Archive for September, 2020

The report is an all-inclusive research study of the global CRISPR And CRISPR-Associated (Cas) Genes Market taking into accounts the growth factors, recent trends, developments, opportunities, and competitive landscape. The CRISPR And CRISPR-Associated (Cas) Genes Market analyst and researchers have done a wide analysis of the global CRISPR And CRISPR-Associated (Cas) Genes Market with the help of research methodologies such as PESTLE and Porters Five Forces breakdown. They have provided exact and consistent market data and useful recommendations with an aim to help the players gain an insight into the overall current and upcoming market scenario.

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Leading manufacturers of CRISPR And CRISPR-Associated (Cas) Genes Market:

Caribou BiosciencesAddgeneCRISPR THERAPEUTICSMerck KGaAMirus Bio LLCEditas MedicineTakara Bio USAThermo Fisher ScientificHorizon Discovery GroupIntellia TherapeuticsGE Healthcare Dharmacon

The report presents a detailed competitive landscape along with a comprehensive analysis of the market share and size, product range, product innovation, technological advancements, and market patterns. The CRISPR And CRISPR-Associated (Cas) Genes Market report incorporates the study of recent developments in the market, such as product launches, mergers, acquisitions, collaborations, joint ventures, and partnerships, among others. The report offers a futuristic outlook of the market scenario for the forecast period of 2020-2026. The CRISPR And CRISPR-Associated (Cas) Genes Market regional analysis covers North America, Europe, Latin America, Asia-Pacific, and Middle East & Africa.

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CRISPR And CRISPR-Associated (Cas) Genes Market can be segmented into Product Types as

Genome EditingGenetic engineeringgRNA Database/Gene LibrarCRISPR PlasmidHuman Stem CellsGenetically Modified Organisms/CropsCell Line Engineering

CRISPR And CRISPR-Associated (Cas) Genes Market can be segmented into Applications as

Biotechnology CompaniesPharmaceutical CompaniesAcademic InstitutesResearch and Development Institutes

To breakdown the huge study that spreads through geographies, products, and end-use segments, among other market-specific segments, the authors present the CAGR of each segment during the years of anticipate. CAGR is a simplistic illustration of enlargement that visibly projects which segment register the highest/least growth through the forecast period 2020-2026.

The newest report about the CRISPR And CRISPR-Associated (Cas) Genes market provides a thorough estimate of the business vertical in question, alongside a brief overview of the industry segments. An extremely workable estimation of the present industry scenario has been delivering in the study, and the CRISPR And CRISPR-Associated (Cas) Genes market size with regards to the revenue and volume have also been mention.

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CRISPR And CRISPR-Associated (Cas) Genes Market Comprehensive Study Explores Huge Growth with Demand by Forecast 2024 - The Market Records

Global Genome Editing Market: Overview

Also known as genome editing with engineered nucleases (GEEN), genome editing is a method of altering DNA within a cell in a safe manner. The technique is also used for removing, adding, or modifying DNA in the genome. By thus editing the genome, it is possible to change the primary characteristic features of an organism or a cell.

The global genome editing market can be segmented on the basis of delivery method, technology, application, and geography. By technology, the global genome editing market can be segmented into Flp-In, CRISPR, PiggyBac, and ZFN. Based on delivery method, in vivo and ex vivo can be the two broad segments of the global genome editing market. By application, the global genome editing market can be categorized into medicine, academic research, and biotechnology.

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Global Genome Editing Market: Key Trends

Since genome editing is gaining rising adoption in the domain of scientific research for attaining a better understanding of biological aspects of organisms and how they work, the global genome editing market is likely to promise considerable growth over the forthcoming years. More importantly, genome editing is being used by medical technologies, where it can be used for modifying human blood cells which can then be placed back in the body for treating conditions such as AIDS and leukemia. The technology can also be potentially utilized to combat infections such as MRSA as well as simple genetic disorders including hemophilia and muscular dystrophy.

Global Genome Editing Market: Market Potential

As more easy-to-use and flexible genome technologies are being developed, greater potential of genome editing is being recognized across bioprocessing and treatment modalities. For instance, in May 2017, MilliporeSigma announced that it successfully developed a novel genome editing tool which can make the CRISPR system more productive, specific, and flexible. The researchers thus have a more number of experimental options along with faster results.

All this can lead to a growing rate of drug development, enabling access to more advanced therapies. Proxy-CRISPR, the new technique, makes access to earlier inaccessible aspects of the genome possible. As most of the existing CRISPR systems cannot manage without re-engineering of human cells, the new method is expected to gain more popularity by virtue of the elimination of the need for re-engineering, simplifying the procedures.

Several other market players are focusing on clinical studies with a view to produce effective treatments for different health conditions. For example, another major genome editing firm, Editas Medicine, Inc. announced the results of its pre-clinical study displaying the success of the CEP290 gene present in the retina of primates in the same month. With the positive results of the study, the companys belief in the vast potential of its candidate in the treatment of a genetically inherited retinal degenerative disease, Leber congenital amaurosis type 10, affecting childrens eyesight has been reinforced.

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Global Genome Editing Market: Regional Outlook

By geography, the global genome editing market can be segmented into Latin America, Europe, Asia Pacific, the Middle East and Africa, and North America. North America registered the highest growth in the past, and has been claiming the largest portion of the global genome editing market presently. The extraordinary growth of this region can be attributed to greater adoption of cutting edge technologies across several research organizations. The U.S., being the hub of research activities, is expected to emerge as the leading contributor. Asia Pacific is also likely to witness tremendous demand for genome editing over the forthcoming period, assisting the expansion of the global genome editing market.

Global Genome Editing Market: Competitive Analysis

CRISPR THERAPEUTICS, Caribou Biosciences, Inc., Sigma Aldrich Corporation, Sangamo, Intellia Therapeutics, Inc., Editas Medicine, Thermo Fisher Scientific, Inc., and Recombinetics, Inc are some of the key firms operating in the global genome editing market.

More here:
Genome Editing Market Poised to Expand at a Robust Pace Over 2025 - The Daily Chronicle

Mosquitoes are probably most people's least favorite insect.

Mosquito-borne diseases include malaria, yellow fever, and the Zika virus all of which can cause extreme sickness, birth defects, or death. Per year, malaria is responsible for the deaths of over 400,000 people, while dengue fever causes about 20,000, according to the World Health Organization.

Vaccines have been in development for dengue fever, malaria, and Zika, but production can be very slow and result in low efficacy. Drugs can help, but can be expensive. Insecticides are successful in the short term, but mosquitoes can become resistant, and mass-release insecticide can have health impacts on plants and wildlife.

Instead, othershave focused their attention on treating the mosquitos, not the symptoms:genetically modifying mosquitos themselves to only produce sterile offspring, effectively wiping out an entire population of insects. Modern genetic modification techniques are fast, and work at thescale of genes but they mimic the plant breedingfocused on phenotype humans have been doing since the beginning of history (like, say, selective breeding of crops, which humans have been doing since agriculture was invented).

There are two general approaches to producing genetically modified mosquitoes: 1) modifying the reproductive ability of male mosquitoes so that they cannot produce offspring, and 2) modifying both male and female mosquitoes so that they are resistant to diseases or incapable of transmitting them to other species. Oxitec, the biotechnology company behind a genetically modified (GM) mosquito Aedes aegypti, has tested the first approach to GM mosquito releases in field trials in parts of Brazil, the Cayman Islands, and Malaysia. These male mosquitoes mate with a female, exchanging a gene mutation that causes the larvae to die unless they are given an antibiotic. These trials have shown to reduce mosquito populations from 80-95%, reducing dengue fever cases by 91%.

GM mosquitoes are successful in reducing mosquito populations, and reducing disease spread. But there are still many scientific questions that haven't been fully answered, including concern over public health, mosquito resistance risks, and environmental consequences associated with genetic modification of a wild-living organism. Many people believe that genetic modification goes against the natural order of the world, or that we don't know the full range of possible outcomes for releasing modified organisms into the environment.

And the skeptics have a point: GM crops, initially thought to reduce our need for pesticide applications, have resulted in increased herbicide applications in the face of the development of resistant weeds and insect pests. In the case of genetically modified corn targeting the European corn borer pest, known as Bt corn, resistance has driven an increase in pesticide use by about 7% over a 15 year period.

A map showing dengue spread in the US as of August 2020, with outbreaks in Puerto Rico, Texas, Florida, and Wisconsin, among others

Via CDC

Studies testing the safety of GM crops are widespread, but the public often sees these announcements coming from huge corporations like General Mills, aiming at selling a GM product. In a 2014 Pew Research Center survey, only 37% of adults felt that GM foods were safe to eat, and only 26% felt GM foods were safe to eat if they stated they had less scientific knowledge than their peers. And yet, multiple science and health organizations have deemed GM crops as safe for human consumption.

But what does all of this uncertainty about GM crops mean for GM mosquitoes? Oxitec is currently the first and only company to be approved to release genetically modified mosquitoes, beginning in 2009 (though not announced until 2010) with the release of 3 million genetically modified mosquitoes in the Cayman Islands in response to high levels of pesticide resistance in the mosquito population. In 2015, in response to an increase in Zika virus in the country, Brazil's National Biosafety Committee approved the release of GM mosquitoes in Juazeiro, in northeast Brazil. The trials were successful, with Oxitec concluding the modified mosquitoes reduced dengue fever mosquito populations by 95%.

It's important to note that Oxitec was not the first to alter the genetics of insects for population control. The genetic approach to render insects infertile or resistant to disease is based on the Sterile Insect Technique (SIT). SIT was developed in the 1930s as a way to reduce insect populations. The technique renders male insects sterile by exposing them to extremely high levels of radiation, before releasing them to mate with females. The technique has great success, eradicating multiple pest species such as the screwworm in the 1950s and reducing pink bollworm moth populations in California beginning in 1967.

A workflow describing proposed stages for developing, testing, and releasing genetically modified mosquitos

Via WHO

Following the success of the initial trials, Oxitec had another GM mosquito release in Jacobina, Bahia, Brazil in 2015. About 450,000 mosquitoes were released each week for 27 months, developed using a strain from Cuba outcrossed with a strain from Mexico. The Brazil trials had little-to-no pushback in the region, with many residents believing the GM mosquito resulted in a drop in dengue fever cases and limiting the success of Zika virus spread.

Oxitec applied to the EPA in the United States to release the modified insect containing a gene in GM male mosquitoes that would spread to and kill offspring in the larval stage in 2012, after outbreaks in 2009 and 2010 of dengue fever in the Florida Keys. But when the public found out about the potential experiment, a petition on Change.org gathered over 100,000 signatures against the project (it's at over 235,000 today). Many skeptics reference Oxitec's rush to use GM mosquitoes in the field in their initial Cayman Island trials without properly consulting the public.

Oxitec scrapped their original release mechanism which would allow female mosquitoes to escape from the release capsule due to public pressure. Instead, they developed a newly branded version of the mosquito technology, coining it "friendly mosquito technology." The new GM mosquito is programmed to only release males from an Oxitec capsule placed in water, and not the biting females. The "safe" name refers to the fact that male mosquitoes do not bite, so the company's release would not increase numbers of female-biting mosquitoes.

