. The company hopes to raise $133 million. On January 11, 2021, Sensei announced the closing of a $30 million Series B financing, co-led by Apeiron Investment Group and Catalio Capital Management. Additional investors included Pura Vida, several international family offices, and Cambrina Biopharma, Moore Strategic Ventures, Steve Jurvetsons Future Ventures, and Presight Capital.

Sensei focuses on immunotherapies for cancer and infectious diseases. It has developed a phage-based platform, ImmunoPhage. A phage is a virus that infects bacteria. This platform allows for the rapid creation of immune activating agents that fully engage the immune system. It has used it to develop a library of ImmunoPhage called Phortress targeting multiple tumor associated antigens. Its most advanced program is SNS-301, a first-in-class ImmunoPhage targeting Aspartyl beta Hydroxylase (ASPH) in advanced squamous cell carcinoma of the head and neck (SCCHN).

Landos Biopharma Landos announced plans to raise $100 million for its IPO of 6,250,000 shares of common stock at $16 per share. Landos is working to develop novel oral therapeutics for autoimmune diseases. On January 4, 2021, the company announced positive results from a first-in-patients 12-week Phase II proof-of-concept trial of BT-11, an oral gut-restricted LANCL2 modulator in mild to moderate ulcerative colitis (UC).

We are excited by the very promising Phase II trial results evaluating BT-11, as it marks the first potential therapeutic to engage the novel target LANCL2, said Josep Bassaganya-Riera, chairman, president and chief executive officer of Landos. We believe this novel mechanism of action is responsible for modulating key immunological mechanisms associated with various autoimmune diseases such as ulcerative colitis and Crohns disease.

Aspira Womens Health Aspira announced plans to raise $45 million with its IPO of 6 million shares at a price of $7.50 per share. It plans to use the proceeds for sales and marketing, working capital and other general corporate purposes. The company, formerly known as Vermillion, is developing and commercializing innovative testing options and bio-analytical solutions to help doctors evaluate risk, optimize patient management and improve gynecologic health. OV1 plus combines its OVA1 and OVERA to detect risk of ovarian malignancy in women with adnexal masses. Aspira GenetiX offers targeted and comprehensive genetic testing options with a gynecologic focus.

On November 19, 2020, the company inked a collaborative research deal with Baylor Genetics in Houston to co-develop a novel Ovarian Cancer early-detection test.

We look forward to combining the strengths from each respective research team, and working together on our OVA360 clinical study, specifically in our mutual goal of characterizing an Ovarian Cancer molecular profile, said Lesley Northrop, chief scientific officer at Aspira, in a November statement.

Equillium Equillium entered into a securities purchase agreement with life science institutional investment funds managed by Decheng Capital, to acquire 4,285,710 units from Equilliuim. Each unit consists of one share of common stock and a warrant to purchase 0.3 of a share of common stock. The purchase price per unit is $7, priced above the market under Nasdaq rules. The warrants will have an exercise price of $14 per share, will be immediately exercisable, and will expire on the earlier of the fifth anniversary of issuance or the 15th calendar date after the data on which Equillium closes a financing raising a minimum of $25 million at a price per share of no less than $25.

Equillium is focused on immunology and developing new products for severe autoimmune and inflammatory disorders. On December 7, 2020, the company presented data at the 2020 American Society of Hematology (ASH) Annual Meeting and Exposition on the impact of itolizumab on effector T cell function in graft-versus-host autoimmune and inflammatory disorders. The findings demonstrated that the CD6-ALCAM pathway is crucial in modulating effector T cell function, and that inhibition of CD6 signaling with itolizumab can modulate multiple effector T cell subsets.

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Life Sciences Companies Ringing the Nasdaq Bell this Week - BioSpace

GlobalStem Cell Therapy Market,by Cell Source (Adult Stem Cells, Induced Pluripotent Stem Cells, Embryonic Stem Cells, and Others), by Application (Musculoskeletal Disorders, Wounds and Injuries, Cancer, Autoimmune Disorders, and Others), and byRegion(North America, Latin America, Europe, Asia Pacific, Middle East, and Africa),was valued atUS$ 7,313.6million in 2018, and is expected to exhibit a CAGR of21.1%over the forecast period (2019-2027),as highlighted in a new report published by .Increasing application of stem cells for the treatment of patients with blood-related cancers, spinal cord injury and other diseases are the leading factors that are expected to drive growth of stem cell therapy market over the forecast period.

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According to the National Spinal Cord Injury Statistical Center, 2016, the annual incidence of spinal cord injury (SCI) is approximately 54 cases per million population in the U.S. or approximately 17,000 new SCI cases each year. Moreover, according to the Leukemia and Lymphoma Society, 2017, around 172,910 people in the U.S. were diagnosed with leukemia, lymphoma or myeloma in 2017, thus leading to increasing adoption of stem cells for its efficient treatment. Increasing product launches by key players such as medium for developing embryonic stem cells is expected to propel the market growth over the forecast period.

For instance, in January 2019, STEMCELL Technologies launched mTeSR Plus, a feeder-free human pluripotent stem cell (hPSC) maintenance medium for avoiding conditions associated with DNA damage, genomic instability, and growth arrest in hPSCs. With the launch of mTeSR, the company has expanded its portfolio of mediums for maintenance of human embryonic stem (ES) cells and induced pluripotent stem (iPS) cells.

Increasing research and development of induced pluripotent stem cells coupled with clinical trials is expected to boost growth of the stem cell therapy market over the forecast period. For instance, in April 2019, Fate Therapeutics in collaboration with UC San Diego researchers launched Off-the-shelf immunotherapy (FT500) developed from human induced pluripotent stem cells. The therapy is currently undergoing clinical trials for the treatment of advanced solid tumors.

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North America is expected to hold a dominant position in the stem cell therapy market over the forecast period, owing increasing number of clinical trials to cater to unmet medical needs of the patients is a major factor driving growth of the stem cell therapy market. For instance, in April, 2019, UCLA-UCI Alpha Stem Cell Clinic participated in a new clinical research study to investigate a stem cell product CTX0E03 DP, in order to improve function in people with chronic disability from ischemic stroke. The study, called PISCES III, is currently in phase IIb clinical study. Moreover, manufacturers are focused on collaborating with academic researchers to help expand the potential use of newborn stem cell therapies that may be available to patients, which is expected to facilitate growth of the market over the forecast period. For instance, in February 2018, the Institute of Integrative Biology entered into a collaboration with Anika Therapeutics, Inc., to develop an injectable mesenchymal stem cell (MSC) therapy for the treatment of osteoarthritis in patients.

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Stem Cell Therapy Market to Score Past US$ 40.3 Billion Valuation by 2027: At a CAGR of 21.1% KSU | The Sentinel Newspaper - KSU | The Sentinel...

Colon cancer, also called colorectal cancer, is an umbrella term for cancer that starts anywhere in the colon or rectum. The American Cancer Society estimates that there will be 104,270 new cases of colon cancer this year.

Some cases of colon cancer will be MSI-high, or MSI-H, which stands for microsatellite instability-high. It refers to a piece of genetic coding and means theres a lot of instability in the tumor.

Your MSI status gives your doctor an idea of how the cancer will behave. It can also help guide treatment decisions.

MSI-high colon cancer involves tumors with a high amount of instability. It occurs when mismatch repair (MMR) genes, whose job is to correct errors that happen during cell division, stop functioning properly.

When the MMR system is defective, it stops making repairs, allowing errors to accumulate. Thats how a tumor becomes highly unstable.

Doctors will use a tissue biopsy, usually following surgery, to test whether a tumor is MSI-high.

About 15 percent of colon cancer tumors are MSI-high, according to a 2016 study. About 3 percent of these are associated with a hereditary disorder called Lynch syndrome.

MSI-high cancer cells look and behave in an abnormal way. Thats not necessarily a bad thing when it comes to colon cancer, though.

While many cancer cells can easily hide from the immune system, MSI-high cancer cells stand out. That makes it easier for the immune system to recognize them as invaders. They also tend to respond well to treatment.

Treatment for colon cancer depends on several factors, such as the stage and location of the tumors. MSI status can play an important role in forming a treatment plan.

Here are some treatment options for colon cancer:

Surgery can remove many tumors in the colon.

In a procedure called segmental colectomy, the surgeon removes part of the colon, then attaches the ends together. In the early stages of colon cancer, surgery may be the only treatment you need.

Cancer that has spread to other organs or tissues can sometimes be surgically removed as well.

Regional and systemic chemotherapy can help shrink tumors and prevent cancer from spreading. It can be used alone or in combination with other treatments.

Chemotherapy drugs used to treat colon cancer include:

Immunotherapy is a promising treatment choice for many types of cancer. Its a way of strengthening your own immune system to fight cancer.

Three immune checkpoint inhibitors are approved to treat MSI-high metastatic colon cancer. Theyre all given through intravenous infusions.

Two of these drugs can only be used when cancer has progressed after treatment with certain chemotherapy drugs.

In 2017, the Food and Drug Administration (FDA) granted accelerated approval to nivolumab (Opdivo).

A year later, the agency granted accelerated approval to ipilimumab (Yervoy). This drug can only be used in combination with nivolumab, not by itself.

Theres also pembrolizumab (Keytruda). Its a first-line immunotherapy for metastatic MSI-high colon cancer. That means you dont have to try chemotherapy first. It was approved by the FDA in 2020.

In a clinical trial, researchers compared pembrolizumab to chemotherapy as a first-line therapy for MSI-high metastatic colon cancer. Pembrolizumab led to significantly longer progression-free survival. Trial participants who were treated with pembrolizumab also had fewer adverse events than those in the chemotherapy group.

Targeted therapies for colon cancer help prevent tumors from forming new blood vessels. These include:

Theyre administered intravenously, often in combination with chemotherapy.

Other drugs target epidermal growth factor receptor (EGFR), a protein that helps cancer grow. Some treatments include cetuximab (Erbitux) and panitumumab (Vectibix).

For cancer involving BRAF gene mutations, doctors may use targeted therapy drugs such as encorafenib (Braftovi) and regorafenib (Stivarga).

Radiation targets high-energy rays to a specific area of the body. This can help shrink tumors and kill cancer cells.

Treatment options for people with colon cancer have changed a lot in recent years, and theres more to come.

Clinical trials help test innovative new therapies. Ask your doctor about clinical trials that may be a good fit for you.

According to the American Cancer Society, the 5-year relative survival rate for colon cancer is:

Keep in mind that these numbers are based on people diagnosed between 2010 and 2016. Thats before immunotherapies were approved to treat colon cancer. The outlook for people with the disease may be better today.

Outlook may be better in people with MSI-high colon cancer compared with MSI-low cancer. According to a 2018 study on 603 people who had surgery for colon cancer, those with MSI-high cancer had a slightly better survival rate.

While statistics can give you a sense of the outlook for people with colon cancer in general, your personal outlook can be different. It depends on a variety of factors, such as:

Your doctor can review your health history to give you a better idea of what to expect.

MSI stands for microsatellite instability. MSI-high is a subset of colon cancer in which the tumor cells have a lot of instability. These cancer cells look and behave in an abnormal way.

The striking abnormalities make it hard for cancer cells to hide from the immune system. In general, MSI-high colon cancers respond better to treatment than other colon cancers.

Within the last few years, three immunotherapy drugs were approved to treat metastatic MSI-high colon cancers.

Two are for use when cancer hasnt responded to chemotherapy.

The third, pembrolizumab, is now approved as a first-line treatment for metastatic MSI-high colon cancer.

Talk with your doctor to find out which MSI-high colon cancer treatment options are right for you.

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MSI-High and Colon Cancer: What It Means, Treatment Options, and More - Healthline

Targeted therapies are medications that block certain molecules that affect cancers ability to grow and spread. They are different from standard chemotherapy and have several potential benefits as a lung cancer treatment.

According to Dr. Timothy Byun, a hematologist and oncologist with the Center for Cancer Prevention and Treatment at St. Joseph Hospital in Orange County, CA, some Food and Drug Administration (FDA)-approved medications target specific genetic mutations that are responsible for lung cancer.

These drugs are often the therapy of choice for people with advanced stage non-small cell lung cancer (NSCLC), said Dr. Byun. NSCLC accounts for an estimated 84% of all lung cancer diagnoses, according to the American Cancer Society.

In general, targeted therapies have higher response rates and [a] longer duration of benefit compared [with] chemotherapy in these patient subsets, said Dr. Byun.

However, targeted therapies also have some potential drawbacks. Dr. Byun explained that, as with chemotherapy, a persons cancer may develop a resistance to these medications. A person may also develop some side effects from taking these medications.

There are several different targeted therapies for people with lung cancer. Individuals should talk with their doctor about which option is best for them.

Here are the most common mutations involved in lung cancer, the targeted therapies that treat them, and some potential side effects related to taking these medications.