A map showing Aedes aegypti's range in the United States and Indigenous reservations

Via CDC

But environmental groups are still unhappy. Many believe the engineered mosquitoes are still putting Floridians at risk, and worry that spreading modified genes from male mosquitoes into the wild mosquito population could cause cascading effects on the food chain. Birds, insects, and mammals all feed on mosquitoes; environmentalists are concerned about any affect the genes could have on these organisms. Many scientists have speculated about whether the elimination of the mosquito would have any impact on other creatures, but others are hesitant to promote the purposeful extinction of a species.

There are also other options for reducing diseases spread by mosquitoes. A non-profit organization in Brazil known as Eliminate Dengue is working to breed mosquitoes that are less likely to spread disease. The group is focused on breeding mosquitoes to carry on a parasite widely found and naturally occurring in mosquitoes, Wolbachia, which has been shown to inhibit the mosquito's ability to spread viruses such as dengue fever or chikungunya. Eliminate Dengue, funded through the Gates Foundation, has released these mosquitoes in 40 areas worldwide. The parasite stays in the population, reducing the need to release modified mosquitoes year after year.

The majority of scientists believe that GM mosquitoes pose no human health risks. However, the possibility of hybridization between GM mosquitoes and wild mosquitoes, passing the genetically modified genetics to wild populations and into the ecosystem, is concerning to many.

The potential good may outweigh the potential bad. But with EPA approval and test releases of genetically modified mosquitoes planned for the Florida Keys and Texas, the future is already here. Only time and data will tell.

More:
You should be excited that scientists are releasing 750 million genetically modified mosquitoes this year - Massive Science

A promising therapy for heart attacks uses stem cells to repair the damaged areas of the heart. However, getting the transplanted cells to stay at the site is a challenge. Now scientists have created a new type of off-the-shelf cardiac patch that overcomes these limitations.

The leading cause of death in the United States is coronary heart disease, which kills about 360,000 per year. Heart attacks result from the loss of blood flow to part of the heart muscle. This can be caused by fat, cholesterol and other substances forming plaque in the coronary arteries that supply oxygenated blood to the heart.

When the plaque breaks, a clot forms around it, which can prevent blood flow to a part of the heart and kill cells. The degree of damage depends on the area of the heart supplied by the blocked artery.

Treatments for a heart attack include limiting the original damage and blocking the secondary damage, which reduces long-term consequences and saves lives. As the heart heals, the damaged area forms scar tissue, which cannot pump blood like normal heart tissue, and it can affect the performance of the rest of the heart.

Cell therapy for heart attacks involves using cardiac stromal cells to encourage the heart to heal with muscle cells rather than scar tissue. Cardiac stromal cells interact with muscle cells and release chemical signals to encourage muscle cell growth.

This approach has only moderate benefits, because cardiac stromal cells are fragile and must be carefully stored and transported. Making matters worse, some stem cells can grow out of control and become tumors. Using a patients own cells has some advantages, but its expensive and time consuming. Theres also the problem of preventing the beating heart from washing the cells away.

Several types of scaffolds have been developed to keep the cardiac stromal cells at the proper location. However, these scaffolds dont overcome the cost and difficulties of isolating and expanding the stem cells.

Now a group of scientists has created a new type of artificial cardiac patch. It consists of a scaffolding matrix made from pig cardiac tissue, from which all cells have been removed. They then created artificial cardiac stromal cells by putting the important healing components from cardiac stromal cells into biodegradable microparticles within that matrix. The synthetic cardiac stromal cells mimic the therapeutic features of live stem cells while overcoming their storage and survival problems, and the matrix preserves the structures and activity found in cardiac tissue.

The artificial cardiac patch was shown to hold the synthetic cardiac stromal cells in place on the heart. In heart attack experiments in both rodents and pigs, the patch resulted in a 50 percent improvement in heart function and a 30 percent reduction in scarring when compared to no treatment.

Medical Discovery News is hosted by professors Norbert Herzog at Quinnipiac University, and David Niesel of the University of Texas Medical Branch. Learn more at http://www.medicaldiscoverynews.com.

Continued here:
Heart attack patches may save lives in US and beyond - Galveston County Daily News

Kyoto A project to make induced pluripotent stem cells, known as iPS cells, promptly and widely available at lower cost will get underway next year.

The My iPS Project will feature the creation of iPS cells, which can change into various types of functional cells, from the blood or other tissues of the patients themselves, to avoid rejection when a transplant is performed.

The project will be led by the CiRA Foundation at Kyoto University, which has taken over the business of stockpiling iPS cells from the university's Center for iPS Research and Application.

Headed by Shinya Yamanaka, a stem cell researcher and professor at the university who was awarded the Nobel Prize in Physiology or Medicine in 2012 for his pioneering work in iPS cell technology, the foundation was set up in September 2019 to make the business an independent operation financed by earnings and donations. It became a public interest foundation in April.

When a transplant is performed, the rejection of cells occurs if human leukocyte antigen, or HLA, from the donor is different from that of the recipient.

But with iPS cells produced from a person who has inherited the same type of HLA from his or her parents, rejection is considered rare for cells transplanted in another person with the same type of the antigen.

Using this knowledge, CiRA at Kyoto University has produced 27 kinds of iPS cells from the blood of seven healthy people and supplied them to research institutions and private companies for use in clinical studies and trials to facilitate regenerative medicine.

In 2017, research institutions such as Riken transplanted retina cells produced from the iPS cells in five patients suffering from intractable eye diseases. The first transplants of their kind in the world were followed by the transplants of nerve cells to the brain of a Parkinson's disease patient at Kyoto University and of a cardiac muscle sheet to a cardiac patient at Osaka University.

But the iPS cells stored by CiRA are of four kinds in terms of HLA type, estimated to eliminate rejection for only about 40 percent of all transplants for Japanese people. At CiRA, furthermore, iPS cells are manually cultivated by three well-trained people who are also responsible for preventing the entry of impurities and checking quality.

CiRA, therefore, can produce iPS cells only for three patients per year and transplants cost 40 million per person.

To reduce rejection, the foundation will develop technology to culture iPS cells from the blood or other tissues of the patients themselves and lower the cost of transplants. Starting in 2021, it will build a facility for automated processes from cultivation to inspection to stockpiling.

The project will be financed from the 5 billion that Tadashi Yanai, president and chairman of Fast Retailing Co., has pledged to donate to Kyoto University over 10 years.

The facility, with a total floor space of 1,500 square meters, will have many cylindrical, automated incubators as tall as human beings. It is planned to be completed in January 2025 so that its technology can be exhibited at the World Exposition to be held in Osaka in the year. To show appreciation for the donation, the facility will carry the name Yanai.

The project will realize the "ideal use" of iPS cells, Yamanaka said, declaring the aim of supplying them to 1,000 patients per year at 1 million per person.

Read this article:
Kyoto University project aims to supply iPS cells widely at low cost - The Japan Times

Autologous Stem Cell and Non-Stem Cell Based Therapies Market research report assists the business in every sphere of trade to easily take the unmatched decisions, to tackle the toughest business questions and diminish the risk of failure. Competitive analysis performed in this market report puts forth the moves of the key players in the industry such as new product launches, expansions, agreements, joint ventures, partnerships, and recent acquisitions. By precisely understanding and keeping into thought customer requirement, one step or combination of many steps has been employed to make out this most excellent Autologous Stem Cell and Non-Stem Cell Based Therapies Market report.

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TheGlobalAutologous Stem Cell and Non-Stem Cell Based Therapies Marketis expected to reach USD113.04 billion by 2025, from USD 87.59 billion in 2017 growing at a CAGR of 3.7% during the forecast period of 2018 to 2025. The upcoming market report contains data for historic years 2015 & 2016, the base year of calculation is 2017 and the forecast period is 2018 to 2025.

Some of the major players operating in the globalautologous stem cell and non-stem cell based therapies marketareAntria (Cro), Bioheart, Brainstorm Cell Therapeutics, Cytori, Dendreon Corporation, Fibrocell, Genesis Biopharma, Georgia Health Sciences University, Neostem, Opexa Therapeutics, Orgenesis, Regenexx, Regeneus, Tengion, Tigenix, Virxsys and many more.

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Market Definition:Global Autologous Stem Cell and Non-Stem Cell Based Therapies Market

In autologous stem-cell transplantation persons own undifferentiated cells or stem cells are collected and transplanted back to the person after intensive therapy. These therapies are performed by means of hematopoietic stem cells, in some of the cases cardiac cells are used to fix the damages caused due to heart attacks. The autologous stem cell and non-stem cell based therapies are used in the treatment of various diseases such as neurodegenerative diseases, cardiovascular diseases, cancer and autoimmune diseases, infectious disease.

According to World Health Organization (WHO), cardiovascular disease (CVD) causes more than half of all deaths across the European Region. The disease leads to death or frequently it is caused by AIDS, tuberculosis and malaria combined in Europe. With the prevalence of cancer and diabetes in all age groups globally the need of steam cell based therapies is increasing, according to article published by the US National Library of Medicine National Institutes of Health, it was reported that around 382 million people had diabetes in 2013 and the number is growing at alarming rate which has increased the need to improve treatment and therapies regarding the diseases.

Market Segmentation:Global Autologous Stem Cell and Non-Stem Cell Based Therapies Market

Major Autologous Stem Cell and Non-Stem Cell Based Therapies Market Drivers and Restraints:

Introduction of novel autologous stem cell based therapies in regenerative medicine

Reduction in transplant associated risks

Prevalence of cancer and diabetes in all age groups

High cost of autologous cellular therapies

Lack of skilled professionals

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Autologous Stem Cell and Non-Stem Cell Based Therapies Market 2020-2025 Global Briefing, Growth Analysis And Opportunities Outlook | Major Giants ...

Exosome Therapeutic Market analysis report encompasses infinite knowledge and information on what the markets definition, classifications, applications, and engagements are and also explains the drivers & restraints of the market which is obtained from SWOT analysis. Gathered market data and information is denoted very neatly with the help of most appropriate graphs, charts or tables in the entire report. Utilization of well established tools and techniques in this Exosome Therapeutic Market document helps to turn complex market insights into simpler version. Competitive analysis studies of this market report provides with the ideas about the strategies of key players in the market.

A large scale Exosome Therapeutic Market report endows with the data and statistics on the current state of the industry which directs companies and investors interested in this market. By applying market intelligence for this market research report, industry expert measure strategic options, summarize successful action plans and support companies with critical bottom-line decisions. The most appropriate, unique, and creditable global market report has been brought to important customers and clients depending upon their specific business needs. Businesses can accomplish great benefits with the different & all-inclusive segments covered in the Exosome Therapeutic Market research report hence every bit of market is tackled carefully.

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Market Analysis and Insights:Global Exosome Therapeutic Market

Exosome therapeutic market is expected to gain market growth in the forecast period of 2019 to 2026. Data Bridge Market Research analyses that the market is growing with a CAGR of 21.9% in the forecast period of 2019 to 2026 and expected to reach USD 31,691.52 million by 2026 from USD 6,500.00 million in 2018. Increasing prevalence of lyme disease, chronic inflammation, autoimmune disease and other chronic degenerative diseases are the factors for the market growth.