Cancerous tumors need a steady blood supply to grow. Angiogenesis inhibitors are a type of long-term medication that targets the blood vessels supporting cancer cells. By blocking the growth of these blood vessels, this targeted treatment can slow or stop the growth of lung cancer.

A doctor is most likely to prescribe bevacizumab (Avastin) or ramucirumab (Cyramza) to treat lung cancer.

Side effects vary based on which part of the blood cells the medication targets. Some common side effects can include:

Serious side effects are more rare. These may include:

Epidermal growth factor receptor (EGFR) is a protein on the outside of cells that controls their growth and division. EGFR genetic mutations can cause too much EGFR to be present on cells, which causes cancer to grow faster.

EGFR mutations affect around 3745% of people with lung cancer who dont smoke and 714% of people with lung cancer who do smoke.

For advanced stage NSCLC with common EGFR mutations, EGFR targeted therapy such as osimertinib is the preferred first line of treatment, said Dr. Byun.

EGFR inhibitors block the signals from EGFR to prevent the growth and division of cancer cells.

Some common EGFR inhibitors for lung cancer include:

Some common side effects of EGFR inhibitors can include:

These side effects can be severe enough that doctors recommend that people stop taking EGFR inhibitors.

For example, said Dr. Byun, if a patient experiences a severe adverse reaction such as interstitial lung disease related to osimertinib, which occurs infrequently, then the medication is discontinued and the patient would be considered for chemotherapy.

According to one 2018 study, most people with lung cancer develop resistance to EGFR inhibitors. This is often because the cancer cells develop new EGFR mutations. One common mutation is called T790M.

Osimertinib (Tagrisso) is a drug that targets cells with the T790M mutation. Side effects of this drug are similar to those of other EGFR inhibitors.

Necitumumab (Portrazza) is an EGFR inhibitor that mimics immune system proteins.

A doctor will give the medication intravenously and will often prescribe it with chemotherapy for people with advanced squamous cell NSCLC. It can cause similar side effects to those of other EGFR inhibitors.

Anaplastic lymphoma kinase (ALK) is a protein that supports cell growth. Some people with lung cancer have a mutation in the ALK gene, which causes more ALK proteins to form. This drives cancer growth and spread.

ALK mutations occur in about 12% of people who dont smoke and 2% of people who currently or who used to smoke.

ALK inhibitors are medications that target cancers with the ALK mutation. Certain people can use them after or instead of chemotherapy.

Some examples of these drugs include:

It is possible to experience side effects from taking this drug, including:

Some more severe and rare side effects include:

The American Cancer Society estimate that 12% of NSCLC cases have a ROS1 mutation, which affects how cancer cells grow and spread.

According to one recent study, ROS1 inhibitors are a well-established targeted therapy for people with a type of NSCLC lung cancer known as adenocarcinoma. Adenocarcinoma accounts for 5060% of lung cancer in people who dont smoke.

If a doctor identifies a ROS1 mutation, they may prescribe one of the following medications:

People can use these medications instead of chemotherapy or when chemotherapy or other therapies stop working.

Some potential side effects may include:

More severe side effects may include:

About 12% of people with NSCLC develop RET gene mutations. These changes affect a protein involved in cell growth, and they often occur with other mutations. As a result, a doctor may prescribe RET-targeting medications with EGFR-targeting medications for the best effect against cancer.

Medications to target RET genes include selpercatinib (Retevmo) and pralsetinib (Gavreto).

Some common side effects can include:

More severe and rare side effects may include:

According to one 2015 study, changes in the mesenchymal-epithelial transition (MET) gene are present in about 5% of lung cancer cases. The MET gene creates the MET protein, which is involved in cell invasion, growth, migration, and metastasis.

Capmatinib (Tabrecta) is the first FDA-approved MET-targeting medication for treating metastatic NSCLC.

Researchers are currently studying many other types of MET-inhibitor, including emibetuzumab, tivantinib, cabozantiniband, and onartuzumab.

Some potential side effects of these drugs may include:

Rarely, these drugs can cause liver damage and swelling or scarring in the lungs.

Recent research suggests that very few people with NSCLC develop an NTRK gene change that can cause the cancer to grow and spread.

Doctors typically prescribe two medications for this mutation in people with advanced lung cancer: larotrectinib (Vitrakvi) and entrectinib (Rozlytrek). Both are typically given when the cancer does not respond to other treatments.

Some potential side effects include:

Some other, less common side effects include:

A number of different drugs provide targeted treatment for many people with lung cancer.

These medications work by honing in on certain mutations in genes involved in lung cancer and stopping the reproduction, growth, and spread of cancer cells.

People may use targeted therapies with, instead of, or following other cancer therapies, including chemotherapy.

Continue reading here:
Targeted therapy for lung cancer: Side effects and more - Medical News Today

New York, Feb. 05, 2021 (GLOBE NEWSWIRE) -- Reportlinker.com announces the release of the report "Gene Therapy Market by Therapeutic Approach, Type of Gene Therapy, Type of Vectors Used, Therapeutic Areas, Route of Administration, and Key Geographical Regions: Industry Trends and Global Forecasts, 2020-2030" - https://www.reportlinker.com/p06020737/?utm_source=GNW Considering the current pace of research and product development activity in this field, experts believe that the number of clinical research initiatives involving gene therapies are likely to grow by 17% annually. In this context, the USFDA released a notification, mentioning that it now expects to receive twice as many gene therapy applications each year, starting 2020. Despite the ongoing pandemic, it is worth highlighting that gene therapy companies raised approximately USD 5.5 billion in capital investments, in 2020 alone. This is indicative of the promising therapeutic potential of this emerging class of pharmacological interventions, which has led investors to bet heavily on the success of different gene therapy candidates in the long term.

Several technology platforms are currently available for discovery and development of various types of gene therapies. In fact, advances in bioanalytical methods (such as genome sequencing), and genome editing and manipulation technologies (such as molecular switches), have enabled the development of novel therapy development tools / platforms. In fact, technology licensing is a lucrative source of income for stakeholders in this industry, particularly for those with proprietary gene editing platforms. Given the growing demand for interventions that focus on the amelioration of the underlying (genetic) causes of diseases, it is expected that the gene therapy pipeline will continue to steadily expand. Moreover, promising results from ongoing clinical research initiatives are likely to bring in more investments to support therapy product development initiatives in this domain. Therefore, we are led to believe that the global gene therapy market is poised to witness significant growth in the foreseen future.

SCOPE OF THE REPORT The Gene Therapy Market (4th Edition) by Therapeutic Approach (Gene Augmentation, Oncolytic Viral Therapy, Immunotherapy and Others), Type of Gene Therapy (Ex vivo and In vivo), Type of Vectors used (Adeno Associated Virus, Adenovirus, Herpes Simplex Virus, Lentivirus, Plasmid DNA, Retrovirus and Others), Target Therapeutic Areas (Autoimmune Disorders, Cardiovascular Diseases, Dermatological Disorders, Genetic Disorders, Hematological Disorders, Metabolic Disorders, Muscle-related Diseases, Oncological Disorders, Ophthalmic Diseases and Others), Route of Administration (Intraarticular, Intracerebellar, Intradermal, Intramuscular, Intratumoral, Intravenous, Intravesical, Intravitreal, Subretinal and Others), and Key Geographical Regions (US, EU5 and rest of the world): Industry Trends and Global Forecasts, 2020-2030 report features an extensive study of the current market landscape of gene therapies, primarily focusing on gene augmentation-based therapies, oncolytic viral therapies, immunotherapies and gene editing therapies. The study also features an elaborate discussion on the future potential of this evolving market.

Amongst other elements, the report features: - A detailed review of the overall market landscape of gene therapies and gene editing therapies, including information on phase of development (marketed, clinical, preclinical and discovery) of pipeline candidates, key therapeutic areas (autoimmune disorders, cardiovascular diseases, dermatological disorders, genetic disorders, hematological disorders, immunological disorders, infectious diseases, inflammatory disorders, liver diseases, metabolic disorders, muscle-related diseases, nervous system disorders, oncological disorders, ophthalmic diseases and others), target disease indication(s), type of vector used, type of gene, therapeutic approach (gene augmentation, oncolytic viral therapy and others), type of gene therapy (ex vivo and in vivo), route of administration and special drug designation(s) awarded (if any). - A detailed review of the players engaged in the development of gene therapies, along with information on their year of establishment, company size, location of headquarters, regional landscape and key players engaged in this domain. - An elaborate discussion on the various types of viral and non-viral vectors, along with information on design, manufacturing requirements, advantages and limitations of currently available gene delivery vectors. - A discussion on the regulatory landscape related to gene therapies across various geographies, namely North America (the US and Canada), Europe and Asia-Pacific (Australia, China, Hong Kong, Japan and South Korea), providing details related to the various challenges associated with obtaining reimbursements for gene therapies. - Detailed profiles of marketed and late stage (phase II/III and above) gene therapies, including development timeline of the therapy, information on the current development status, mechanism of action, affiliated technology, strength of patent portfolio, dosage and manufacturing details, as well as details related to the developer company. - An elaborate discussion on the various commercialization strategies that can be adopted by drug developers across different stages of therapy development, including prior to drug launch, at / during drug launch and post-marketing. - A review of the various emerging technologies and therapy development platforms that are being used to design and manufacture gene therapies, featuring detailed profiles of technologies that were / are being used for the development of four or more products / product candidates. - An in-depth analysis of various patents that have been filed / granted related to gene therapies and gene editing therapies, since 2016. The analysis assesses several relevant parameters associated with the patents, including type of patent (granted patents, patent applications and others), publication year, regional applicability, CPC symbols, emerging focus areas, leading industry players (in terms of the number of patents filed / granted), and patent valuation. - A detailed analysis of the various mergers and acquisitions that have taken place within this domain, during the period 2015-2020, based on several relevant parameters, such as year of agreement, type of deal, geographical location of the companies involved, key value drivers, highest phase of development of the acquired company product and target therapeutic area. - An analysis of the investments made at various stages of development in companies that are focused in this area, between 2015-2020, including seed financing, venture capital financing, IPOs, secondary offerings, debt financing, grants and other equity offerings. - A detailed geographical clinical trial analysis of completed, ongoing and planned studies of numerous gene therapies, based on various relevant parameters, such as trial registration year, trial status, trial phase, target therapeutic area, geography, type of sponsor, prominent treatment sites and enrolled patient population. - An analysis of the various factors that are likely to influence the pricing of gene therapies, featuring different models / approaches that may be adopted by manufacturers to decide the prices of these therapies. - An analysis of the big biopharma players engaged in this domain, featuring a heat map based on parameters, such as number of gene therapies under development, funding information, partnership activity and strength of patent portfolio. - An informed estimate of the annual demand for gene therapies, taking into account the marketed gene-based therapies and clinical studies evaluating gene therapies; the analysis also takes into consideration various relevant parameters, such as target patient population, dosing frequency and dose strength. - A case study on the prevalent and emerging trends related to vector manufacturing, along with information on companies offering contract services for manufacturing vectors. The study also includes a detailed discussion on the manufacturing processes associated with various types of vectors. - A discussion on the various operating models adopted by gene therapy developers for supply chain management, highlighting the stakeholders involved, factors affecting the supply of therapeutic products and challenges encountered by developers across the different stages of the gene therapy supply chain.

One of the key objectives of the report was to estimate the existing market size and the future opportunity associated with gene therapies, for the next decade. Based on multiple parameters, such as target patient population, likely adoption rates and expected pricing, we have provided informed estimates on the evolution of the market for the period 2020-2030. The report also features the likely distribution of the current and forecasted opportunity across [A] therapeutic approach (gene augmentation, oncolytic viral therapy, immunotherapy and others), [B] type of gene therapy (ex vivo and in vivo), [C] type of vectors used (adeno associated virus, adenovirus, herpes simplex virus, lentivirus, plasmid DNA, retrovirus and others), [D] target therapeutic areas (autoimmune disorders, cardiovascular diseases, dermatological disorders, genetic disorders, hematological disorders, metabolic disorders, muscle-related diseases, oncological disorders, ophthalmic diseases and others), [E] route of administration (intraarticular, intracerebellar, intradermal, intramuscular, intratumoral, intravenous, intravesical, intravitreal, subretinal and others), and [F] key geographical regions (US, EU5 and rest of the world). In order to account for future uncertainties and to add robustness to our model, we have provided three market forecast scenarios, namely conservative, base and optimistic scenarios, representing different tracks of the industrys growth.