The major players covered in theExosome Therapeutic Marketreport areevox THERAPEUTICS, EXOCOBIO, Exopharm, AEGLE Therapeutics, United Therapeutics Corporation, Codiak BioSciences, Jazz Pharmaceuticals, Inc., Boehringer Ingelheim International GmbH, ReNeuron Group plc, Capricor Therapeutics, Avalon Globocare Corp., CREATIVE MEDICAL TECHNOLOGY HOLDINGS INC., Stem Cells Group among other players domestic and global.Exosome therapeutic market share data is available for Global, North America, Europe, Asia-Pacific, and Latin America separately. DBMR analysts understand competitive strengths and provide competitive analysis for each competitor separately.

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Exosomes are used to transfer RNA, DNA, and proteins to other cells in the body by making alteration in the function of the target cells. Increasing research activities in exosome therapeutic is augmenting the market growth as demand for exosome therapeutic has increased among healthcare professionals.

Increased number of exosome therapeutics as compared to the past few years will accelerate the market growth. Companies are receiving funding for exosome therapeutic research and clinical trials. For instance, In September 2018, EXOCOBIO has raised USD 27 million in its series B funding. The company has raised USD 46 million as series a funding in April 2017. The series B funding will help the company to set up GMP-compliant exosome industrial facilities to enhance production of exosomes to commercialize in cosmetics and pharmaceutical industry.

Increasing demand for anti-aging therapies will also drive the market. Unmet medical needs such as very few therapeutic are approved by the regulatory authority for the treatment in comparison to the demand in global exosome therapeutics market will hamper the market growth market. Availability of various exosome isolation and purification techniques is further creates new opportunities for exosome therapeutics as they will help company in isolation and purification of exosomes from dendritic cells, mesenchymal stem cells, blood, milk, body fluids, saliva, and urine and from others sources. Such policies support exosome therapeutic market growth in the forecast period to 2019-2026.

This exosome therapeutic market report provides details of market share, new developments, and product pipeline analysis, impact of domestic and localised market players, analyses opportunities in terms of emerging revenue pockets, changes in market regulations, product approvals, strategic decisions, product launches, geographic expansions, and technological innovations in the market. To understand the analysis and the market scenario contact us for anAnalyst Brief, our team will help you create a revenue impact solution to achieve your desired goal.

Global Exosome Therapeutic Market Scope and Market Size

Global exosome therapeutic market is segmented of the basis of type, source, therapy, transporting capacity, application, route of administration and end user. The growth among segments helps you analyse niche pockets of growth and strategies to approach the market and determine your core application areas and the difference in your target markets.

Based on type, the market is segmented into natural exosomes and hybrid exosomes. Natural exosomes are dominating in the market because natural exosomes are used in various biological and pathological processes as well as natural exosomes has many advantages such as good biocompatibility and reduced clearance rate compare than hybrid exosomes.

Exosome is an extracellular vesicle which is released from cells, particularly from stem cells. Exosome functions as vehicle for particular proteins and genetic information and other cells. Exosome plays a vital role in the rejuvenation and communication of all the cells in our body while not themselves being cells at all. Research has projected that communication between cells is significant in maintenance of healthy cellular terrain. Chronic disease, age, genetic disorders and environmental factors can affect stem cells communication with other cells and can lead to distribution in the healing process. The growth of the global exosome therapeutic market reflects global and country-wide increase in prevalence of autoimmune disease, chronic inflammation, Lyme disease and chronic degenerative diseases, along with increasing demand for anti-aging therapies. Additionally major factors expected to contribute in growth of the global exosome therapeutic market in future are emerging therapeutic value of exosome, availability of various exosome isolation and purification techniques, technological advancements in exosome and rising healthcare infrastructure.

Rising demand of exosome therapeutic across the globe as exosome therapeutic is expected to be one of the most prominent therapies for autoimmune disease, chronic inflammation, Lyme disease and chronic degenerative diseases treatment, according to clinical researches exosomes help to processes regulation within the body during treatment of autoimmune disease, chronic inflammation, Lyme disease and chronic degenerative diseases. This factor has increased the research activities in exosome therapeutic development around the world for exosome therapeutic. Hence, this factor is leading the clinician and researches to shift towards exosome therapeutic. In the current scenario the exosome therapeutic are highly used in treatment of autoimmune disease, chronic inflammation, Lyme disease and chronic degenerative diseases and as anti-aging therapy as it Exosomes has proliferation of fibroblast cells which is significant in maintenance of skin elasticity and strength.

Based on source, the market is segmented into dendritic cells, mesenchymal stem cells, blood, milk, body fluids, saliva, urine and others. Mesenchymal stem cells are dominating in the market because mesenchymal stem cells (MSCs) are self-renewable, multipotent, easily manageable and customarily stretchy in vitro with exceptional genomic stability. Mesenchymal stem cells have a high capacity for genetic manipulation in vitro and also have good potential to produce. It is widely used in treatment of inflammatory and degenerative disease offspring cells encompassing the transgene after transplantation.

Based on therapy, the market is segmented into immunotherapy, gene therapy and chemotherapy. Chemotherapy is dominating in the market because chemotherapy is basically used in treatment of cancer which is major public health issues. The multidrug resistance (MDR) proteins and various tumors associated exosomes such as miRNA and IncRNA are include in in chemotherapy associated resistance.

Based on transporting capacity, the market is segmented into bio macromolecules and small molecules. Bio macromolecules are dominating in the market because bio macromolecules transmit particular biomolecular information and are basically investigated for their delicate properties such as biomarker source and delivery system.

Based on application, the market is segmented into oncology, neurology, metabolic disorders, cardiac disorders, blood disorders, inflammatory disorders, gynecology disorders, organ transplantation and others. Oncology segment is dominating in the market due to rising incidence of various cancers such as lung cancer, breast cancer, leukemia, skin cancer, lymphoma. As per the National Cancer Institute, in 2018 around 1,735,350 new cases of cancer was diagnosed in the U.S. As per the American Cancer Society Inc in 2019 approximately 268,600 new cases of breast cancer diagnosed in the U.S.

Based on route of administration, the market is segmented into oral and parenteral. Parenteral route is dominating in the market because it provides low drug concentration, free from first fast metabolism, low toxicity as compared to oral route as well as it is suitable in unconscious patients, complicated to swallow drug etc.

The exosome therapeutic market, by end user, is segmented into hospitals, diagnostic centers and research & academic institutes. Hospitals are dominating in the market because hospitals provide better treatment facilities and skilled staff as well as treatment available at affordable cost in government hospitals.

Exosome therapeutic Market Country Level Analysis

The global exosome therapeutic market is analysed and market size information is provided by country by type, source, therapy, transporting capacity, application, route of administration and end user as referenced above.

The countries covered in the exosome therapeutic market report are U.S. and Mexico in North America, Turkey in Europe, South Korea, Australia, Hong Kong in the Asia-Pacific, Argentina, Colombia, Peru, Chile, Ecuador, Venezuela, Panama, Dominican Republic, El Salvador, Paraguay, Costa Rica, Puerto Rico, Nicaragua, Uruguay as part of Latin America.

Country Level Analysis, By Type

North America dominates the exosome therapeutic market as the U.S. is leader in exosome therapeutic manufacturing as well as research activities required for exosome therapeutics. At present time Stem Cells Group holding shares around 60.00%. In addition global exosomes therapeutics manufacturers like EXOCOBIO, evox THERAPEUTICS and others are intensifying their efforts in China. The Europe region is expected to grow with the highest growth rate in the forecast period of 2019 to 2026 because of increasing research activities in exosome therapeutic by population.

The country section of the report also provides individual market impacting factors and changes in regulation in the market domestically that impacts the current and future trends of the market. Data points such as new sales, replacement sales, country demographics, regulatory acts and import-export tariffs are some of the major pointers used to forecast the market scenario for individual countries. Also, presence and availability of global brands and their challenges faced due to large or scarce competition from local and domestic brands, impact of sales channels are considered while providing forecast analysis of the country data.

Huge Investment by Automakers for Exosome Therapeutics and New Technology Penetration

Global exosome therapeutic market also provides you with detailed market analysis for every country growth in pharma industry with exosome therapeutic sales, impact of technological development in exosome therapeutic and changes in regulatory scenarios with their support for the exosome therapeutic market. The data is available for historic period 2010 to 2017.

Competitive Landscape and Exosome Therapeutic Market Share Analysis

Global exosome therapeutic market competitive landscape provides details by competitor. Details included are company overview, company financials, revenue generated, market potential, investment in research and development, new market initiatives, global presence, production sites and facilities, company strengths and weaknesses, product launch, product trials pipelines, concept cars, product approvals, patents, product width and breadth, application dominance, technology lifeline curve. The above data points provided are only related to the companys focus related to global exosome therapeutic market.

Many joint ventures and developments are also initiated by the companies worldwide which are also accelerating the global exosome therapeutic market.

For instance,

Partnership, joint ventures and other strategies enhances the company market share with increased coverage and presence. It also provides the benefit for organisation to improve their offering for exosome therapeutics through expanded model range.

Customization Available:Global Exosome Therapeutic Market

Data Bridge Market Researchis a leader in advanced formative research. We take pride in servicing our existing and new customers with data and analysis that match and suits their goal. The report can be customised to include price trend analysis of target brands understanding the market for additional countries (ask for the list of countries), clinical trial results data, literature review, refurbished market and product base analysis. Market analysis of target competitors can be analysed from technology-based analysis to market portfolio strategies. We can add as many competitors that you require data about in the format and data style you are looking for. Our team of analysts can also provide you data in crude raw excel files pivot tables (Factbook) or can assist you in creating presentations from the data sets available in the report.

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Exosome Therapeutic Market Latest Industry Size, Growth, Share, Emerging Demands, and Competitive Landscape || Major Gaints Jazz Pharmaceuticals,...

A Cleveland Clinic study shows that 5 to 10 percent of surgically induced weight loss is associated with improved life expectancy and cardiovascular health. In comparison, about 20 percent weight loss is necessary to observe similar benefits with a non-surgical treatment. The findings also show that metabolic surgery may contribute health benefits that are independent of weight loss. The study is published in the October issue of Annals of Surgery.

Ali Aminian, M.D.

This large observational study looked at 7,201 Cleveland Clinic patients: 1,223 patients with obesity and type 2 diabetes who underwent metabolic surgery (bariatric or weight loss surgery) were matched to 5978 patients who received usual medical care. About 80 percent of the patients had hypertension, 74 percent had dyslipidemia (elevated triglycerides and cholesterol), and 31 percent were taking insulin to treat their diabetes.

Using different statistical models, the effects of weight loss were studied to identify the minimum weight loss needed to decrease the risk of death and of experiencing major adverse cardiovascular events, such as coronary artery events, cerebrovascular events, heart failure, kidney disease, and atrial fibrillation.