The opinions and insights presented in this study were influenced by discussions conducted with multiple stakeholders in this domain. The report features detailed transcripts of interviews held with the following individuals: - Adam Rogers (CEO, Hemera Biosciences) - Al Hawkins (CEO, Milo Biotechnology) - Buel Dan Rodgers (Founder & CEO, AAVogen) - Christopher Reinhard (CEO and Chairman, Gene Therapeutics (previously known as Cardium Therapeutics)) - Michael Triplett (CEO, Myonexus Therapeutics) - Robert Jan Lamers (CEO, Arthrogen) - Ryo Kubota (CEO, Chairman & President, Acucela) - Tom Wilton (CBO, LogicBio Therapeutics) - Jeffrey Hung (CCO, Vigene Biosciences) - Cedric Szpirer (Executive & Scientific Director, Delphi Genetics) - Marco Schmeer (Project Manager) & Tatjana Buchholz (Marketing Manager, PlasmidFactory) - Molly Cameron (Corporate Communications Manager, Orchard Therapeutics)

All actual figures have been sourced and analyzed from publicly available information forums and primary research discussions. Financial figures mentioned in this report are in USD, unless otherwise specified.

RESEARCH METHODOLOGY The data presented in this report has been gathered via secondary and primary research. For all our projects, we conduct interviews with experts in the area (academia, industry, medical practice and other associations) to solicit their opinions on emerging trends in the market. This is primarily useful for us to draw out our own opinion on how the market will evolve across different regions and technology segments. Where possible, the available data has been checked for accuracy from multiple sources of information.

The secondary sources of information include - Annual reports - Investor presentations - SEC filings - Industry databases - News releases from company websites - Government policy documents - Industry analysts views

While the focus has been on forecasting the market over the coming decade, the report also provides our independent view on various emerging trends in the industry. This opinion is solely based on our knowledge, research and understanding of the relevant market, gathered from various secondary and primary sources of information.

KEY QUESTIONS ANSWERED - Who are the leading industry players engaged in the development of gene therapies? - How many gene therapy candidates are present in the current development pipeline? Which key disease indications are targeted by such products? - Which types of vectors are most commonly used for effective delivery of gene therapies? - What are the key regulatory requirements for gene therapy approval, across various geographies? - Which commercialization strategies are most commonly adopted by gene therapy developers, across different stages of development? - What are the different pricing models and reimbursement strategies currently being adopted for gene therapies? - What are the various technology platforms that are either available in the market or are being designed for the development of gene therapies? - Who are the key CMOs / CDMOs engaged in supplying viral / plasmid vectors for gene therapy development? - What are the key value drivers of the merger and acquisition activity in the gene therapy industry? - Who are the key stakeholders that have actively made investments in the gene therapy domain? - Which are the most active trial sites (in terms of number of clinical studies being conducted) related to this domain? - How is the current and future market opportunity likely to be distributed across key market segments?

CHAPTER OUTLINES Chapter 2 provides an executive summary of the key insights captured in our research. It offers a high-level view on the current state of the market for gene therapies and its likely evolution in the short-mid term and long term.

Chapter 3 provides a general overview of gene therapies, including a discussion on their historical background. It further highlights the different types of gene therapies (namely somatic and germline therapies, and in vivo and ex vivo therapies), potential application areas of such products and route of administration of these therapeutic interventions. In addition, it provides information on the concept of gene editing, highlighting key historical milestones, applications and various techniques used for gene editing. The also chapter includes a discussion on the advantages and disadvantages associated with gene therapies. Further, it features a brief discussion on the ethical and social concerns related to gene therapies, while highlighting future constraints and challenges related to the manufacturing and commercial viability of such product candidates.

Chapter 4 provides a general introduction to the various types of viral and non-viral gene delivery vectors. It includes a detailed discussion on the design, manufacturing requirements, advantages and limitations of currently available vectors.

Chapter 5 features a detailed discussion on the regulatory landscape related to gene therapies across various geographies, such as the US, Canada, Europe, Australia, China, Hong Kong, Japan and South Korea. Further, it highlights an emerging concept of reimbursement which was recently adopted by multiple gene therapy developers, along with a discussion on several issues associated with reimbursement of gene therapies.

Chapter 6 includes information on over 800 gene therapies and gene editing therapies that are currently approved or are in different stages of development. It features a detailed analysis of pipeline molecules, based on several relevant parameters, such as key therapeutic areas (autoimmune disorders, cardiovascular diseases, dermatological disorders, genetic disorders, hematological disorders, immunological disorders, infectious diseases, inflammatory disorders, liver diseases, metabolic disorders, muscle-related diseases, nervous system disorders, oncological disorders, ophthalmic diseases and others), target disease indication(s), phase of development (marketed, clinical, preclinical and discovery), type of vector used, type of gene, type of gene therapy (ex vivo and in vivo), therapeutic approach (gene augmentation, oncolytic viral therapy and others), route of administration and special drug designation (if any). Further, we have presented a grid analysis of gene therapies based on phase of development, therapeutic area and therapeutic approach.

Chapter 7 provides a detailed review of the players engaged in the development of gene therapies, along with information on their year of establishment, company size, location of headquarters, regional landscape and key players engaged in this domain. Further, we have presented a logo landscape of product developers in North America, Europe and the Asia-Pacific region on the basis of company size.

Chapter 8 provides detailed profiles of marketed gene therapies. Each profile includes information about the innovator company, its product pipeline (focused on gene therapy only), development timeline of the therapy, its mechanism of action, target indication, current status of development, details related to manufacturing, dosage and sales, the companys patent portfolio and collaborations focused on its gene therapy product / technology.

Chapter 9 features an elaborate discussion on the various strategies that can be adopted by therapy developers across key commercialization stages, including prior to drug launch, during drug launch and post-launch. In addition, it presents an in-depth analysis of the key commercialization strategies that have been adopted by developers of gene therapies approved during the period 2015-2020.

Chapter 10 provides detailed profiles of drugs that are in advanced stages of clinical development (phase II/III and above). Each drug profile provides information on the current developmental status of the drug, its route of administration, developers, primary target indication, special drug designation received, target gene, dosage, mechanism of action, technology, patent portfolio, clinical trials and collaborations (if any).

Chapter 11 provides a list of technology platforms that are either available in the market or in the process of being designed for the development of gene therapies. In addition, it features brief profiles of some of the key technologies. Each profile features details on the various pipeline molecules that have been / are being developed using the technology, its advantages and the partnerships that have been established related to the technology platform. Further, the chapter includes detailed discussions on various novel and innovative technologies, along with brief information about key technology providers.

Chapter 12 highlights the potential target indications (segregated by therapeutic areas) that are currently the prime focus of companies developing gene therapies. These include genetic disorders, metabolic disorders, nervous system disorders, oncological disorders and ophthalmic diseases.

Chapter 13 provides an overview of the various patents that have been filed / granted in relation to gene therapy and gene editing technologies. It also features a detailed analysis, highlighting the prevalent trends related to type of patent, publication year, regional applicability, CPC symbols, emerging areas and leading industry players (in terms of number of patents filed). In addition, it features a competitive benchmarking analysis of the patent portfolios of leading industry players and patent valuation. For the purpose of this analysis, we have taken into consideration patents that have been filed / granted since 2016.

Chapter 14 features a detailed analysis of the various mergers and acquisitions that have taken place within this domain, during the period 2015-2020, based on several relevant parameters, such as year of agreement, type of deal, geographical location of the companies involved, key value drivers, highest phase of development of the acquired company product and target therapeutic area.

Chapter 15 presents details on various funding instances, investments and grants reported within the gene therapy domain. The chapter includes information on various types of investments (such as venture capital financing, debt financing, grants, capital raised from IPO and subsequent offerings) received by the companies between 2015 and 2020, highlighting the growing interest of the venture capital community and other strategic investors in this market.

Chapter 16 presents a detailed, geographical clinical trial analysis of completed, ongoing and planned studies focused on gene therapies, based on various relevant parameters, such as trial registration year, trial status, trial phase, target therapeutic area, geography, type of sponsor, prominent treatment sites and enrolled patient population.

Chapter 17 highlights our views on the various factors that may be taken into consideration while deciding the price of a gene therapy. It features discussions on different pricing models / approaches, based on the size of the target population, which a pharmaceutical company may choose to adopt in order to decide the price of its proprietary products.

Chapter 18 highlights top big biopharma players engaged in the field of gene therapy, featuring a heat map analysis based on several parameters, including therapeutic area, type of vector used, therapeutic approach and type of gene therapy.

Chapter 19 features an informed estimate of the annual demand for gene therapies, taking into account the marketed gene-based therapies and clinical studies evaluating gene therapies; the analysis also takes into consideration various relevant parameters, such as target patient population, dosing frequency and dose strength.

Chapter 20 presents an elaborate market forecast analysis, highlighting the future potential of the market till the year 2030. It also includes future sales projections of gene therapies that are either marketed or in advanced stages of clinical development (phase II/III and above). Sales potential and growth opportunity were estimated based on the target patient population, likely adoption rates, existing / future competition from other drug classes and the likely price of products. The chapter also presents a detailed market segmentation on the basis of [A] therapeutic approach (gene augmentation, oncolytic viral therapy, immunotherapy and others), [B] type of gene therapy (ex vivo and in vivo), [C] type of vector used (adeno associated virus, adenovirus, herpes simplex virus, lentivirus, plasmid DNA, retrovirus and others), [D] target therapeutic area (autoimmune disorders, cardiovascular diseases, dermatological disorders, genetic disorders, hematological disorders, metabolic disorders, muscle-related diseases, oncological disorders, ophthalmic diseases and others), [E] route of administration (intraarticular, intracerebellar, intradermal, intramuscular, intratumoral, intravenous, intravesical, intravitreal, subretinal and others), and [F] key geographical regions (US, EU5 and rest of the world).

Chapter 21 provides insights on viral vector manufacturing, highlighting the steps and processes related to manufacturing and bioprocessing of vectors. In addition, it features the challenges that exist in this domain. Further, the chapter provides details on various players that offer contract manufacturing services for viral and plasmid vectors.

Chapter 22 provides a glimpse of the gene therapy supply chain. It discusses the steps for implementing a robust model and provides information related to the global regulations for supply chain. Moreover, the chapter discusses the challenges associated with supply chain of gene therapies. In addition, it features the technological solutions that can be adopted for the management of gene therapy supply chain.

Chapter 23 summarizes the overall report, wherein we have mentioned all the key facts and figures described in the previous chapters. The chapter also highlights important evolutionary trends that were identified during the course of the study and are expected to influence the future of the gene therapy market.

Chapter 24 is a collection of interview transcripts of the discussions that were held with key stakeholders in this market. The chapter provides details of interviews held with Adam Rogers (CEO, Hemera Biosciences), Al Hawkins (CEO, Milo Biotechnology), Buel Dan Rodgers (Founder & CEO, AAVogen), Christopher Reinhard (CEO & Chairman, Gene Therapeutics (previously known as Cardium Therapeutics)), Michael Triplett (CEO, Myonexus Therapeutics), Robert Jan Lamers (CEO, Arthrogen), Ryo Kubota (CEO, Chairman & President, Acucela), Tom Wilton (CBO, LogicBio Therapeutics), Jeffrey Hung (CCO, Vigene Biosciences), Cedric Szpirer (Executive & Scientific Director, Delphi Genetics), Marco Schmeer (Project Manager) & Tatjana Buchholz (Marketing Manager, PlasmidFactory) and Molly Cameron (Corporate Communications Manager, Orchard Therapeutics). In addition, a brief profile of each company has been provided.

Chapter 25 is an appendix, which provides tabulated data and numbers for all the figures included in the report.

Chapter 26 is an appendix, which contains a list of companies and organizations mentioned in this report.Read the full report: https://www.reportlinker.com/p06020737/?utm_source=GNW

About ReportlinkerReportLinker is an award-winning market research solution. Reportlinker finds and organizes the latest industry data so you get all the market research you need - instantly, in one place.

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Originally posted here:
Gene Therapy Market by Therapeutic Approach, Type of Gene Therapy, Type of Vectors Used, Therapeutic Areas, Route of Administration, and Key...

First participant dosed in the RESOLUTESM trial, a Phase 1/2 dose-escalation study of SPK-3006

Enrollment of approximately 20 total study participants is ongoing

PHILADELPHIA, Feb. 01, 2021 (GLOBE NEWSWIRE) -- Spark Therapeutics, a member of the Roche Group (SIX: RO, ROG; OTCQX: RHHBY) and a fully integrated, commercial gene therapy company dedicated to challenging the inevitability of genetic disease, today announced the dosing of the first participant in the Phase 1/2 RESOLUTESM trial of SPK-3006, an investigational liver-directed adeno-associated viral (AAV) vector gene therapy for late-onset Pompe disease (LOPD), a rare, inherited lysosomal storage disorder.

Dosing the first participant in the Phase 1/2 RESOLUTE trial of investigational SPK-3006 for late-onset Pompe disease is an important milestone and first step to what we hope will ultimately allow us to bring an innovative gene therapy to these patients, said Gallia G. Levy, M.D., Ph.D., chief medical officer of Spark Therapeutics. We are deeply appreciative of the ongoing collaboration of the Pompe disease community as we continue to enroll participants in this Phase 1/2 study.