Following metabolic surgery, the risk of death and major heart complications appears to decrease after about 5 percent and 10 percent weight loss, respectively. Whereas, in the nonsurgical group, both the risk of death and major cardiovascular complications decreased after losing approximately 20 percent of body weight, said Ali Aminian, M.D., director of Cleveland Clinics Bariatric & Metabolic Institute, and lead author of the study.

Steve Nissen, M.D.

This study suggests greater heart disease benefits are achieved with less weight loss following metabolic surgery than medical weight loss using lifestyle interventions. The study findings suggest that there are important benefits of metabolic surgery independent of the weight loss achieved, said Steven Nissen, M.D., Chief Academic Officer of the Heart, Vascular & Thoracic Institute at Cleveland Clinic, and the studys senior author.

The groundbreaking STAMPEDE study showed metabolic surgerys beneficial effects on blood glucose control. Since then, additional studies have observed health benefits other than weight loss following metabolic surgery. In fact, this research is a secondary analysis of a large study that showed weight-loss surgery is associated with a 40 percent reduction in risk of death and heart complications in patients with type 2 diabetes and obesity.

Researchers continue to study the physiological changes in the surgically modified gastrointestinal tract, the impact on hormone secretion and the microbiome. Those beneficial changes may contribute to the cardiovascular and survival benefits of metabolic surgery, independent of weight loss. More research is needed to better understand the underlying mechanisms for the health benefits of metabolic surgery in patients who have obesity and type 2 diabetes.

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Cleveland Clinic Study Identifies Weight-Loss Threshold for Cardiovascular and Survival Benefits in Patients with Obesity and Diabetes - Health...

If youre living with hypothyroidism, youre likely already familiar with the common symptoms, which can include fatigue and weight gain. But if youre a woman around the age of menopause which can range from 40 to 58, with the average onset at age 51 you may also experience identical issues, according to the North American Menopause Society (NAMS). So if youre in middle age, it can be hard to know whats really going on, and how to get relief.

Read on for what you should know about your menopausal years if you have hypothyroidism.

Menopause is a natural transition that women experience, and its the permanent end of menstruation. After a woman has had her final period and doesnt menstruate for 12 consecutive months, shes been through menopause, according to the NAMS.

During perimenopause the time leading up to menopause and menopause, hormonal fluctuations are common. This can even be when hypothyroidism develops in some women. One theory is that fluctuation in estrogen levels may increase inflammation, creating the ideal environment for development of autoimmune diseases such as Hashimoto's thyroiditis, which may lead to hypothyroidism, explains Cheryl R. Rosenfeld, DO, a spokesperson for the American Association of Clinical Endocrinologists, partner at North Jersey Endocrine Consultants, and adjunct clinical associate professor of medicine at the Touro College of Osteopathic Medicine in New York City. The drop in estrogen is what causes menopausal symptoms.

Hypothyroidism occurs when the thyroid gland does not make enough thyroid hormone. The most common cause is inflammation of the thyroid, but it may also be due to a number of other causes, including too much or too little iodine in the body, taking medications such as lithium, and inflammation of the thyroid, according to the American Thyroid Association (ATA).

Even if youve had hypothyroidism for a while, when you reach menopausal age, the lines between hypothyroidism and menopause can start to blur. Symptoms of hypothyroidism can be confused with symptoms due to the menopause transition, says Ekta Kapoor, MBBS, an endocrinologist in the Menopause and Women's Sexual Health Clinic at the Mayo Clinic in Rochester, Minnesota.

These same symptoms can include:

Rosenfeld cautions against using synthetic thyroid hormone medication to alleviate menopause symptoms in women with normal thyroid function. "While there is a great deal of overlap in the symptoms, if the thyroid blood tests are normal, it would be wrong to treat a person without thyroid disease with thyroid hormone or to increase thyroid hormone in a person with hypothyroidism to alleviate symptoms of menopause," she says. "Thyroid hormone is not a cure-all for symptoms it is a treatment for hypothyroidism."

If youre looking for a clue as to whats causing your issues, check your body heat hot flashes and night sweats are unique to menopause, says Dr. Kapoor. But there is an exception: Over-replacement with thyroid hormone can also cause hot flashes and night sweats, notes Kapoor. So work with your healthcare provider to ensure you are getting an appropriate amount of thyroid hormone treatment. If you are and youre still waking up sweating, thats a good indication that you are indeed experiencing menopause symptoms.

Hypothyroidism can worsen menopause symptoms. So rather than shrug off your symptoms, its important to maintain hypothyroidism treatment during menopause. Undertreated thyroid disease may cause increased cholesterol, leading to atherosclerosis and possibly heart attack or stroke, says Rosenfeld. In addition, hypothyroidism may cause high blood pressure, leading to similar heart complications. Overtreatment with thyroid hormone is also a known cause of osteoporosis, Kapoor adds.

Healthcare providers know to adjust thyroid treatment based on your current needs, and that includes during menopause. When estrogen levels drop at the time of menopause, people who are on levothyroxine to treat hypothyroidism may experience a change in their thyroid hormone needs, usually downward, says Rosenfeld. And conversely, hormone therapy which is commonly used to treat menopause symptoms can necessitate the opposite. If a woman treated with thyroid hormone starts estrogen, her dose of levothyroxine may have to be increased, says Kapoor.

A study published in December 2018 in the journal Climacteric stated that the decision to use hormone therapy for menopause should be individualized in women with thyroid disorders such as hypothyroidism.

The bottom line: Work closely with your healthcare provider so your thyroid symptoms and your menopause symptoms can be kept to a minimum.

Original post:
What to Know About Menopause if You Have Hypothyroidism - Everyday Health

A year and a half after landing a $70 million Series C round, Shanghai-based InventisBio has more than doubled that amount in a hefty Series D.

Merck vet Yaolin Wang launched Inventis from scratch back in 2015. In 2017, the biotech had 7 staffers, a $19 million Series B and an ambitious two- to three-year timeline to its first approval. While the company has yet to snag a regulatory OK, its lead candidate targeting EGFR T790M mutations is currently in registrational trials as a first- and second-line treatment in non-small cell lung cancer.

Those trials, the company said, are progressing smoothly.

The $147 million Series D will be used to give the biotechs gout and breast cancer drugs a big push.D-0502, an oral selective estrogen receptor degrader (SERD) and estrogen receptor antagonist, is currently in Phase I development for hormone receptor-positive breast cancer. D-0120 is in Phase I for gout. Wang plans to launch both candidates into Phase II trials in China and the US.

The D round was led by Hillhouse affiliate GL Ventures. Returning investors Lilly Asia Venture, OrbiMed Asia, Pudong Innotek, AdvanTech Capital and CMB International chipped in, as well as new investors Qiming Venture Partners, Janchor, AIHC Capital, Matrix Partner China, Dyee Capital and E Fund Capital.

In 2017, upon announcing its Series B, InventisBio also said it was developing drugs that can be combined with other immune-oncology targeted therapies, such as PD-1 antibody, for various cancer indications. A year later, the biotech agreed to out-license its NSCLC candidate to Betta Pharma for 230 million ($33.7 million). The deal gives Betta Pharma exclusive commercialization rights in China, while InventisBio keeps its options in other regions of the globe.

InventisBio is committed to developing first- and best-in-class innovative drugs for cancer and other major diseases, Wang said in a statement.

More:
InventisBio reels in $147M to push gout and breast cancer drugs along - Endpoints News

September is Polycystic Ovarian Syndrome Month, and while this is a condition that is hard to treat, as hormones go into overdrive and create symptoms throughout the body, there are dietary ways to alleviate some of the worst symptoms, and most of the foods doctors recommend are plant-based.

PolyCystic Ovarian Syndrome, also known as PCOS, is one of the most common endocrine disorders among women of childbearing age. In the United States, 1 in 10 reproductive-age women will experience PCOS in their lifetime.

The syndrome is thought to be caused by hormonal imbalance, which is linked to metabolism problems that may affect a woman's overall health, reproductive health, and appearance. Specifically, PCOS patients are likely to have high levels of androgens, or testosterone, whichin turncan lead to symptoms such as excess body hair or Hirsutism, and cystic acne, irregular or painful menstrual cycles, orsmall cysts on the ovaries, that only show up on an ultrasound. Moreover, 80 percent of PCOS patients tend to be overweight or have excess fat around the belly, which leads to difficulty processing the hormone, insulin. Because of the excess weight associated with the condition, PCOS patients can often have higher rates of sleep apnea, joint pain, and trouble with conceiving.

While there are medical and pharmaceutical treatment options to manage symptoms, lifestyle changes are key to long-term success when treating PCOS, and dietary changes are often recommended as the first intervention.Dr. Shebani Sethi Dalai, MD, MS, the Founding Director of Stanfords Metabolic Psychiatry Clinic, and Silicon Valley Metabolic Psychiatry advises that patients need to focus on the connection between metabolic function and mental health as well, to help come up with a strategy for dealing with PCOSby reducing inflammation and insulin resistance through food choices.

Research shows that healthy eating and physical activity can help manage symptoms of PCOS, says Dr. Sethi Dalai. This includes blood sugar and insulin resistance, which we often see in PCOS and is a cause for infertility in young reproductive-age women.

Food choices matter when dealing with PCOS, according to astudy published in the International Journal of Reproductive Medicine, which examined the relationship between a Healthy Eating Index (HEI) and PCOS. Researchers found that the consumption of whole grains, plant proteins, and fewer refined grains was associated with a lower risk of PCOS in women ages 20-40. Here are the six best foods to eat if you are dealing with PCOS.

Green leafy veggies like broccoli, brussels sprouts, and cabbage are filled with tons of magnesium, which are incredibly helpful in tackling insulin resistance, seen in many individuals with PCOS. One study published 2019 in Food Science & Nutrition found that increasing dietary fiber and magnesium may assist in reducing insulin resistance and hyperandrogenemia (the increased production of androgens, common in women with PCOS). Leafy greens, such as spinach, collard greens, and mustard greens contain high amounts compared to other vegetables. One cup of cooked spinach has 157mg of magnesium (37% DV). Leafy greens are also filled with nutritious vitamins and minerals, such as iron, manganese, and Vitamins A, C, and K.

Fibrous, non-starchy green vegetables can be consumed with each meal. They are helpful in reducing blood sugar and they add to the magnesium daily value needed, adds Dr. Sethi Dalai.

This family of nutrient-dense plants of chickpeas, beans, lentils, peas, and soybeans are loaded with rich fiber, protein, and zero cholesterol. A 2018 study published in Nutrients examined the association between certain beans and pulses in effectively reducing insulin resistance. Findings supported how legumes such as soybeans and pulses are known to be beneficial for diabetes management as a result of their low glycemic index, which leads to a low rise in blood sugar after consumption. Research shows that women who have PCOS have a higher risk of developing type 2 diabetes, due to high glucose levels and insulin resistance. Lentils and chickpeas can be an easy protein boost to your plant-based tacos, salads, and soup!