The RESOLUTE trial is an open-label Phase 1/2, dose-escalation gene transfer study designed to evaluate the safety, tolerability and efficacy of a single intravenous infusion of investigational SPK-3006, an AAV vector-based gene therapy, developed in collaboration with Genethon, in adults with clinically moderate LOPD currently receiving enzyme replacement therapy. The study is expected to enroll approximately 20 participants receiving the investigational gene therapy in sequential, dose-level cohorts. Additional details are available on ClinicalTrials.gov (NCT04093349).

We are honored to have the first participant dosed in this clinical trial, which we hope will lead us to introduce a novel therapeutic option for patients living with late-onset Pompe disease, said Principal Investigator Tahseen Mozaffar, M.D., University of California Irvine Health.

The International Pompe Association has been proud to collaborate with Spark Therapeutics to enhance the Pompe disease communitys understanding of gene therapy research, said Tiffany House, International Pompe Association Board Chairman. We look forward to the progress in the Phase 1/2 RESOLUTE trial, as well as the ongoing work aimed at developing gene therapies that have the potential to help individuals living with genetic diseases.

Pompe disease is a rare, inherited lysosomal storage disorder. It is a progressive, often life-limiting disease caused by the buildup of a complex sugar, glycogen, in the bodys cells. Mutations in the gene encoding acid alpha-glucosidase (GAA) result in deficiencies of the GAA enzyme and limit the breakdown of glycogen. For patients living with LOPD, the respiratory system, locomotion and maintenance of gait are the most critically impacted. These symptoms commonly result in patients becoming wheelchair bound and requiring respiratory support, which may result in reduced life-expectancy.

About SPK-3006 for Pompe diseaseSPK-3006is an investigational liver-directed AAV gene therapy for the potential treatment of late-onset Pompe disease (LOPD).SPK-3006has been engineered to produce a modified enzyme (secretable GAA) that is produced by the liver, which may result in sustained GAA plasma levels and could potentially provide greater uptake in muscle tissue. The transgene integrates technologies designed at and licensed from Genethon, where the in-vivo proof of concept in pre-clinical models was demonstrated. Spark Therapeutics retains global commercialization rights toSPK-3006.

About Spark Therapeutics AtSpark Therapeutics, a fully integrated, commercial company committed to discovering, developing and delivering gene therapies, we challengethe inevitability of genetic diseases,includingblindness, hemophilia, lysosomal storage disorders and neurodegenerative diseases.We currently have four programs in clinical trials.At Spark, a member of the Roche Group, we see the path to a world where no life is limited by genetic disease. For more information, visit http://www.sparktx.com, and follow us on Twitter and LinkedIn.

Media Contact:Kevin Giordanocommunications@sparktx.com(215) 294-9942

See the article here:
Spark Therapeutics Announces First Participant Dosed in Phase 1/2 Study of Investigational Gene Therapy for Late-Onset Pompe Disease - BioSpace

- First potentially disease-modifying gene therapy for GM2 gangliosidosis to enter clinical studies

- Expect to continue patient identification, screening, and enrollment in Stage 1 of the study throughout 2021

NEW YORK and RESEARCH TRIANGLE PARK, N.C., Feb. 03, 2021 (GLOBE NEWSWIRE) -- Sio Gene Therapies Inc. (NASDAQ: SIOX), a clinical-stage company focused on developing gene therapies to radically transform the lives of patients with neurodegenerative diseases, today announced that the first patient with infantile Tay-Sachs disease has been dosed in a Phase 1/2 trial evaluating AXO-AAV-GM2,an investigational gene therapy for the treatment of GM2 gangliosidosis, also known as Tay-Sachs or Sandhoff disease.

We are proud to bring the first potentially disease-modifying treatment for GM2 gangliosidosis to the clinic, which is a milestone for Sio, for patients, and for the field of gene therapy, said Gavin Corcoran, M.D., Chief R&D Officer of Sio. By restoring lysosomal enzyme activity where it is essential, AXO-AAV-GM2 has the potential to change the course of this disease and help affected children attain and retain important neuro-developmental milestones. The prior expanded access study of AXO-AAV-GM2 provided important proof-of-concept data and we look forward to the results of the first stage of our study as we strive to develop a treatment for children suffering from this rapidly progressive and fatal disease.

Florian Eichler, M.D., Director of the Leukodystrophy Service of the Center for Rare Neurological Diseases at Massachusetts General Hospital, and principal investigator, added, To date, the current GM2 treatment landscape is limited to supportive care, underscoring the significant need for new treatment options to address this devastating pediatric neurodegenerative disease. AXO-AAV-GM2 has significant potential to address the clinical manifestations of both Tay Sachs and Sandhoff diseases, and as a result, the dosing of this patient represents a major step forward for this therapy. We look forward to evaluating the results of this study and advancing the first potentially disease-modifying treatment option for patients with GM2.

The Phase 1/2 study (NCT04669535) is an open-label, two-stage clinical trial designed to evaluate safety and dose-escalation (Stage 1) and safety and efficacy (Stage 2) of surgical delivery of AXO-AAV-GM2 directly to the brain and spinal cord of pediatric participants with both infantile and juvenile GM2 gangliosidosis. AXO-AAV-GM2 has been granted Orphan Drug and Rare Pediatric Disease Designation by the FDA and is the first investigational gene therapy to enter clinical trials for GM2 gangliosidosis. In 2019, clinical evidence from two patients under an expanded access IND found that treatment with AXO-AAV-GM2 was generally well-tolerated and associated with improved bioactivity outcomes.

The families of children with Sandhoff and Tay-Sachs diseases show incredible bravery in choosing to participate in investigational studies of novel therapeutics like AXO-AAV-GM2. We share their hope that this treatment can halt or reverse the otherwise inexorable course of these tragic diseases, said Terence R. Flotte, MD, Professor of Pediatrics and Dean at the University of Massachusetts Medical School and principal investigator of the trial.

GM2 gangliosidosis is a set of rare, monogenic neurodegenerative lysosomal storage disorders caused by mutations in the genes that encode the enzyme -Hexosaminidase A. It can be categorized into two distinct diseases, Tay-Sachs disease, which results from a mutation in the gene encoding the alpha subunit of the -Hexosaminidase A enzyme (HEXA), and Sandhoff disease, which results from a mutation in the gene encoding the beta subunit of the -Hexosaminidase A enzyme (HEXB). Children affected by GM2 gangliosidosis suffer from a progressively debilitating disease course and reduced life expectancy.

Sue Kahn, Executive Director of National Tay-Sachs & Allied Diseases Association(NTSAD), added, This news represents the culmination of many years of work to advance this research and immense support from the GM2 community, and it underscores the dire need for new treatment options capable of providing meaningful benefits to patients and families. We are extremely excited by the progress Sio has made and the hope it brings to our community.

Sio aims to advance the program through strategic partnerships with leading research organizations. The Company has a partnership with Viralgen, an AskBio subsidiary, to support AAV-based vector manufacturing of clinical trial material for the registrational study. Additionally, through an existing genetic testing collaboration with Invitae, ongoing partnership with GM2 gangliosidosis patient groups, and collaboration with leading academic researchers at the University of Massachusetts Medical School and Massachusetts General Hospital, Sio has begun patient identification and screening activities for the ongoing clinical study.

About AXO-AAV-GM2

AXO-AAV-GM2 is an investigational gene therapy for GM2 gangliosidosis (also known as Tay-Sachs and Sandhoff diseases), a set of rare and fatal pediatric neurodegenerative genetic disorders caused by defects in the HEXA (leading to Tay-Sachs disease) or HEXB (leading to Sandhoff disease) genes that encode the two subunits of the -hexosaminidase A (HexA) enzyme. These genetic defects lead to progressive neurodegeneration and shortened life expectancy. AXO-AAV-GM2 aims to restore HexA function by introducing a functional copy of the HEXA and HEXB genes via delivery of two co-administered AAVrh8 vectors.

About Sio Gene TherapiesSio Gene Therapies combines cutting-edge science with bold imagination to develop genetic medicines that aim to radically improve the lives of patients. Our current pipeline of clinical-stage candidates includes the first potentially curative AAV-based gene therapies for GM1 gangliosidosis and Tay-Sachs/Sandhoff diseases, which are rare and uniformly fatal pediatric conditions caused by single gene deficiencies. We are also expanding the reach of gene therapy to highly prevalent conditions such as Parkinsons disease, which affects millions of patients globally. Led by an experienced team of gene therapy development experts, and supported by collaborations with premier academic, industry, and patient advocacy organizations, Sio is focused on accelerating its candidates through clinical trials to liberate patients with debilitating diseases through the transformational power of gene therapies. For more information, visit http://www.siogtx.com.

In 2018, Sio licensed exclusive worldwide rights from the University of Massachusetts Medical School for the development and commercialization of gene therapy programs for GM1 gangliosidosis and GM2 gangliosidosis, including Tay-Sachs and Sandhoff diseases.

Forward-Looking Statements

This press release contains forward-looking statements for the purposes of the safe harbor provisions under The Private Securities Litigation Reform Act of 1995 and other federal securities laws. The use of words such as expect potentially, and potential, and other similar expressions are intended to identify forward-looking statements. For example, all statements Sio makes regarding costs associated with its operating activities are forward-looking. All forward-looking statements are based on estimates and assumptions by Sios management that, although Sio believes to be reasonable, are inherently uncertain. All forward-looking statements are subject to risks and uncertainties that may cause actual results to differ materially from those that Sio expected. Such risks and uncertainties include, among others, the impact of the Covid-19 pandemic on our operations, the initiation and conduct of preclinical studies and clinical trials; the availability of data from clinical trials; the development of a suspension-based manufacturing process for AXO-Lenti-PD; the scaling up of manufacturing, the expectations for regulatory submissions and approvals; the continued development of our gene therapy product candidates and platforms; Sios scientific approach and general development progress; and the availability or commercial potential of Sios product candidates. These statements are also subject to a number of material risks and uncertainties that are described in Sios most recent Quarterly Report on Form 10-Q filed with the Securities and Exchange Commission on November 13, 2020, as updated by its subsequent filings with the Securities and Exchange Commission. Any forward-looking statement speaks only as of the date on which it was made. Sio undertakes no obligation to publicly update or revise any forward-looking statement, whether as a result of new information, future events or otherwise.

Contacts:

Media

Josephine Belluardo, Ph.D.LifeSci Communications(646) 751-4361jo@lifescicomms.cominfo@siogtx.com

Investors and Analysts

Parag V. Meswani, Pharm.D.Sio Gene Therapies Inc.Chief Commercial Officerinvestors@siogtx.com

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Sio Gene Therapies Announces First Patient Dosed in Clinical Trial of AXO-AAV-GM2 in Patients with Tay-Sachs and Sandhoff Disease (GM2 Gangliosidosis)...

Vancouver, British Columbia, Feb. 04, 2021 (GLOBE NEWSWIRE) -- The Global Regenerative Medicine Market is predicted to attain a market valuation of USD 6.49 billion by 2027, growing at a CAGR of 9.3% throughout the estimated period, according to a recent analysis by Emergen Research. Targeted therapy of specific disease indication and chronic illnesses are anticipated to alter the dynamics of the healthcare field. The escalating prevalence of chronic health conditions and increasing patient pool of geriatric populace coupled with neurodegenerative disorders, cancers, orthopedic, and other age-related conditions are further bolstering the industrys expansion.

The numerous applications and subsequent advancements in tissue engineering, gene therapy, nanotechnology, and stem cells research are foreseen to boost the scope of regenerative medicine. 3D printing is playing a pivotal role in stem cells research as it allows for the easy restoration of structural and functional properties.

North America is predicted to occupy a significant share of the market in the projected timeframe and the growth can be attributed to the increasing number of academic institutions and universities extensively exploring regenerative medicine approaches based on stem cells.

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For the purpose of this report, Emergen Research has segregated the Global Regenerative Medicine Market on the basis of product, therapeutic category, application, and region:

Product Outlook (Volume, Million Tons; Revenue, USD Billion; 2017-2027)

Therapeutic Category Outlook (Volume, Million Tons; Revenue, USD Billion; 2017-2027)

Application Outlook (Volume, Million Tons; Revenue, USD Billion; 2017-2027)

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Regional Outlook (Volume, Million Tons; Revenue, USD Billion; 2017-2027)

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Regenerative Medicine Market to be Valued at USD 6.49 Billion by 2027 | The Escalating Burden of Chronic Diseases and Genetic Aberrations will be the...