Unsaturated fats have been proven to balance hormones and optimize insulin levels in women with PCOS. Healthy fats such as certain nuts and seeds are filled with Omega-3 fatty acids, which has abundant health benefits for the body and the brain. A study published in 2017 in Experimental and Clinical Endocrinology & Diabetes found that supplementing with omega-3 fatty acids for 12 weeks drove a reduction in testosterone levels and improved insulin resistance in women with PCOS, through a randomized double-blind placebo-controlled trial.

Use healthier, less processed oils such as olive or avocado oil with higher heat cooking. Try eating avocado in your salad or with your breakfast. Your breakfast should have adequate fat and protein, such as egg (or substitute a plant-based protein if you are vegan) and avocado, says Dr. Sethi Dalai.

Findings from a 2018 study published in The Journal of Metabolic Diseases supported evidence that oxidative stress and decreased antioxidant status are often linked with PCOS. Colorful berries are extremely beneficial for patients with PCOS as they are filled with antioxidants that may help reduce oxidative stress. They also help control free radicals, unstable molecules that can cause damage to your body when in high amounts. Berries contain polyphenols, which have been shown to help in the management of weight, diabetes, and indigestion. Highbush blueberries (560mg), blackberries (260mg), and strawberries (235mg) are some of the colorful berries highest in polyphenols.

Berries are a good fruit choice because they are high in fiber and low in sugar compared to other fruits like mango, watermelon, or banana, says Dr. Sethi Dalai. They can also serve as a healthy dessert option.

The gut microbiota is the largest population of microorganisms in the human body, which reside in the intestine. Research has shown that gut microbiota can cause insulin resistance and may contribute to the development of PCOS by impacting energy absorption, the brain-gut axis, and much more. A 2020 study explored the consumption of probiotics to treat PCOS. Findings from the research suggested that probiotics can be used to regulate gut microbiota and treat metabolic diseases, which also points to a new therapeutic direction for the treatment of the metabolic abnormalities associated with PCOS. While many dairy products are filled with probiotics, there are a multitude of fermented vegan options such as sauerkraut, pickles, kimchi, and kombucha.

A study published in 2018 in the European Journal of Endocrinology supported evidence that women with PCOS are at high risk of contracting type 2 diabetes than their counterparts. Whole grains high in fiber, such as rolled oats, bulgur, quinoa, and buckwheat, are slow-release carbohydrates. This means that they have a low glycemic index, releasing sugar into the blood at a slower, more regular pace. Therefore, they are less likely to cause spikes in blood sugar levels. They will also give you a boost of energy and keep you satiated for long periods of time.

If you are choosing between simple versus complex carbohydrates, always choose complex since the glycemic index is lower, adds Dr. Sethi Dalai. While whole grains are higher in fiber, I would not recommend excessive consumption for someone with insulin resistance.

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The 6 Best Foods to Eat to Alleviate PCOS Symptoms - The Beet

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Gallstones are hardened deposits of digestive fluid that can form in your gallbladder, an organ on the right side of your abdomen that holds bile to be released into your small intestine.

Gallstones range in size from as small as a grain of sand to as large as a golf ball, according to the Mayo Clinic. Some people develop just one gallstone, while others develop many gallstones at the same time.

People who develop gallstones and experience symptoms (caused when gallstones lodge in a duct, resulting in a blockage) often require surgery to remove them. Those without symptoms, however, usually dont require treatment.

Symptoms include:

Sudden and rapidly intensifying pain in the upper right portion of your abdomen and/or the center of your abdomen, just below your breastbone Back pain between your shoulder blades Pain in your right shoulder Nausea or vomitingPain associated with gallstones can last several minutes to a few hours.See a doctor if you experience pain so intense that you cant sit still or find a comfortable position, yellowing of your skin and the whites of your eyes (jaundice), or high fever with chills.Possible causes of gallstones: Your bile contains too much cholesterol. Normally, your bile contains enough chemicals to dissolve the cholesterol excreted by your liver. Your bile contains too much bilirubin, a chemical produced when your body breaks down red blood cells. Certain conditions cause your liver to make too much bilirubin, including cirrhosis, biliary tract infections and certain blood disorders. Your gallbladder doesnt empty correctly. Possible risk factors: Being female Being 40 or older Being a Native American Being a Mexican American Being overweight or obese Being sedentary Being pregnant Eating a high-fat diet Eating a high-cholesterol diet Eating a low-fiber diet Having a family history of gallstones Having diabetes Having certain blood disorders, such as sickle cell anemia or leukemia Losing weight very quickly Taking medications that contain estrogen, such as oral contraceptives or hormone therapy drugs Having liver diseaseTo reduce risk of gallstones: Dont skip meals or fast. Try to stick to your usual meal times each day. Lose weight gradually, a maximum of 1-2 pounds per week. Eat more high-fiber foods, such as fruits, vegetables and whole grains. Maintain a healthy weight.

Read more:
Gallstones: What they are, how to prevent them - The Providence Journal

Global Bone Marrow Transplant Rejection Treatment Market reports provide in-depth analysis of Top Players, Geography, End users, Applications, Competitor analysis, Revenue, Price, Gross Margin, Market Share, Import-Export data, Trends and Forecast. The study will feature estimates in terms of sales revenue and consumption from 2020 to 2025, at the global level and across the major regions mentioned above. The study has been created using a unique research methodology specifically designed for this market.

Quantitative information includes Bone Marrow Transplant Rejection Treatment market estimates and forecast for a upcoming years, at the global level, split across the key segments covered under the scope of the study, and the major regions and countries. Sales revenue and consumption estimates, year-on-year growth analysis, price estimation and trend analysis, etc. will be a part of quantitative information for the mentioned segments and regions/countries.

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Qualitative information will discuss the key factors driving the restraining the growth of the market, and the possible growth opportunities of the market, regulatory scenario, value chain and supply chain analysis, export and import analysis, attractive investment proposition, and Porters 5 Forces analysis among others will be a part of qualitative information. Further, justification for the estimates for each segments, and regions will also be provided in qualitative form.

The major types mentioned in the report are Azathioprine, Adrenocorticotropic Hormone, Cyclophosphamide, Cyclosporine A, Others and the applications covered in the report are Hospital, Clinic, Others,.

Major players profiled in the report include The Bellicum Pharmaceuticals, Inc., Bio-Cancer Treatment International Limited, Biogen Inc, Boryung Pharmaceutical Co., Ltd., Bristol-Myers Squibb Company, Cantex Pharmaceuticals, Inc., Capricor Therapeutics, Inc., Cell Source, Inc., Cell2B S.A., CellECT Bio, Inc., Cleveland BioLabs, Inc., Compugen Ltd., Cynata Therapeutics Limited, Cytodyn Inc., Dompe Farmaceutici S.p.A., Dr. Falk Pharma GmbH, Escape Therapeutics, Inc., F. Hoffmann-La Roche Ltd., Fate Therapeutics, Inc., Generon (Shanghai) Corporation Ltd., Gilead Sciences, Inc., GlaxoSmithKline Plc, Idera Pharmaceuticals, Inc..

The study will also feature the key companies operating in the industry, their product/business portfolio, market share, financial status, regional share, segment revenue, SWOT analysis, key strategies including mergers and acquisitions, product developments, joint ventures and partnerships an expansions among others, and their latest news as well. The study will also provide a list of emerging players in the Bone Marrow Transplant Rejection Treatment market.

Based on regions, the market is classified into North America, Europe, Asia Pacific, Middle East and Africa and Latin America. The study will provide detailed qualitative and quantitative information on the above mentioned segments for every region and country covered under the scope of the study.

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Global Bone Marrow Transplant Rejection Treatment Market Outlook by Types, Applications, End Users and Opportunities to 2025 - The Daily Chronicle

By Pam BelluckAnnrene Rowe was getting ready to celebrate her 10th wedding anniversary this summer when she noticed a bald spot on her scalp. In the following days, her thick, shoulder-length hair started falling out in clumps, bunching up in the shower drain.

I was crying hysterically, said Rowe, 67, of Anna Maria, Florida.

Rowe, who was hospitalised for 12 days in April with symptoms of the coronavirus, soon found strikingly similar stories in online groups of Covid-19 survivors. Many said that several months after contracting the virus, they began shedding startling amounts of hair.

Doctors say they too are seeing many more patients with hair loss, a phenomenon they believe is indeed related to the coronavirus pandemic, affecting both people who had the virus and those who never became sick.

In normal times, some people shed noticeable amounts of hair after a profoundly stressful experience such as an illness, major surgery or emotional trauma.

Theres many, many stresses in many ways surrounding this pandemic, and were still seeing hair loss because a lot of the stress hasnt gone away, said Dr. Shilpi Khetarpal, an associate professor of dermatology at the Cleveland Clinic.

Before the pandemic, there were weeks when Khetarpal didnt see a single patient with hair loss of this type. Now, she said, about 20 such patients a week come in. One was a woman having difficulty home-schooling two young children while also working from home. Another was a second-grade teacher anxiously trying to ensure that all her students had computers and internet access for online instruction.

In a July survey about post-Covid symptoms among 1,567 members of a survivors group, 423 people reported unusual hair loss, according to the group, Survivor Corps, and Natalie Lambert, an associate research professor at Indiana University School of Medicine, who helped conduct the survey.

Making healthy lifestyle changes in terms of diet, exercise and managing stress, in addition to other factors, can go a long way in helping the immune system get the boost it requires.

On International Immunology Day, Chennai-based clinical nutritionist, lactation consultant and diabetes educator - Ramya Ramachandran - shares five tips that can give your life a healthy spin.

Ginger, garlic, star anise, omega-3 fats, flax seeds are some foods that have antiviral and anti-inflammatory properties. These can be included in your meals daily. Its also important to focus on micronutrients which have a major impact on health.

To add the required amount of micronutrients to any diet, one can look at home fortification as an alternative. In this method, a commercially available product is conveniently utilised on a daily basis to prevent nutrition deficiencies. Power mixes of rice and flour avaiable in the market is one such example of food fortification, which increases the micronutrient value of home-cooked meals.

Quit SmokingThis is one of the basic requirements. Smoking is a big NO for the overall wellbeing of the body as it negatively impacts on both adaptive and innate immunity. It slowly kills the natural effectiveness of the immune systems defences. If you are a regular smoker, it is advisable to quit it as soon as possible. One can also adopt alternatives like nicotine patches as it can helps to quit smoking and are relatively less harmful.

Rest Your Mind And Soul With MeditationChronic stress is responsible for releasing hormone cortisol, which can ruin the overall immune response of the body. Meditation goes a long way in keeping stress in check. Various mobile-based apps can help one to remain peaceful and mindful throughout the day with various features. Other healthy strategies to deal with stress may include yoga and pranayama.

A minimum of 7-8 hours of regular sleep is a must to maintain a healthy circadian rhythm, and help the bodys T-Cells (also known as immune cells) to kill the pathogens. A night of good sleep also helps in burning calories.

Some of them had Covid, but not all of them, she said. Its the stress of the situation. They were apart from their families. They worked for many hours.

For most patients the condition should be temporary, doctors say, but it could last months.

There are two types of hair loss the pandemic seems to be triggering, experts say.