January was a busy month for the US Food and Drug Administrations precision medicine efforts, as the agency produced guidance on ASO drugs for patients with debilitating or life-threatening genetic disorders and guidance on manufacturing considerations for certain cellular and gene therapy products during the COVID-19 pandemic.

The agency first issued a draft guidance to facilitate the development of individualized antisense oligonucleotide (ASO) drugs for patients with severely debilitating or life-threatening genetic disorders (ASO Guidance). The Food and Drug Administration (FDA) also issued a guidance, with immediate effect, on manufacturing considerations for licensed and investigational cellular and gene therapy products during the COVID-19 public health emergency (Manufacturing Guidance). Sponsors investigating or marketing these products should pay special attention to the discussion in these documents, as FDA outlines its approach to COVID-19 and development considerations with respect to these personalized therapies.

The Manufacturing Guidance supplements FDAs June 2020 guidance on Good Manufacturing Practice Considerations for Responding to COVID-19 Infection in Employees in Drug and Biological Products Manufacturing. However, because cell and gene therapy (CGT) manufacturers may face special challenges, FDA recommends that CGT manufacturers perform risk assessments to identify, evaluate, and mitigate factors that may allow for the transmission of SARS-CoV-2 through CGT products. Any plans should take into account FDAs view that allogeneic products may be associated with a higher risk of infection compared to autologous products.

FDA specifically recommends the following:

As always, any adopted risk assessment and mitigation strategies must be documented and approved by the manufacturers quality unit, should include scientific justification and literature references, and should be submitted to FDA.

Turning away from the current COVID-19 crisis, FDA indicated that it is also looking ahead to the continued advancement of personalized therapies, issuing the ASO Guidance to assist sponsor investigators in the development of individualized ASO products for severely debilitating or life-threatening genetic diseases that are tailored to a patients specific genetic variant. As noted by FDA, the ASO Guidance is targeted to academic investigators, who may be less familiar with FDAs requirements and less experienced in interacting with FDA.

While the specific impetus for this guidance is unclear, assumedly FDA is receiving more inquiries regarding individualized ASO drugs from investigators, patients, or those acting on their behalf. Regardless of the reason, healthcare institutions where ASO products are used should familiarize themselves with FDAs requirements and processes to ensure that any use of an investigational ASO product accords with FDAs regulations. It will also be important that manufacturers supporting the use of ASO products or that later intend to work with ASO product investigators ensure that programs comply with FDAs regulations via contractual agreements and, as appropriate, due diligence.

For these programs, FDA recommends the following:

The ASO Guidance is likely a first step in the development of individualized therapies. As stated by FDA, the agency is optimistic that development of [ASO] individualized drug products may spur gene sequencing that leads to the development of additional individualized drug products. Accordingly, through the ASO Guidance, FDA aims to determine the most effective and efficient way to bring personalized drugs to patients, while ensuring the right risk-benefit balance.

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FDA Issues More Guidance on Gene and Cell Therapy Products - JD Supra

NEW YORK and RESEARCH TRIANGLE PARK, N.C., Feb. 04, 2021 (GLOBE NEWSWIRE) -- Sio Gene Therapies Inc. (NASDAQ: SIOX), a clinical-stage company focused on developing gene therapies to radically improve the lives of patients with neurodegenerative diseases, announced today that it has received $11.6 million from the closing of the sale of Arvelle Therapeutics to Angelini Pharma. Per the terms of the sale, additional payments to Sio Gene Therapies Inc. are expected over time, including a payment of approximately $4.8 million by mid-2021 upon marketing approval of cenobamate by the European Medicines Agency (EMA).

About Sio Gene Therapies

Sio Gene Therapies combines cutting-edge science with bold imagination to develop genetic medicines that aim to radically improve the lives of patients. Our current pipeline of clinical-stage candidates includes the first potentially curative AAV-based gene therapies for GM1 gangliosidosis and Tay-Sachs/Sandhoff diseases, which are rare and uniformly fatal pediatric conditions caused by single gene deficiencies. We are also expanding the reach of gene therapy to highly prevalent conditions such as Parkinsons disease, which affects millions of patients globally. Led by an experienced team of gene therapy development experts, and supported by collaborations with premier academic, industry and patient advocacy organizations, Sio is focused on accelerating its candidates through clinical trials to liberate patients with debilitating diseases through the transformational power of gene therapies. For more information, visit http://www.siogtx.com.

Forward-Looking Statements

This press release contains forward-looking statements for the purposes of the safe harbor provisions under The Private Securities Litigation Reform Act of 1995 and other federal securities laws. The use of words such as expect and other similar expressions are intended to identify forward-looking statements. For example, all statements Sio makes regarding amounts to be received and income tax liabilities associated with the sale of its stake in Arvelle Therapeutics, as well as the duration of its ability to support its development programs, are forward-looking. All forward-looking statements are based on estimates and assumptions by Sios management that, although Sio believes to be reasonable, are inherently uncertain. All forward-looking statements are subject to risks and uncertainties that may cause actual results to differ materially from those that Sio expected. Such risks and uncertainties include, among others, the impact of the Covid-19 pandemic on our operations, the initiation and conduct of preclinical studies and clinical trials; the availability of data from clinical trials; the development of a suspension-based manufacturing process for AXO-Lenti-PD; the scaling up of manufacturing, the expectations for regulatory submissions and approvals; the continued development of our gene therapy product candidates and platforms; Sios scientific approach and general development progress; and the availability or commercial potential of Sios product candidates. These statements are also subject to a number of material risks and uncertainties that are described in Sios most recent Quarterly Report on Form 10-Q filed with the Securities and Exchange Commission on November 13, 2020, as updated by its subsequent filings with the Securities and Exchange Commission. Any forward-looking statement speaks only as of the date on which it was made. Sio undertakes no obligation to publicly update or revise any forward-looking statement, whether as a result of new information, future events or otherwise.

Contacts:

Media

Josephine Belluardo, Ph.D.LifeSci Communications(646) 751-4361jo@lifescicomms.cominfo@siogtx.com

Investors and Analysts

David NassifSio Gene Therapies Inc.Chief Financial Officer and General Counsel(646) 677-6770investors@siogtx.com

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Sio Gene Therapies Announces Receipt of $11.6 Million from Closing of the Sale of Arvelle Therapeutics - BioSpace

REDWOOD CITY, Calif., Feb. 02, 2021 (GLOBE NEWSWIRE) -- Adverum Biotechnologies, Inc.(Nasdaq: ADVM), a clinical-stage gene therapy company targeting unmet medical needs in ocular and rare diseases, today announced the publication of preclinical data on ADVM-022 intravitreal (IVT) gene therapy in Translational Vision Science & Technology (TVST), an official journal of the Association for Research in Vision and Ophthalmology (ARVO). ADVM-022 is in clinical trials for wet AMD and DME, and this preclinical study in NHPs is the longest safety and expression study to date, with measurements out 30 months following a single IVT injection.

There is a growing body of both clinical and preclinical data demonstrating durable efficacy and favorable safety profile following a single IVT injection of ADVM-022, said Laurent Fischer, M.D., chief executive officer at Adverum Biotechnologies. In this preclinical study, we saw long-term, sustained aflibercept expression out to 30 months following ADVM-022. The levels of aflibercept were sustained at therapeutic levels, with no measurable adverse effects on normal retinal structure and function. We are excited to work on developing ADVM-022 as a potential one and done IVT injection therapy that may dramatically reduce the treatment burden for patients living with wet AMD and DME.

Szilrd Kiss, M.D., academic retina specialist, added, Currently, patients with wet AMD are treated with frequent anti-VEGF intravitreal injections to maintain their vision. One of the highest priorities in research today is to develop therapies that extend the duration of efficacy following treatment, enabling patients to preserve sight for months or years following treatment. The preclinical data on ADVM-022 demonstrate long-term safety and aflibercept expression following a single intravitreal injection of this novel IVT injection gene therapy. We are excited to continue to assess ADVM-022 as it demonstrates the potential to improve real-world visual outcomes over intermittent anti-VEGF injections for patients living with wet AMD.

The publication, titled Long-Term Safety Evaluation of Continuous Intraocular Delivery of Aflibercept by the Intravitreal Gene Therapy Candidate ADVM-022 in Nonhuman Primates, reported the following:

The full online publication can be accessed from the TVST website.

About ADVM-022 Gene TherapyADVM-022 utilizes a propriety vector capsid, AAV.7m8, carrying an aflibercept coding sequence under the control of a proprietary expression cassette. ADVM-022 is administered as a one-time intravitreal injection (IVT), designed to deliver long-term efficacy and reduce the burden of frequent anti-VEGF injections, optimize patient compliance and improve vision outcomes for patients with wet age-related macular degeneration (wet AMD) and diabetic macular edema (DME).

In recognition of the need for new treatment options for wet AMD, the U.S. Food and Drug Administration granted Fast Track designation for ADVM-022 for the treatment of wet AMD.

Adverum is currently evaluating ADVM-022 in the OPTIC Phase 1 clinical trial in patients with wet AMD and the INFINITY Phase 2 trial in patients with DME at 2 x 10^11 vg/eye and 6 x 10^11 vg/eye doses. The Company plans to begin a pivotal trial in mid-2021 for ADVM-022 in wet AMD.

About Adverum BiotechnologiesAdverum Biotechnologies (Nasdaq: ADVM) is a clinical-stage gene therapy company targeting unmet medical needs in serious ocular and rare diseases. Adverum is advancing the clinical development of its novel gene therapy candidate, ADVM-022, as a one-time, intravitreal injection for the treatment of patients with wet age-related macular degeneration and diabetic macular edema. For more information, please visit http://www.adverum.com.

Forward-looking StatementsStatements contained in this press release regarding the events or results that may occur in the future are forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Such statements include but are not limited to statements regarding: the potential for ADVM-022 in treating patients with wet AMD and DME; the potential efficacy and safety of ADVM-022 in wet AMD and DME; Adverums expectations as to its plans to advance ADVM-022 in wet AMD by initiating a pivotal trial mid-2021. Actual results could differ materially from those anticipated in such forward-looking statements as a result of various risks and uncertainties, which include risks inherent to, without limitation: Adverums novel technology, which makes it difficult to predict the time and cost of product candidate development and obtaining regulatory approval; the results of early clinical trials not always being predictive of future results; the potential for future complications or side effects in connection with use of ADVM-022. Risks and uncertainties facing Adverum are described more fully in Adverums Form 10-Q filed with theSEConNovember 5, 2020under the heading Risk Factors. All forward-looking statements contained in this press release speak only as of the date on which they were made. Adverum undertakes no obligation to update such statements to reflect events that occur or circumstances that exist after the date on which they were made.

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Adverum Biotechnologies Announces Publication of Preclinical Long-Term Safety Data on ADVM-022 IVT Gene Therapy - BioSpace

NEW YORK, Feb. 3, 2021 /PRNewswire/ -- Aruvant Sciences, a private company focused on developing gene therapies for rare diseases,today announced that the European Medicines Agency (EMA) granted Priority Medicines (PRIME) designation to ARU-1801, a one-time investigational gene therapy for sickle cell disease (SCD).

"PRIME designation from EMAhighlightsthe importance of ARU-1801, administeredwith only reduced intensity conditioning,for the treatment ofindividuals with severe sickle cell disease,"said Will Chou, M.D., Aruvant chief executiveofficer."With PRIME,we will be able to work closely with EMAon the development of ARU-1801, with the goal of rapidlybringingthispotential cure toSCD patients in Europe."

PRIME was created by the European Medicines Agency (EMA) to enhance support for the development of innovative medicinesthat target an unmet medical needand demonstratethe potential to achieve relevant clinical outcomes on morbidity, mortality or underlying disease progression. The PRIME designation offersenhanced early interaction with companiesdeveloping promising medicines, to optimize development plans and speed up evaluation. PRIME focuses on medicines that may offer a major therapeutic advantage over existing treatments, or that benefit patients without treatment options.

ARU-1801 was designated PRIME status based on clinical data from the MOMENTUMstudy, an ongoing Phase 1/2 trial of ARU-1801 in patients with severe sickle cell disease, that demonstrate meaningful,durable reductions in disease burden.

About ARU-1801ARU-1801 is designed to address the limitations of current curative treatment options, such as low donor availability and the risk of graft-versus-host disease (GvHD) seen with allogeneic stem cell transplants. Unlike investigational gene therapies and gene editing approaches which require fully myeloablative conditioning, the unique characteristics of ARU-1801 allow it to be given with reduced intensity conditioning ("RIC"). Compared to myeloablative approaches, the lower dose chemotherapy regimen underlying RIC has the potential to reduce not only hospital length of stay, but also the risk of short- and long-term adverse events such as infection and infertility. Preliminary clinical data from the MOMENTUMstudy, an ongoing Phase 1/2 trial of ARU-1801 in patients with severe sickle cell disease, demonstrate continuing durable reductions in disease burden.

The MOMENTUM StudyAruvant is conducting the MOMENTUM study, which is evaluating ARU-1801, a one-time potentially curative investigational gene therapy for patients with SCD. This Phase 1/2 study is currently enrolling participants, and information may be found at http://www.momentumtrials.comwhich includes a patient brochure, an eligibility questionnaireand information for healthcare providers.

About Aruvant SciencesAruvant Sciences, part of the Roivant family of companies, is a clinical-stage biopharmaceutical company focused on developing and commercializing gene therapies for the treatment of rare diseases. The company has a talentedteamwith extensive experience in the development, manufacturing and commercialization of gene therapy products. Aruvant has an activeresearchprogram with a lead product candidate, ARU-1801, in development for individuals suffering fromsickle cell disease(SCD). ARU-1801, an investigational lentiviral gene therapy, is being studied in aPhase 1/2 clinical trial,the MOMENTUM study, as a one-time potentially curative treatment for SCD. Preliminary clinical data demonstrate engraftment of ARU-1801 and amelioration of SCD is possible with one dose of reduced intensity chemotherapy. For more information on the clinical study, please visit http://www.momentumtrials.comand for more on the company, please visitwww.aruvant.com. Follow Aruvant on Facebook, Twitter @AruvantSciencesand on Instagram @Aruvant_Sciences.

About RoivantRoivant's mission is to improve the delivery of healthcare to patients by treating every inefficiency as an opportunity. Roivant develops transformative medicines faster by building technologies and developing talent in creative ways, leveraging the Roivant platform to launch Vants nimble and focused biopharmaceutical and health technology companies. For more information, please visit http://www.roivant.com.

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Aruvant Announces the European Medicines Agency (EMA) Granted Priority Medicines (PRIME) Designation to ARU-1801 for the Treatment of Sickle Cell...

Nottingham, UK 2nd February 2021 Life Science Newswire Albumedix Ltd. (Albumedix), an enabler of advanced therapies and the world leader in recombinant human albumin (rAlb), announced today that they have entered into a collaboration agreement with the Cell and Gene Therapy Catapult (CGT Catapult) to investigate the use of Albumedix proprietary albumin-based solutions for advanced therapy applications, including viral vectors manufacturing.

This agreement reflects the continued efforts of Albumedix to engage with the industry and expand upon its knowledge in the field, and the CGT Catapults mission to drive the growth of the UK cell and gene therapy industry by helping cell and gene therapy organisations across the world translate early-stage research into commercially viable and investable therapies.

Albumedix Chief Executive Officer; Jonas Skjdt Mller commented on the collaboration:

With a mission to empower excellence in advanced therapies, we are committed to continuously playing an integral part in enabling our customers to advance in a fast-moving industry. For us to do so, we continuously look at other industry leaders to establish collaborations. Continuing to learn from each other allows innovation in the market to advance, and Albumedix to support our customers with in-depth knowledge of how rAlb can uniquely benefit their therapies. Cell and Gene Therapy Catapult is the ideal partner; located in our own backyard here in the UK and with incredible skills, knowledge and drive to advance the cell and gene therapy industry we are excited about this collaboration.

Matthew Durdy, Chief Executive Officer at Cell and Gene Therapy Catapult commented:

The opportunity to collaborate with a leading company like Albumedix Ltd in order to assess and drive the potential of their latest technology is something that we embrace. The prospect of improving manufacturing of viral vectors such as Adeno-associated virus (AAV) through this exciting technology is something which could significantly benefit and advance the wider cell and gene therapy field.

Activities under this agreement will be carried out both at the CGT Catapult facility in Braintree and at Albumedix new R&D center, with state-of-the-art laboratories specifically designed for the process optimization, characterization and formulation development of advanced therapies.

Get in touch with Albumedix today by emailing communications@albumedix.com to learn more about their Recombumin range of world leading recombinant human albumin products. Reach out to Cell and Gene Therapy Catapult by emailing communications@ct.catapult.org.uk to learn more about how they can help your organisation to translate early stage research into commercially viable and investable therapies.

About Albumedix Dedicated to Better Health

Albumedix is a science-driven, life-science company focused on enabling the creation of superior biopharmaceuticals utilizing our recombinant human albumin products. We believe in empowering excellence to enable advanced therapies and facilitate otherwise unstable drug candidates reach patients worldwide. We are proud to be recognized as the world leader in recombinant human albumin with products and technologies used in clinical and marketed drugs by pharmaceutical and medical device companies worldwide. Headquartered in Nottingham, England with more than 100 people all committed to improving patient quality of life. We are just as passionate about albumin and albumin-enabled therapies today as we were when we started more than 35 years ago. For more information, please reach out to Albumedix at communications@albumedix.com or visit http://www.albumedix.com

About Cell and Gene Therapy Catapult

The Cell and Gene Therapy Catapult was established as an independent centre of excellence to advance the growth of the UK cell and gene therapy industry, by bridging the gap between scientific research and full-scale commercialisation. With more than 330 employees focusing on cell and gene therapy technologies, it works with partners in academia and industry to ensure these life-changing therapies can be developed for use in health services throughout the world. It offers leading-edge capability, technology and innovation to enable companies to take products into clinical trials and provide clinical, process development, manufacturing, regulatory, health economics and market access expertise. Its aim is to make the UK the most compelling and logical choice for UK and international partners to develop and commercialise these advanced therapies. The Cell and Gene Therapy Catapult works with Innovate UK. For more information please visit ct.catapult.org.uk or visit http://www.gov.uk/innovate-uk.

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Albumedix enters into collaboration agreement with Cell and Gene Therapy Catapult - PharmiWeb.com

London, Feb. 02, 2021 (GLOBE NEWSWIRE) -- Roots Analysis has announced the addition of Gene Therapy Market (4th Edition), 2020-2030 report to its list of offerings.

Success of approved gene therapies has resulted in a surge in interest of biopharmaceutical developers in this rapidly evolving domain. Presently, the ability of gene therapies to treat diverse disease indications is considered among the most prominent drivers of this market. In addition, promising clinical results of pipeline candidates are anticipated to draw in more investments to support product development initiatives.

To order this 720+ page report, which features 220+ figures and 375+ tables, please visit this link

Key Market Insights

Around 800 gene therapies are currently being developed across different stages Apart from 10 approved products, most of the aforementioned therapies (65%) are in the early stages of development (discovery / preclinical), while the rest are being evaluated in clinical trials. It is worth mentioning that more than 40% of clinical stage candidates are intended for the treatment of oncological disorders.

Over 65% of innovator companies focused on gene therapy development, are based in North AmericaInterestingly, more than 75 players based in the same region, are start-ups, while over 35 are mid-sized players, and 10 are large and very large firms. Since the majority of gene therapy developers are headquartered in the US, it is considered a key R&D hub for such advanced therapy medicinal products.

There are 400+ registered gene therapy focused clinical trials, worldwideClinical research activity, in terms of number of trials registered, is reported to have increased at a CAGR of 12% during the period 2015-2020. Of the total number of trials, close to 25% have already been completed, and 35% claim to be actively recruiting.

USD 25.4 billion has been invested by both private and public investors, since 2015So far, a significant proportion of the capital raised has been through secondary offerings (USD 12.9 billion). On the other hand, around USD 5 billion was invested by venture capital investors, representing 20% of the total amount.

Close to 20,000 patents have been filed / published related to gene therapies, since 2016Around 30% of the total number of applications were related to gene editing-based therapies, while the remaining were associated with gene therapies. Further, majority of the patent assignees were industry players, however, the contribution of non-industry players in the overall patent filing activity has increased considerably (CAGR of 16%), over the past few years.

There have been several mergers and acquisitions in this market during the period 2015-2019 In fact, M&A activity is reported to have increased at a CAGR of more than 40%. Key drivers of the acquisitions mentioned in the report include, therapeutic area expansion, access to a novel technology / platform, drug class consolidation and drug class expansion.

North America and Europe are anticipated to capture over 90% of the market share, in terms of sales revenues, in 2030In vivo gene therapies currently represent a significant share of the market, and this trend is unlikely to change in the foreseen future, as several such candidates are being evaluated in late stages. In addition, more than 130,000+ patients are projected to use gene therapies in 2030 and the demand for gene therapies is expected to grow at an annualized rate of 29% and 31% during the periods 2020-2025 and 2025-2030, respectively.

To request a sample copy / brochure of this report, please visit this link

Key Questions Answered

The USD 14.6 billion (by 2030) financial opportunity within the gene therapy market has been analyzed across the following segments:

The report features inputs from eminent industry stakeholders, according to whom, gene therapies exhibit the potential to become a promising alternative for the treatment of genetic disorders. The report includes detailed transcripts of discussions held with the following experts:

The research includes brief profiles of key players (listed below) engaged in the development of gene therapies; each profile features an overview of the therapy, current development status, clinical trials and its results (if available), target indication, route of administration, and recent developments (if available).

For additional details, please visit https://www.rootsanalysis.com/reports/view_document/gene-therapies-market/268.html or email sales@rootsanalysis.com

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The gene therapy market is projected to be worth USD 14.6 billion in 2030, growing at a CAGR of 30%, over the next decade, claims Roots Analysis -...

Global Retinal Gene Therapy Market: Overview

The retinal gene therapy market is estimated to expand at an exponential growth rate. For the use of gene therapy, retina is considered a highly desirable target as it an irreplaceable part of a body. The global retinal gene therapy market is likely to be influenced by the promise its holds for the treatment of various forms of inherited and non-inherited blindness. Furthermore, this therapy can also be used in the treatment of rare genetic retinal diseases, such as Leber's congenital amaurosis, which is likely to augur well for the development of the global retinal gene therapy market during the forecast period, from 2020 to 2030. It is expected that the global retinal gene therapy market is anticipated to witness the entry of new players, with the presence of promising candidates in the phases of drug approval process.

Read Report Overview - https://www.transparencymarketresearch.com/retinal-gene-therapy-market.html

Type, application, and region are the three important parameters based on which the global retinal gene therapy market has been classified. Such detailed analysis of the market comes with the sole purpose to provide stakeholders with a detailed and clear analysis of the global retinal gene therapy market.

Read Report Overview - https://www.transparencymarketresearch.com/retinal-gene-therapy-market.html

Global Retinal Gene Therapy Market: Notable Developments

One of the important market developments that give a quick view of the dynamics pertaining to the global retinal gene therapy market is mentioned as below:

There is only one player in this global retinal gene therapy market, which is mentioned as below:

Request for Analysis of COVID-19 Impact on Retinal Gene Therapy Market- https://www.transparencymarketresearch.com/sample/sample.php?flag=covid19&rep_id=78059

Global Retinal Gene Therapy Market: Key Trends

The global retinal gene therapy market is characterized by the presence of the following restraints, drivers, and opportunities.

Advent of High-end Technologies to Support Development of the Market

Mostly in the cases of inherited retinal diseases, retinal gene therapy is performed. Gene therapy is capable of bettering vision impairment through mutation in RPE65 gene. Luxturna, a recently introduced gene therapy is utilized for the treatment of patients suffering from type 2 Leber's congenital amaurosis. This disease is a form of inherited disease that causes impairment in vision at the time of birth, which leads to a highly progressive degeneration. At present, there are many retinal gene therapy at the clinical trial phase and those are utilizing recombinant viruses. This factor is likely to increase the scope of growth for the global retinal gene therapy market over the period of assessment, from 2020 to 2030.

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In addition to that, the emergence of new market players together with the advent of high-end technological developments is likely to encourage growth of the global retinal gene therapy market during the forecast period. It is estimated that retinal gene therapy is likely to come up as a standard form of treatment for such retina-related diseases.

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Global Retinal Gene Therapy Market: Geographical Analysis

North America is clearly at the forefront of the growth of the global retinal gene therapy market at the very moment. It is estimated that the region will continue to retain its dominance over the period of forecast, from 2020 to 2030. So far, the product that has been approved for use is from a manufacturer from this region. Europe is likely to emerge as another lucrative region in the global retinal gene therapy market over the period of forecast.

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Retinal Gene Therapy Market: Advent of High-end Technologies to Support Development of the Market - BioSpace

SAN DIEGO, Feb. 5, 2021 /PRNewswire/ -- Angiocrine Bioscience, Inc., a clinical-stage biopharmaceutical company today announced that the American Society for Transplantation and Cellular Therapy (ASTCT) and the Center for International Blood & Marrow Transplant Research (CIBMTR) has selected Angiocrine's AB-205 Phase 1b/2 study results for an oral presentation.Intravenous AB-205 is being developed to treat diffuse damage of vascular niches of multiple organs caused by off-target cytotoxicity from high-dose chemotherapy (HDT) in the course of conditioning patients undergoing autologous hematopoietic cell transplantation to effect a cure of aggressive lymphomas.Treating the damaged vascular niches enable prompt repair of multiple organs.In case of HDT, the most severely and frequently affected organ systems are oral-gastrointestinal and hematopoietic.By enabling multi-organ repair, AB-205 has the potential of substantially reducing the incidence of severe transplant-related complications that can be life-threatening and prolong hospitalization.