In one condition, called telogen effluvium, people shed much more than the typical 50 to 100 hairs per day, usually beginning several months after a stressful experience. It essentially involves a shifting or tripping of the hair growth system, said Dr. Sara Hogan, a dermatologist at the David Geffen School of Medicine at the University of California, Los Angeles, who has been seeing up to seven patients a day with the condition.

In healthy hair cycles, most hairs are in a growing phase, with a small percentage in a short resting phase and only about 10% of hairs in a shedding or telogen phase. But with telogen effluvium, people are shedding more, growing less, Khetarpal said, and up to 50% of hair might skip ahead to the shedding phase, with only about 40% in the growth phase.

The phenomenon, which some women also experience after pregnancy, typically lasts about six months, but if stressful situations persist or recur, some people develop a chronic shedding condition, Hogan said.

The other hair loss condition that is increasing now is alopecia areata, in which the immune system attacks hair follicles, usually starting with a patch of hair on the scalp or beard, said Dr. Mohammad Jafferany, a psychiatrist and dermatologist at Central Michigan University.

It is known to be associated with or exacerbated by psychological stress, Jafferany said.

Guttman-Yassky said that she has seen a huge increase in this type of alopecia.

Not all of the patients had Covid-19, she said, but the ones who did tended to progress very quickly from one or two bald patches to losing hair all over the body, including eyebrows and eyelashes. She said that might be because the storm of inflammation that some Covid patients experience elevates immune molecules linked to conditions like alopecia.

Experts dont know exactly why stress triggers these conditions, which affect both women and men. It might be related to increased levels of cortisol, a stress hormone, or to effects on blood supply, Hogan said.

The hair loss itself can cause more stress, Khetarpal said, especially for women, whose hair is often more closely tied to identity and self-confidence.

Its your trademark, said Mary Lou Ostling, 77, a retired educator who lives in the Stuyvesant Town neighborhood of Manhattan. She was hospitalized for Covid-19 for eight days in the early spring and later noticed that my hair started coming out in chunks, she said. I always was clearing hair out of the comb, brush, the sink.

Ostling said she also could tell that her hair wasnt growing much because she wasnt seeing roots that contrasted with the color she had previously dyed it.

Ive always had very long, very thick, very curly hair, she said.

But in July, she had it cut.

I couldnt deal with it anymore, Ostling said.

When she came home from the hairdresser, she said, my husband was just staring at me. He said, I think I have a different wife. It was very depressing. She said she has finally begun to detect some hair growth.

Experts recommend good nutrition, vitamins like biotin and stress-reduction techniques like yoga, scalp massage or mindfulness meditation. Some also recommend minoxidil, a hair growth drug, but Hogan warns patients that it can initially cause more hair loss before it starts working.

With alopecia areata, Guttman-Yassky said, some cases resolve without treatment and some are helped by steroid injections, but some can become permanent, especially if not treated early.

For people depressed or traumatized by hair loss, Jafferany recommends psychotherapy but not necessarily medication because some antidepressants and anti-anxiety medications can exacerbate hair loss.

When Liz Weidhorn, 44, of Fair Lawn, New Jersey, who tested positive for Covid-19 in March, noticed members of an online COVID group bemoaning hair loss, she told herself that if it happened to her, I will take it with grace, and Ill get a kick-ass hat, she recalled.

But recently after showering, I looked at my wet hair, and I could see so much scalp, she said, and I couldnt believe how emotional I got. She cried and called her husband in to look at it.

Its really shocking, she said. It got me very sad.

Weidhorn, who writes a blog about baking pastry, started taking biotin, had her mother cut her hair and is considering getting a headband.

Rowe, who managed the front desk for a wellness spa, has gone further.

I tried putting my hair in one of those messy buns, but it looks terrible with the bald spots on the sides, she said. So she got wigs: a really short pixie one, a pageboy one, a long curly one and a strawberry blonde one, she said. Im trying to make the best of it.

Hogan said some patients find the situation so upsetting they avoided washing or brushing their hair because they noticed the hair loss more during those activities. She tells them they shouldnt be afraid of normal grooming.

She added, Patients dont like this when I say this, but they come around to it: Hair is not crucial for your survival.

Original post:
Losing hair is turning out to be another consequence of the pandemic, not just for Covid-19 patients but also those who haven't caught the virus -...

Not all of the patients had Covid-19, she said, but the ones who did tended to progress very quickly from one or two bald patches to losing hair all over the body, including eyebrows and eyelashes. She said that might be because the storm of inflammation that some Covid patients experience elevates immune molecules linked to conditions like alopecia.

Experts dont know exactly why stress triggers these conditions, which affect both women and men. It might be related to increased levels of cortisol, a stress hormone, or to effects on blood supply, Dr. Hogan said.

The hair loss itself can cause more stress, Dr. Khetarpal said, especially for women, whose hair is often more closely tied to identity and self-confidence.

Its your trademark, said Mary Lou Ostling, 77, a retired educator who lives in the Stuyvesant Town neighborhood of Manhattan. She was hospitalized for Covid-19 for eight days in the early spring and later noticed that my hair started coming out in chunks, she said. I always was clearing hair out of the comb, brush, the sink.

Ms. Ostling said she also could tell that her hair wasnt growing much because she wasnt seeing roots that contrasted with the color she had previously dyed it.

Ive always had very long, very thick, very curly hair, she said. But in July, I simply had a lot of it all cut off. I couldnt deal with it anymore.

When she came home from the hairdresser, she said, my husband was just staring at me. He said, I think I have a different wife. It was very depressing. She said she has finally begun to detect some hair growth.

See the article here:
Losing Your Hair Can Be Another Consequence of the Pandemic - The New York Times

The Global Blood Testing Market analysis report published on Dataintelo.com is a detailed study of market size, share and dynamics covered in XX pages and is an illustrative sample demonstrating market trends. This is a latest report, covering the current COVID-19 impact on the market. The pandemic of Coronavirus (COVID-19) has affected every aspect of life globally. This has brought along several changes in market conditions. The rapidly changing market scenario and initial and future assessment of the impact is covered in the report. It covers the entire market with an in-depth study on revenue growth and profitability. The report also delivers on key players along with strategic standpoint pertaining to price and promotion.

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Global Blood Testing Market Size & Share, by ProductsGlucoseLipidBUNA1CCRPVitamin DALTASTThyroid Stimulating Hormone

Global Blood Testing Market Size & Share, ApplicationsHospitalsClinic and Diagnostic CentersOthers

Key PlayersAbbott LaboratoriesBio-Rad LaboratoriesF. Hoffmann La RocheSiemens Healthineers

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Blood Testing Market 2020 Global Share, Growth, Size, Opportunities, Trends, Regional Overview, Leading Company Analysis And Forecast To 2026 |...

Culture-adapted AAV2 is a viral vector which is used to deliver gene therapy to the liver. However, clinical trials targeting diseases of the liver have had an 'unexpectedly low success rate' using the vector, according to researchers: who have now found that naturally occurring AAVs may be more effective.

The prototypical AAV2 discovered more than 50 years ago provides the serotype on which the field of AAV vectorology and gene therapy is based. The researchers from Australias Childrens Medical Research Institute (CMRI) say the discovery 'will shake the foundations of the field of AAV-based gene therapeutics and will mark the beginning of a new era not only for biomedical research, but most importantly, for millions of patients affected by genetic disorders'.

One area of interest in gene therapy is using AAVs to target the liver, which is involved in genetic disorders such as haemophilia and various enzyme deficiencies.

AAV2 is a viral vector used to deliver gene therapy to the liver, carrying therapeutic DNA to target cells in the body. It binds to a receptor on the target cell. However, the researchers found that while AAV2 binds to the attachment receptors - heparan sulfate proteoglycans (HSPCs) - it does so too tightly.

This means that the vector can get trapped on other cells in the body and not the target liver cells. This reduces the number of vectors that deliver their therapeutic cargo to the liver, diminishing therapeutic efficacy.

The teams of Dr Leszek Lisowski, Head of the Translational Vectorology Research Unit, and Prof Ian Alexander, Head of the Gene Therapy Research Unit, then turned to naturally occurring vectors isolated from liver samples. They found that these which use an as of yet unknown receptor are much more successful at delivering therapies to the liver.

CMRI researchers are now able to make vectors in the lab that use this better receptor, instead of HSPGs, potentially making the next generation of gene therapy targeting the liver 'vastly more successful'.

Theorizing that manufacturing methods could be playing a role, the researcherscompared traditional AAV vectors grown in culture with naturally occurring vectors that they isolated from liver samples. They observed that the cultured vectors rapidly mutated as they replicated in the lab: with these changes making the vectors bind more tightly to molecules called HSPGs on the surface of liver cells, but also impeding their ability to infect humanized liver cells in mice.

In contrast, the naturally occurring vectors infected liver cells more efficiently and bound less tightly to HSPGs, although these effects disappeared when the scientists grew the natural vectors in culture over time.

This really challenges a basic concept in our field that binding strongly to HSPG was essential for AAVs' entry into human cells and suggests that vectors targeting the other receptor used by natural AAVs, of human liver origin, are likely to be more effective for clinical gene therapy applications, said Dr Lisowski. The prototypical AAV2, discovered over 50yrs ago, is the serotype on which the entire field of AAV vectorology and gene therapy is based.

Our study sheds new light and challenges our previous understanding and corrects misconceptions about how the vector binds to the cells.

Researchers at the CMRI can now start to improve on the use of vectors to help children with liver conditions. A better vector can increase safety and improve efficiency, while the increased therapeutic efficacy will mean lower doses are needed and thus reduce the cost of treatment.

The insights on adeno-associated virus receptor binding can potentially be extended to other tissues beyond the liver, add researchers. This makes this a very impactful study which will change the trajectory of AAV-based gene therapies.

Adeno-associated viruses (AAVs) were discovered in the 1960s. The vectorization of AAV2, a human isolate, in 1984 set in motion the development the use of the viral vector in gene therapy.

The liver is a key target for developing more efficient AAV vector delivery, given its direct involvement in a number of genetic and acquired diseases.

Source: Science Translational Medicine, September 9, 2020.DOI: 10.1126/scitranslmed.aba3312

Title: Restoring the natural tropism of AAV2 vectors for human liver

Authors: M. Cabanes-Creus; C.V. Hallwirth; A. Westhaus; B.H. Ng; S.H.Y. Liao; E. Zhu; R.G. Navarro; G. Baltazar; M. Drouyer; S. Scott; G.J. Logan; S.L. Ginn; I.E. Alexander; L. Lisowski at University of Sydney in Westmead, NSW, Australia; C.V. Hallwirth; S. Scott; G.J. Logan; S.L. Ginn; I.E. Alexander at Sydney Children's Hospitals Network in Westmead, NSW, Australia; A. Westhaus; G. Santilli; A.J. Thrasher at University College London in London, UK.