"Our investigators and Angiocrine are honored to be selected by ASTCT & CIBMTR to present at its annual meeting this February," commented Paul Finnegan, MD, Angiocrine's CEO. "We look forward to Dr. Lihua Budde's presentation of AB-205's efficacy and safety results from our Phase 1b/2 study as well as preparing for the upcoming Phase 3 registration study for this indication."

Session Name: Oral Abstract - Session D - Acute Regimen-Related Toxicity and Supportive Care

Title of Abstract: Results of an Open Label Dose Escalation Trial of AB-205 (E-CELcells) in Adults with Lymphoma Undergoing High-Dose Therapy and Autologous Hematopoietic Cell Transplantation (HDT-AHCT)Session Date: Monday, February 8, 2021Session Time: 2:30 PM - 4:00 PM CSTPresentation Time: 3:00 PM CST

About Severe Toxicities and Complications during High-Dose Chemotherapy (HDT) and Autologous Hematopoietic Cell Transplant (AHCT)HDT followed by AHCT is considered a standard-of-care consolidative treatment to cure patients with aggressive systemic lymphoma who have failed 1st-line chemotherapy and respond to chemotherapy induction. Although highly effective in eradicating aggressive cancer cells, HDT also causes collateral damage to healthy tissue, which can lead to severe toxicities and serious, costly complications.The most affected organ system is the lining of the oral-gastrointestinal (GI) tract. The oral GI tract renews its mucosal lining every 3 to 7 days. Because of the collateral damage from HDT, the oral GI tract loses its ability to renew its lining, leading to inflammation (mucositis) and breakdown. The patient suffers from debilitating nausea, vomiting and diarrhea, refractory to medications and prolonging hospitalization.Severe oral GI complications can occur as frequently as 50% and cause profound misery.The rates and severity increase with age and, thus, many older patients are turned away from this potentially curative therapy due to concerns over complication risks.

About AB-205AB-205 represents a new and unique approach to repairing damaged tissues through advanced cell-and-gene therapy. AB-205 consists of allogeneic (off-the shelf) 'universal' E-CEL (human engineered cord endothelial) cells. Intravenous AB-205 is given after completion of HDT and on the same day as AHCT.AB-205's immediate action repairs damaged tissue and thereby prevents (reduces) the extent of breakdown of tissues, which is the root cause of severe toxicities experienced by patients. Reducing or preventing severe toxicities can lead to better quality of life and shorter stay in the hospitali.e., savings to the healthcare system. AB-205 was recently granted both the Regenerative Medicine Advanced Therapy (RMAT) Designation and Orphan Drug Designation (ODD) by the U.S. Food and Drug Administration (FDA). Angiocrine is currently advancing intravenous AB-205 into a multi-center single registration Phase 3 trial.

About Angiocrine Bioscience, Inc.Angiocrine Bioscience is a clinical-stage biotechnology company developing a radically new way to biologically repair damaged and diseased tissues and organs. Based on its novel and proprietary E-CEL platform, Angiocrine is developing multiple E-CEL therapies designed to repair damaged tissue from age-related degenerative disease of the musculoskeletal system; immune diseases that attack vessels and tissues; and ischemic diseases involving soft tissue, central nervous system and the heart.

For additional information, please contact:

Angiocrine Bioscience, Inc.John R. Jaskowiak(877) 784-8496IR@angiocrinebio.com

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Angiocrine Bioscience Announces Oral Presentation of Intravenous AB-205 Data during the Annual Transplantation & Cellular Therapy Meetings of ASCT...

Feb. 3, 2021 12:30 UTC

ST. LOUIS--(BUSINESS WIRE)-- M6P Therapeutics, a privately held life sciences company developing next-generation recombinant enzyme and gene therapies for lysosomal storage disorders (LSDs), today announced its scientific advisory board (SAB) that will support the Companys mission of translating its innovative bicistronic-S1S3 technology platform into best-in-class therapies that address unmet needs within the LSD community. The Companys platform enables improved biodistribution of recombinant enzymes to target tissues and efficient cross-correction for gene therapies.

As we work to advance our robust pipeline, we seek the input and support of a world-class team of scientific advisors with deep expertise in genetics, rare diseases, and lysosomal storage and metabolic disorders in particular, said Pawel Krysiak, president and chief executive officer of M6P Therapeutics. The collective insights, knowledge, commitment, and expertise of our scientific advisory board will help us translate this high science into potential medical benefit for the individuals affected by these serious conditions.

By combining the substantial expertise of the SAB with the expertise of the Companys internal R&D team in recombinant enzyme and gene therapies, M6P Therapeutics is well positioned to rapidly advance its deep pipeline of LSD programs. The members of the SAB are:

M6P Therapeutics bicistronic-S1S3 technology platform enhances mannose 6-phosphate content on lysosomal enzymes for both recombinant enzyme and gene therapies, which improves enzyme uptake across target tissues, said Stuart Kornfeld, MD, M6P Therapeutics co-founder and chairman of its SAB. With promising pre-clinical data across numerous LSD programs, this innovation can potentially translate into new and more efficacious treatments, reduced immunogenicity, and more efficient dosing regimens.

About M6P Therapeutics

M6P Therapeutics is a privately held, venture-backed biotechnology company developing the next-generation targeted recombinant enzyme and gene therapies for lysosomal storage disorders (LSDs). M6P Therapeutics proprietary bicistronic-S1S3 platform has the unique ability to enhance phosphorylation of lysosomal enzymes for both enzyme replacement and gene therapies leading to improved biodistribution and cellular uptake of recombinant proteins and efficient cross-correction of gene therapy product. This can potentially lead to more efficacious treatments with lower therapy burden, as well as new therapies for currently untreated diseases. M6P Therapeutics team, proven in rare diseases drug development and commercialization, is dedicated to fulfilling the promise of recombinant enzyme and gene therapies by harnessing the power of protein phosphorylation using its bicistronic-S1S3 platform. M6P Therapeutics mission is to translate advanced science into best-in-class therapies that address unmet needs within the LSD community. For more information, please visit: http://www.m6ptherapeutics.com.

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M6P Therapeutics Announces Formation of Distinguished, Experienced Scientific Advisory Board - BioSpace

The University of Sheffield has been given 5.85 million - the largest single gift from an individual alumnus in its history - to launch a new student support programme and search for new therapies for a range of incurable and debilitating diseases.

The University of Sheffield has been given 5.85 million - the largest single gift from an individual alumnus in its history - to launch a new student support programme and search for new therapies for a range of incurable and debilitating diseases.

The record donation has been made by The Law Family Charitable Foundation (LFCF), which was established by Andrew and Zo Law to support charitable initiatives with an emphasis on education and health.

Andrew Law is Chairman and CEO of Caxton Associates - a global macro hedge fund. He studied Economics at the University of Sheffield and graduated with a First Class honours degree in 1987.

The new student support initiative, named The Law Family Ambition Programme, will run over a five-year period to help disadvantaged students from low participation backgrounds access university, support their success in higher education and help them develop the skills, confidence and social abilities to graduate into successful careers.

It will fund new scholarships, academic mentoring, residential summer schools, career mentoring from successful Sheffield alumni, networking coaching, work placements and employability and skills workshops with businesses.

The landmark donation will also enable the University of Sheffield to expand and enhance its Discover outreach programme, which widens access to professions for pupils from disadvantaged backgrounds throughout the north of England.

Andrew Law said: The Law Family Charitable Foundation is delighted to support the University of Sheffield with the grant award. Since being established in 2011, LFCF has focused upon education and health, together with social mobility and the environment.

The country will only prosper if all of society has the access should it wish to participate fully in leading education opportunities, and supporting the disadvantaged is central to this. I owe a large part of my success to the University of Sheffield and we would like others to have equal opportunities. We are providing 2.85 million to launch a range of activities to help students from disadvantaged backgrounds help gain access and also be supported at the University.

The medical research donation will fund innovative research that could lead to the development of medical therapies for a range of rare genetic diseases which have a devastating effect on people's lives.

The University is already part of a new consortium which aims to accelerate the development of advanced therapies allowing potentially transformative treatments to reach patients sooner. However, the donation will enable the University to develop further partnerships with biotech and pharma companies to help accelerate gene therapy programmes and clinical trials for rare diseases at the same time as supporting regional economic growth and job creation.

The donation presents a real opportunity to drive innovation and excellence in the area of gene therapy and to catalyse the creation of new start-up companies to facilitate commercialisation in the North.

Andrew Law said: It is imperative that more leading UK universities expand their research, exploit their immense expertise for the greater good, and commercialise their success thus creating further growth. The University of Sheffield is developing a national and global reputation in gene therapy. Investing in new medical technology is very capital intensive but is critical to combat rare genetic diseases and cancers. With this in mind we are contributing 3 million to fund the next phase of the gene centres growth.

Professor Koen Lamberts, President and Vice-Chancellor of the University of Sheffield, said: We would like to thank Andrew, Zo and The Law Family Charitable Foundation for making such a generous gift to the University. This donation will make a huge difference to the work we do here at Sheffield. It will drive the next crucial step in research to tackle a range of diseases for which there is currently no cure, as well as provide vital support to students and young people who are considering applying to university. The gift will have a real impact and we are extremely grateful.

For more information on the Law Family Charitable Foundation please visit:

https://www.lawfamilycharitablefoundation.org/

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University of Sheffield receives record donation to support disadvantaged students and pioneering medical research - University of Sheffield News

Over the course of 2020, Sanofisaw significant growth in its specialty care sales primarily driven by its blue-chip asset, Dupixent, as well as an increase in vaccine sales that were driven by demand for differentiated influenza vaccines, the company announced in its year-end financial report.

For the year, Dupixent generated 3.5 billion (about $4.2 billion) for Sanofi. Dupixent, which was co-developed with Regeneron, continues to be a significant driver for Sanofi. In its year-end financial report, Sanofi said sales of Dupixent were driven by continued strong demand in atopic dermatitis (AD) in adult and adolescent patients, rapid adoption in children aged 6 to 11 years, and continued uptake in asthma and chronic rhinosinusitis with nasal polyposis. By the end of 2020, Dupixent has become available in 47 different countries with approximately 230,000 patients on therapy, Sanofi said.

Earlier this year, during a call ahead of the J.P. Morgan Healthcare conference, Frank Nestle, Sanofis chief scientific officer and Global Head of Research, and Dietmar Berger, Sanofis chief medical officer and Global Head of Development, predicted 2021 will be a transformative year for the company. Dupixent plays a significant role in that future, they said.

Sanofi Chief Executive Officer Paul Hudson touted the companys progress in 2020, despite some limitations imposed by the COVID-19 pandemic. He noted the continued uptake in Dupixent use across the globe and also pointed to potential approvals in new indications for the monoclonal antibody that inhibits the signaling of the interleukin-4 and interleukin-13 proteins. The company anticipates approval of Dupixent in pediatric asthma this year.

Other highlights for Sanofi include multiple acquisitions that are expected to provide the company a strong standing in areas like, immune-oncology, synthetic biology and cell and gene therapy. The company is also aiming for innovations with protein degraders, antibody conjugates, nanobodies and multi-specific antibodies, specifically bi- and tri-specific.

Hudson also pointed to its ongoing COVID-19 vaccine development programs. He said clinical trials are expected to begin on its vaccine candidates. Hudson also said the company is making a more immediate contribution to the pandemic fight by providing manufacturing support to BioNTech and Pfizer.

The year-end report wasnt all positives. Sanofi reported a few setbacks, including a Phase II Parkinsons disease study with venglustat. Sanofi said the Phase II study of Parkinsons disease patients with GBA mutations did not meet the primary endpoint at the end of January and the study for this indication was halted. The disclosure is a setback for the companys plans for venglustat. Ahead of the J.P. Morgan conference, company officials said venglustat had the potential to be a pipeline in a pill. The venglustat safety profile continues to be favorable and the development moves forward as planned in other Rare Disease indications, the company said. Venglustat is being assessed in other studies, including Phase III programs for GM2 Gangliosidosis and Autosomal Dominant Polycystic Kidney Disease, as well as Phase II studies in Fabry disease and Gaucher disease Type 3.

Other programs that will not continue include the anti-IL4/IL13 bispecific mAb Romilkimab, which was in a Phase II study for systemic scleroderma, as well as asthma treatment Itepekimab and lymphoma treatment Isatuximab. Both of those were also in Phase II.

Sanofi said it is working to resolve a Complete Response Letter issued by the U.S. Food and Drug Administration during the fourth quarter for sutimlimab, an investigational monoclonal antibody for the treatment of hemolysis in adults with cold agglutinin disease. The CRL was issued due to manufacturing concerns. There were no clinical concerns. Sanofi said it is working with both the FDA and the third-party manufacturer to reach a resolution in a timely manner.

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Sanofi Succeeding with Blue-Chip Asset, Dupixent, While Other Programs Fall Away - BioSpace

This study aims to explore the prevalence of decreased frontal lobe function and its associated factors in women with surgical menopause.This study is a retrospective analysis of a cross-sectional study conducted between October 2013 and July 2014. Data from 164 women with surgical menopause were analyzed. The Montreal Cognitive Assessment Thai version (MoCA-T) test was administered by a psychologist. The MoCA frontal lobe function score was derived from visuospatial/executive, abstraction, attention, and verbal fluency tests. Multivariable logistic regression was used to assess data associated with decreased frontal lobe function.The mean age of participants was 56.36.9years, and the mean time since menopause was 11.37.4years. The prevalence of decreased frontal lobe function score was 73.8%, with an average score of 6.211.84. The independent factor associated with a lower MoCA frontal lobe function score was duration of education greater than 6years.Our study adds information about decreased frontal lobe function in almost three-quarters of women with surgical menopause. Duration of education greater than 6years was a protective factor. Menopause hormone therapy usage did not seem to increase the detrimental effect on frontal lobe function when initiated in young women with surgical menopause.

PubMed

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Effect of surgical menopause and frontal lobe cognitive function. - Physician's Weekly

By Karen Dandurant| news@seacoastonline.com

FDA approves generic EpiPen

The FDA has approved the first official generic version of the EpiPen, a life-saving device for people with severe allergies.

Wochit

PORTSMOUTH -- Most people have heard of an EpiPen, and know, at least marginally, that they are used to help quickly counteract symptoms of severe allergic reactions that can quickly result in anaphylactic shock, which is life threatening and can be fatal.

There are many reasons for EpiPen use. Allergic reactions can come from bee stings, from food or medication allergies and even environmental allergies.

But health care experts say parents are often hesitant to use the device on their children, sometimes delaying until it is too late to stop the dangerous cascade of allergic reactions.

Eric Jaeger, a paramedic, and the EMS educator at Exeter Hospital said he sees hesitation and reluctance to use EpiPen all the time.

Portsmouth health officer: Stay vigilant before COVID variants hit

Parents sent home with an EpiPen are cautious, said Jaeger. There was a death several years ago of a college student who didnt use the EpiPen in time. It prompted me to realize there is still a significant issue, where people are not sure when to use it, or who wait longer than they should. Often people wait for a severe reaction and by that time, within 5-10 minutes, the cascade of symptoms has started, and a person can die.

EpiPens are a type of epinephrine auto-injector. They work by automatically injecting a dose of the hormone epinephrine (also called adrenaline) into a person's body.

Dr. Anupriya Grover, is a primary care physician at Appledore Family Practice in Portsmouth and is the Associate program director at the family medical residency program at Portsmouth Regional Hospital, in association with Tufts School of Medicine.

EpiPens are basically adrenaline and it is used to counteract symptoms that can constrict the airway, said Grover. That is an adverse event. The throat can swell; blood pressure can be lowered, and it can be a very serious condition. Bees and peanuts are commonly known allergies. Adults and children can be allergic to many medications, Tylenol, antibiotics, or opioids. There are airborne allergies that can be severe.

Grover said an EpiPen should be used in any situation where there is an allergy exposure resulting in difficulty breathing or tightening in the chest.

I have come into a home and as a paramedic, the signs of anaphylaxis are clearly there, said Jaeger. The parent is standing there holding an EpiPen. Once the dam has broken, an EpiPen might not pull the child back from the brink. Using it, even if unsure, makes so much more sense.

Read more: COVID precautions appear to reduce seasonal flu; doctors still urge vaccinations

I tell my patients if you are feeling like maybe you should use the pen, use it, said Dr. Nicholas Armellino, an emergency department physician at Exeter Hospital. The only time an EpiPen will not work, is if you do not use it. A lot of people are hesitant for whatever reason but my thought is if you do not feel well, use it. There are no detrimental effects for doing so, no contraindications.

Using an EpiPen is simple but Grover said taking the time to practice, using a fake EpiPen is a good idea.

I teach patients the swing and push method, said Grover. You hold your hands straight out and push into your thigh. The needle is so fine it will go through clothing. Then you hold for 10 seconds. People can practice this with a writing pen. The twin packs often come with their own practice pen.

No one would ever advocate using a medication past its expiration date, except with the use of an EpiPen in an extreme medical emergency.

MoreGuest View: NH must address prescription drug affordability now

Parents should inspect the device, said Grover. There should be no floating particles in it, and the liquid should not be cloudy. However, if you only have access to an expired pen; it is still preferable to not using it at all. Some residual benefits will still be there.

Armellino said the medication might not be fully active if past the expiration date. But he said the chance there is some active medication is definitely worth it in an emergency.

Ashley Child, PharmD at Wentworth-Douglass Hospital, said she tries to answer questions when parents come in to get an EpiPen for their child.

I agree there is some hesitation to use them, said Child. I tell them the rule of thumb is when in doubt, use it. EpiPens are not considered a dangerous medication when used properly. You cant buy it at a store, so if a family has one, it is a needed device, given by prescription. I think there is a small stigma attached to it. Some parents feel if they use it someone will judge them for it, will judge if they needed to. Thats not enough reason not to use it for your child when you feel it is needed. Most allergists will tell you the same thing, thatit's better to use than not to use. And in the case of anaphylaxis, the sooner you use it, the better. Delaying care can be harmful.

Child said using expired medication is never a decision to make without talking to your doctor.

However, studies have shown that after an EpiPen has been expired for six months, it still contains about 60% of its active ingredients, said Child. So, the benefit outweighs the risk.

Grover said EpiPens should be stored at room temperature, and never stored in a car where temperatures can fluctuate.

Cost may be a factor in peoples reluctance to use devices unnecessarily. A twin pack of EpiPens averages about $350, very pricey if a person has no insurance and has to pay out-of-pocket.

They are expensive, said Armellino. Its not actually the medicine that is expensive. Its the delivery system itself. I think there should be some entrepreneurial type who can create a less expensive way to deliver this medicine.

Jaeger said EMTs used to carry EpiPens, but the cost is prohibitive so many are now not being routinely equipped with them.

Child said all pharmacists should explain the pen and its use.

If you have questions ask, said Child. The more education you get, the better and more confident you will feel about using an EpiPen.

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EpiPen advice: Doctors say when in doubt, use it to nip allergic reactions in the bud - Seacoastonline.com

In the world of diabetes research, there are superstar names you just know.

For example, Banting and Best, who discovered insulin. Elliot Joslin, who pioneered modern treatment and care. Kamen, Tamborlane, and others who led technology development.

And then there are the names that may not be as familiar: researchers who spent years in labs studying and testing, who broke down barriers to reveal a better understanding of diabetes and, yes, better outcomes.

Dr. Jesse Roth of the Feinstein Institutes for Medical Research in Manhasset, New York, is one of those quiet heroes.

After 5 decades of lab work that have changed the diabetes treatment landscape, he was recently named one of the Giants in Medicine by the renowned Journal of Clinical Investigation.

His work hastened the discovery of insulin receptors and their molecular interactions within the body. New research by Dr. Roth and his colleagues around hormone-like molecules released within the intestine show promise for preventing pathological inflammation that is closely associated with diabetes and other illnesses, writes the Feinstein Institutes in an earlier press statement.

His lifetime contributions to understanding diabetes continues to lay the groundwork for future advancements.

Turns out none of this may have been the case were it not for a lost job opportunity, a war in Vietnam, and Roths then-bosses going overseas for a bit.

I felt like there was a divine finger pointing me in the right direction, Roth said of his career, still ongoing 50-plus years later.

As a freshly minted graduate from his medical residency program, Roth was at a loss for where to land next. After a well-known prestigious institute turned him down (they werent hiring at the time), a seasoned physician became a mentor and guided him along.

He took me for a drink in a fancy hotel in St. Louis, Roth told DiabetesMine. Id never had such a fancy drink before.

While the drink was sublime, it was the advice that resonated.

He looked me in the eye and said, The guy you want to work with is Rosalyn Yalow. She was at a small but avant-garde place in the Bronx (the Bronx Veterans Administration). Ends up, it was the perfect place for me.

There, Roth had a chance to hone his skills in research and in partnering on studies, he said, especially when the big guns there traveled to Europe on an extended trip.

That time to explore and expand helped him grow as a researcher, he said. And it was noticed.

When they came back, they saw us and me as future stars, he said.

Indeed, Yalow later won a Nobel Prize for her work with Dr. Solomon Berson proving that type 2 diabetes is caused by the bodys inefficient use of insulin, rather than a complete lack of insulin as was previously thought.

When the Vietnam War began, Roth, not wanting to be drafted, asked his boss for help. That boss landed him a job at the National Institutes of Health (NIH). He basically called the NIH and said, Ive got this kid for you! Roth remembered.

And that is where his diabetes-world-changing discoveries took root.

My boss there said to me, These are exciting times. Instead of doing what you were doing before, think of what the most exciting thing you could do would be, he said.

Roth settled on a question to answer: How does a cell know that insulin is there?

At that time, nearly 50 years ago, it was thought that insulin receptors were found in muscles and fat, and nowhere else in the body, Roth said. It was also thought that insulin worked pretty much on its own.

A younger Dr. Jesse Roth

What Roth and his team discovered was groundbreaking in every way: First, insulin receptors exist not just in pockets of muscle and fat, but throughout the entire body (even in the brain).

Second and this opened the door to researchers digging even deeper into the cause and effect of diabetes insulin itself does nothing, Roth said. Its the receptor that drives what the diabetes does.

It was definitely an aha moment, he said. It took us several years to convince people why this was important, which meant we had to stick to it and go several years with no applause. People were not ready for it. They yawned at us.

But Roth and his NIH team knew theyd revealed crucial and useful information.

And they were right. Their discovery led to not just understanding how insulin and receptors work in tandem in a body, but also that insulin works differently in different people.

This finding led to the now common understanding that there arent just one or even two types of diabetes: There are many, and each requires its own research, understanding, and treatment.

It used to be seen as one or two diseases, Roth said. Now, it turns out diabetes is a portfolio of diseases.

That discovery, along with more research looking at how receptors act in each type of diabetes, he said, led to better treatments, since physicians could now look at each type of disease with a unique set of eyes.

Roth said that is what jazzes up a researcher such as himself.

The lab is driven by whats going on in the clinic, he said. Any time there is an improvement clinically, were extremely excited as well as motivated.

For people with diabetes of any type, this knowledge led to more discoveries and, better yet, more precise treatments.

Being able to zero in on things like helping urine flush glucose is one example, Roth said, referring to SGLT2 drugs that leverage that pathway. This treatment has proven highly beneficial to people with type 2 diabetes, and is now sometimes introduced into treatment for those with type 1 as well.

Roths discovery of different forms of diabetes also helped practitioners explore things like exercise, stress, growth, and other factors that can come into play with insulin and receptors.

This has allowed people with diabetes and their medical teams to strive for better management and understand more when things go wonky.

While this may seem simple to anyone newer to diabetes, its important to remember that before this discovery, most people with type 1 diabetes took one injection a day and made few other changes in their dosing or daily activities around diabetes. This led to very imprecise diabetes management.

Today, all that has changed, thanks in large part to the work of Roth and his team.

Roths work over the years helped land him in some challenging and exciting roles.

He served as assistant surgeon general to the U.S. Public Health Service from 1985 to 1991 and as a fellow of the American College of Physicians, and has held leadership positions at the NIH and Johns Hopkins before joining Northwell Health and the Feinstein Institutes in 2000.

Today, Roth is still working hard and has more questions to answer in the lab.

Among them, he said, is further exploring the information he learned early on about the brain.

One area Im excited by is knowing we have insulin receptors all over the brain, he said.

We couldnt figure out what to do there, but there are groups working on this now in Munich and Cologne (Germany), and in the United States. Its exciting to think what could come of that, he said.

Roth would also like to see more investigation into insulin and the nervous system. We started it years ago but could not get people excited about it Now, its getting worked on.

Roth also has another dream that he believes can be reality: I am very optimistic that we can cure type 1 and type 2 diabetes, he said.

Theres no timeline attached to that, but not for lack of effort, he said.

The body is just much more complicated than we think, Roth said.

One more goal has less to do with the lab than the rest of the world, but its one Roth is passionate about: combating racial disparities in treatment.

There is a marked unevenness in care in the United States, Roth said. We dont see this in other places where healthcare is more accessible. We need to change that.

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Meet One of the World's Most Influential Diabetes Scientists - Healthline