Read more:
Researchers explore naturally occurring viral vectors: 'Our study will change the trajectory of AAV-based gene therapies' - BioPharma-Reporter.com

-- Continued functional improvements were observed at 18 months in the low-dose cohort --

-- First look at functional outcomes in high-dose cohort found improvements 6 months after administration --

-- Results in both cohorts continue to reinforce safety and tolerability profile of SRP-9003 --

CAMBRIDGE, Mass., Sept. 28, 2020 (GLOBE NEWSWIRE) -- Sarepta Therapeutics, Inc.(NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, today announced positive results from the ongoing study of SRP-9003 (rAAVrh74.MHCK7.hSGCB), the Companys investigational gene therapy for limb-girdle muscular dystrophy Type 2E (LGMD2E). Results included 18-month functional data from three clinical trial participants in the low-dose cohort and 6-month functional data from three participants in the high-dose cohort. SRP-9003 is in development for the treatment of LGMD2E (also known as beta-sarcoglycanopathy and LGMDR4), a devastating monogenic neuromuscular disease caused by a lack of beta-sarcoglycan proteins. SRP-9003 is a gene construct that transduces skeletal and cardiac muscle, delivering a gene that codes for the full-length beta-sarcoglycan protein, the absence of which is the sole cause of the progressive degeneration and a shortened lifespan characterized by the disease.

There are currently no approved treatments for people with LGMD2E a disease that causes significant disability in children and often leads to early mortality. Its very encouraging that we continue to see consistent, positive data from our investigational gene therapy SRP-9003 across several measures, as we know the community needs more options, said Louise Rodino-Klapac, Ph.D., senior vice president of gene therapy, Sarepta Therapeutics. The improvements in functional measures at 18- and 6- months in participants from both cohorts who received SRP-9003 are distinctly different from what an age-matched, natural history group would predict with LGMD2E. This sustained durability of the response in functional outcomes reinforces that SRP-9003 is getting to the muscle and suggestive of improvement against disease-mediated muscle damage. When coupled with the strong expression results and encouraging safety profile seen to date, todays results increase our confidence in the construct and provide additional evidence as we advance the higher dose of SRP-9003 into the next stage of clinical testing.

Efficient transduction in skeletal muscle and robust beta-sarcoglycan protein expression were seen in both dose cohorts following infusion with SRP-9003, and significant creatine kinase (CK) reductions were observed at 90 days. Cohort-specific results as follows:

Cohort 1 (low dose), at 18 months:

Cohort 2 (high dose), at 6 months:

About SRP-9003 and the studySRP-9003 uses the AAVrh74 vector, which is designed to be systemically and robustly delivered to skeletal, diaphragm and cardiac muscle, making it an ideal candidate to treat peripheral neuromuscular diseases. AAVrh74 has lower immunogenicity rates than reported with other human AAV vectors. The MHCK7 promoter has been chosen for its ability to robustly express in the heart, which is critically important for patients with limb-girdle muscular dystrophy Type 2E (LGMD2E), also known as beta-sarcoglycanopathy and LGMDR4, many of whom die from pulmonary or cardiac complications.

This first-in-human study is evaluating a single intravenous infusion of SRP-9003 among children with LGMD2E between the ages of 4 and 15 years with significant symptoms of disease. The SRP-9003 study has two cohorts, each studying a different dose-per-kilogram based on the weight of the patient. Three participants in the low-dose cohort (Cohort 1) were treated with a one-time infusion of SRP-9003 dosed at 5x1013vg/kg and an additional three participants in the high-dose cohort (Cohort 2) received a one-time infusion dosed at 2x1014vg/kg. The six participants were between the ages of 4 and 13. Post-treatment biopsies were taken at 60 days.

Sarepta has exclusive rights to the LGMD2E gene therapy program initially developed at the Abigail Wexner Research Institute at Nationwide Childrens Hospital.

About Limb-Girdle Muscular DystrophyLimb-girdle muscular dystrophies are genetic diseases that cause progressive, debilitating weakness and wasting that begin in muscles around the hips and shoulders before progressing to muscles in the arms and legs.

Patients with limb-girdle muscular dystrophy Type 2E (LGMD2E) begin showing neuromuscular symptoms such as difficulty running, jumping and climbing stairs before age 10. The disease, which is an autosomal recessive subtype of LGMD, progresses to loss of ambulation in the teen years and often leads to early mortality. There is currently no treatment or cure for LGMD2E.

Sarepta has five LGMD gene therapy programs in development, including subtypes for LGMD2E, LGMD2D, LGMD2C, LGMD2B and LGMD2L, and holds an option for a sixth program for LGMD2A.

AboutSarepta TherapeuticsAt Sarepta, we are leading a revolution in precision genetic medicine and every day is an opportunity to change the lives of people living with rare disease. The Company has built an impressive position in Duchenne muscular dystrophy (DMD) and in gene therapies for limb-girdle muscular dystrophies (LGMDs), mucopolysaccharidosis type IIIA, Charcot-Marie-Tooth (CMT), and other CNS-related disorders, with more than 40 programs in various stages of development. The Companys programs and research focus span several therapeutic modalities, including RNA, gene therapy and gene editing. For more information, please visitwww.sarepta.comor follow us onTwitter,LinkedIn,InstagramandFacebook.

Forward-Looking StatementsThis press release contains "forward-looking statements." Any statements contained in this press release that are not statements of historical fact may be deemed to be forward-looking statements. Words such as "believes," "anticipates," "plans," "expects," "will," "intends," "potential," "possible" and similar expressions are intended to identify forward-looking statements. These forward-looking statements include statements regarding future clinical testing for SRP-9003, SRP-9003 being the ideal candidate to treat peripheral neuromuscular diseases, the potential benefits of SRP-9003 and potential market opportunities.

These forward-looking statements involve risks and uncertainties, many of which are beyond our control. Known risk factors include, among others: success in preclinical trials and clinical trials, especially if based on a small patient sample, does not ensure that later clinical trials will be successful; the data presented in this release may not be consistent with the final data set and analysis thereof or result in a safe or effective treatment benefit; different methodologies, assumptions and applications we utilize to assess particular safety or efficacy parameters may yield different statistical results, and even if we believe the data collected from clinical trials of our product candidates are positive, these data may not be sufficient to support approval by the FDA or foreign regulatory authorities; if the actual number of patients suffering from LGMD is smaller than estimated, our revenue and ability to achieve profitability may be adversely affected; we may not be able to execute on our business plans and goals, including meeting our expected or planned regulatory milestones and timelines, clinical development plans, and bringing our product candidates to market, due to a variety of reasons, some of which may be outside of our control, including possible limitations of company financial and other resources, manufacturing limitations that may not be anticipated or resolved for in a timely manner, regulatory, court or agency decisions, such as decisions by the United States Patent and Trademark Office with respect to patents that cover our product candidates and the COVID-19 pandemic; and even if Sareptas programs result in new commercialized products, Sarepta may not achieve the expected revenues from the sale of such products; and those risks identified under the heading Risk Factors in Sareptas most recent Annual Report on Form 10-K for the year ended December 31, 2019, and most recent Quarterly Report on Form 10-Q filed with the Securities and Exchange Commission (SEC) as well as other SEC filings made by the Company which you are encouraged to review.

Any of the foregoing risks could materially and adversely affect the Companys business, results of operations and the trading price of Sareptas common stock. For a detailed description of risks and uncertainties Sarepta faces, you are encouraged to review the SEC filings made by Sarepta. We caution investors not to place considerable reliance on the forward-looking statements contained in this press release. Sarepta does not undertake any obligation to publicly update its forward-looking statements based on events or circumstances after the date hereof.

InternetPosting of InformationWe routinely post information that may be important to investors in the 'For Investors' section of our website atwww.sarepta.com.Weencourageinvestorsandpotentialinvestorsto consult our website regularly for important information about us.

Source:Sarepta Therapeutics, Inc.

Sarepta Therapeutics, Inc.

Investors:Ian Estepan, 617-274-4052iestepan@sarepta.com

Media:Tracy Sorrentino, 617-301-8566tsorrentino@sarepta.com

The rest is here:
Sarepta Therapeutics Investigational Gene Therapy SRP-9003 for the Treatment of Limb-Girdle Muscular Dystrophy Type 2E Shows Sustained Functional...

BOSTON and LONDON, Sept. 28, 2020 (GLOBE NEWSWIRE) -- Orchard Therapeutics (Nasdaq: ORTX), a global gene therapy leader, today announced that the European Medicines Agency (EMA) has granted Priority Medicines (PRIME) designation to OTL-203, an investigational ex vivo autologous hematopoietic stem cell (HSC) gene therapy in development for the treatment of mucopolysaccharidosis type I (MPS-I) at the San Raffaele Telethon Institute for Gene Therapy (SR-Tiget) in Milan, Italy.

We are encouraged by EMAs decision to grant PRIME designation to OTL-203, which was based on an initial clinical assessment of data supporting the potential benefit of our HSC gene therapy for patients with MPS-IH beyond the current standard of care, said Anne Dupraz-Poiseau, PhD., chief regulatory officer of Orchard. In 2021, we look forward to building upon the promising early data in the ongoing proof-of-concept study and plan to initiate a registrational trial to advance a potential new treatment for patients.

The PRIME program is designed to enhance regulatory support in the EU for the development of promising investigational medicines that, based on early clinical data, may offer a major therapeutic advantage over existing treatments or benefit patients without treatment options. PRIME aims to provide multiple benefits so that these medicines can reach patients earlier: enhanced interaction and early dialogue with EMA, guidance on the overall development plan and regulatory strategy, and the potential for accelerated assessment of the marketing authorization application. For more information please visit the EMA website at http://www.ema.europa.eu.

Additional interim data was recently presented from the ongoing proof-of-concept clinical trial evaluating the safety and efficacy of OTL-203 in the severe Hurler subtype of MPS-I. Eight patients have been treated in the study, which completed enrollment in December 2019. As of July 2020, all patients had been followed for a minimum of six months, with the longest follow-up extending out to 24 months. Orchard expects to release full proof-of-concept results at one year and initiate a registrational study for OTL-203 in 2021.

About OTL-203 and MPS-I

Mucopolysaccharidosis type I (MPS-I) is a rare, inherited neurometabolic disease caused by a deficiency of the alpha-L-iduronidase (IDUA) lysosomal enzyme, which is required to break down sugar molecules called glycosaminoglycans (also known as GAGs). The accumulation of GAGs across multiple organ systems results in symptoms including neurocognitive impairment, skeletal deformity, loss of vision and hearing, and cardiovascular and pulmonary complications. MPS-I occurs at an overall estimated frequency of one in every 100,000 live births. There are three subtypes of MPS-I; approximately 60 percent of children born with MPS-I have the most severe subtype, called Hurler syndrome, and rarely live past the age of 10 when untreated.Treatment options for MPS-I include hematopoietic stem cell transplant and chronic enzyme replacement therapy, both of which have significant limitations. Though early intervention with enzyme replacement therapy has been shown to delay or prevent some clinical features of the condition, it has only limited efficacy on neurological symptoms. OTL-203 is an investigational ex vivo autologous hematopoietic stem cell gene therapy being studied for the treatment of MPS-I. Orchard was granted an exclusive worldwide license to intellectual property rights to research, develop, manufacture and commercialize the gene therapy program for the treatment of MPS-I developed by the San Raffaele Telethon Institute for Gene Therapy in Milan, Italy.

About Orchard

Orchard Therapeutics is a global gene therapy leader dedicated to transforming the lives of people affected by rare diseases through the development of innovative, potentially curative gene therapies. Our ex vivo autologous gene therapy approach harnesses the power of genetically modified blood stem cells and seeks to correct the underlying cause of disease in a single administration. In 2018, Orchard acquired GSKs rare disease gene therapy portfolio, which originated from a pioneering collaboration between GSK and the San Raffaele Telethon Institute for Gene Therapy in Milan, Italy. Orchard now has one of the deepest and most advanced gene therapy product candidate pipelines in the industry spanning multiple therapeutic areas where the disease burden on children, families and caregivers is immense and current treatment options are limited or do not exist.

Orchard has its global headquarters in London and U.S. headquarters in Boston. For more information, please visit http://www.orchard-tx.com, and follow us on Twitter and LinkedIn.

Availability of Other Information About Orchard

Investors and others should note that Orchard communicates with its investors and the public using the company website (www.orchard-tx.com), the investor relations website (ir.orchard-tx.com), and on social media (Twitter and LinkedIn), including but not limited to investor presentations and investor fact sheets, U.S. Securities and Exchange Commission filings, press releases, public conference calls and webcasts. The information that Orchard posts on these channels and websites could be deemed to be material information. As a result, Orchard encourages investors, the media, and others interested in Orchard to review the information that is posted on these channels, including the investor relations website, on a regular basis. This list of channels may be updated from time to time on Orchards investor relations website and may include additional social media channels. The contents of Orchards website or these channels, or any other website that may be accessed from its website or these channels, shall not be deemed incorporated by reference in any filing under the Securities Act of 1933.

Forward-Looking Statements

This press release contains certain forward-looking statements about Orchards strategy, future plans and prospects, which are made pursuant to the safe harbor provisions of the Private Securities Litigation Reform Act of 1995. Such forward-looking statements may be identified by words such as anticipates, believes, expects, plans, intends, projects, and future or similar expressions that are intended to identify forward-looking statements. Forward-looking statements include express or implied statements relating to, among other things, Orchards business strategy and goals, the therapeutic potential of Orchards product candidates, including the product candidate referred to in this release, Orchards expectations regarding the timing of clinical trials for its product candidates, including the product candidate referred to in this release, the timing of interactions with regulators and regulatory submissions related to ongoing and new clinical trials for its product candidates, the timing of announcement of clinical data for its product candidates, and the likelihood that such data will be positive and support further clinical development and regulatory approval of these product candidates. These statements are neither promises nor guarantees and are subject to a variety of risks and uncertainties, many of which are beyond Orchards control, which could cause actual results to differ materially from those contemplated in these forward-looking statements. In particular, these risks and uncertainties include, without limitation: the severity of the impact of the COVID-19 pandemic on Orchards business, including on clinical development, its supply chain and commercial programs; the risk that Orchard will not realize the anticipated benefits of its new strategic plan or the expected cash savings associated with such plan; the risk that any one or more of Orchards product candidates, including the product candidate referred to in this release, will not be successfully developed, approved or commercialized; the risk of cessation or delay of any of Orchards ongoing or planned clinical trials; the risk that Orchard may not successfully recruit or enroll a sufficient number of patients for its clinical trials; the risk that prior results, such as signals of safety, activity or durability of effect, observed from preclinical studies or clinical trials will not be replicated or will not continue in ongoing or future studies or trials involving Orchards product candidates or that long-term adverse safety findings may be discovered; the delay of any of Orchards regulatory submissions; the failure to obtain marketing approval from the applicable regulatory authorities for any of Orchards product candidates or the receipt of restricted marketing approvals; and the risk of delays in Orchards ability to commercialize its product candidates, if approved. Given these uncertainties, the reader is advised not to place any undue reliance on such forward-looking statements.

Other risks and uncertainties faced by Orchard include those identified under the heading "Risk Factors" in Orchards quarterly report on Form 10-Q for the quarter ended June 30, 2020, as filed with the U.S. Securities and Exchange Commission (SEC), as well as subsequent filings and reports filed with the SEC. The forward-looking statements contained in this press release reflect Orchards views as of the date hereof, and Orchard does not assume and specifically disclaims any obligation to publicly update or revise any forward-looking statements, whether as a result of new information, future events or otherwise, except as may be required by law.

Contacts

InvestorsRenee LeckDirector, Investor Relations+1 862-242-0764Renee.Leck@orchard-tx.com

MediaMolly CameronManager, Corporate Communications+1 978-339-3378media@orchard-tx.com

Excerpt from:
Orchard Therapeutics Receives EMA PRIME Designation for OTL-203 for the Treatment of MPS-I - BioSpace

Watch here: https://www.youtube.com/watch?feature=youtu.be&v=VVkQU91KbEs

press release: The UW has a long history of pioneering medical advancements that have transformed the world. From performing the first bone marrow transplant in the United States to cultivating the first laboratory-derived human embryonic stem cells. Now, where will UW medical research go next?

On the next Wisconsin Medicine Livestream, meet trailblazing doctors, researchers, and medical leaders who are charting a bold course to completely alter the health care landscape. During this insightful panel discussion, well explore how gene therapy and cell replacements could hold the keys to treating inherited and acquired blindness. Youll also discover the remarkable potential in xenotransplantation where nonhuman animal source organs are transplanted into human recipients. In addition, you will learn about UW Healths journey to build a multidisciplinary program to serve the community. These, and other, fascinating developments in treatment and care are happening right now at the UW and are the future of medicine. The presentation will be moderated by Robert Golden, the dean of the University of WisconsinMadisons School of Medicine and Public Health.

Our Guests:

David Gamm, professor, Department of Ophthalmology and Visual Sciences; Emmett A. Humble Distinguished Director, McPherson Eye Research Institute; Sandra Lemke Trout Chair in Eye Research

Dr. Gamms lab is at the forefront in developing cell-based therapies to combat retinal degenerative diseases (RDDs). As the director of the McPherson Eye Research Institute and a member of the Waisman Center Stem Cell Research Program, the UW Stem Cell and Regenerative Medicine Center, and the American Society for Clinical Investigation, his efforts are directed toward basic and translational retinal stem cell research. The Gamm Lab uses induced pluripotent stem cells to create retinal tissues composed of authentic human photoreceptor cells rods and cones that can detect light and initiate visual signals in a dish. The aims of his laboratory are to investigate the cellular and molecular events that occur during human retinal development and to generate cells for use in retinal disease modeling and cell replacement therapies. In collaboration with other researchers at UWMadison and around the world, the lab is developing methods to produce and transplant photoreceptors and/or retinal pigment epithelium (RPE) in preparation for future clinical trials. At the same time, the Gamm Lab uses lab-grown photoreceptor and RPE cells to test and advance a host of other experimental treatments, including gene therapies. In so doing, the lab seeks to delay or reverse the effects of blinding disorders, such as retinitis pigmentosa and age-related macular degeneration, and to develop or codevelop effective interventions for these RDDs at all stages of disease.

Dhanansayan Shanmuganayagam, assistant professor, Department of Surgery, School of Medicine and Public Health; Department of Animal and Dairy Sciences, UWMadison; director, Biomedical, and Genomic Research Group

Dr. Shanmuganayagams research focuses on the development and utilization of pigs as homologous models to close the translational gap in human disease research, taking advantage of the overwhelming similarities between pigs and humans in terms of genetics, anatomy, physiology, and immunology. He and his colleagues created the human-sized Wisconsin Miniature Swine breed that is unique to the university. The breed exhibits greater physiological similarity to humans, particularly in vascular biology and in modeling metabolic disorders and obesity. He currently leads genetic engineering of swine at the UW. His team has created more than 15 genetic porcine models including several of pediatric genetic cancer-predisposition disorders such as neurofibromatosis type 1 (NF1). In the context of NF1, his lab is studying the role of alternative splicing of the nf1 gene on the tissue-specific function of neurofibromin and whether gene therapy to modulate the regulation of this splicing can be used as a viable treatment strategy for children with the disorder.

Dr. Shanmuganayagam is also currently leading the efforts to establish the University of Wisconsin Center for Biomedical Swine Research and Innovation (CBSRI) that will leverage the translatability of research in pig models and UWMadisons unique swine and biomedical research infrastructure, resources, and expertise to conduct innovative basic and translational research on human diseases. The central mission of CBSRI is to innovate and accelerate the discovery and development of clinically relevant therapies and technologies. The center will also serve to innovate graduate and medical training. As the only center of its kind in the United States, CBSRI will make UWMadison a hub of translational research and industry-partnered biomedical innovation.

Petros Anagnostopoulos, surgeon in chief, American Family Childrens Hospital; chief, Section of Pediatric Cardiothoracic Surgery; professor, Department of Surgery, Division of Cardiothoracic Surgery

Dr. Anagnostopoulos is certified by the American Board of Thoracic Surgery and the American Board of Surgery. He completed two fellowships, one in cardiothoracic surgery at the University of Pittsburgh School of Medicine and a second in pediatric cardiac surgery at the University of California, San Francisco School of Medicine. He completed his general surgery residency at Henry Ford Hospital in Detroit. Dr. Anagnostopoulos received his MD from the University of Athens Medical School, Greece. His clinical interests include pediatric congenital heart surgery and minimally invasive heart surgery.

Dr. Anagnostopoulos specializes in complex neonatal and infant cardiac reconstructive surgery, pediatric heart surgery, adult congenital cardiac surgery, single ventricle palliation, extracorporeal life support, extracorporeal membrane oxygenation, ventricular assist devices, minimally invasive cardiac surgery, hybrid surgical-catheterization cardiac surgery, off-pump cardiac surgery, complex mitral and tricuspid valve repair, aortic root surgery, tetralogy of Fallot, coronary artery anomalies, Ross operations, obstructive cardiomyopathy, and heart transplantation.

When: Tuesday, Sept. 29, at 7 p.m. CDT

Where: Wisconsin Medicine Livestream: wiscmedicine.org/programs/ending-alzheimers

Read more here:
ONLINE: The Future of Medicine - Isthmus

NEW YORK, Sept. 23, 2020 /PRNewswire/ --Seelos Therapeutics, Inc. (Nasdaq: SEEL), a clinical-stage biopharmaceutical company focused on the development of therapies for central nervous system disorders and rare diseases, announced today the signing of a Sponsored Research Agreement (SRA) with Duke University to use the MPTP-induced Parkinson's Disease (PD) mouse model to establish in vivo proof-of-concept study to demonstrate that administration of LV-dCas9-DNMT3A virus can prevent and/or delay PD and test the efficacy and safety of SLS-004. Subsequently, other pre-clinical models would be utilized to further validate the investigational product.

"We are extremely pleased to begin the in vivo target engagement study with SLS-004 at Duke as it builds onto the work we began in the spring focused on designing a vector capable of inducing and suppressing Parkinson's related phenotypes," said Raj Mehra Ph.D., Chairman and CEO of Seelos. "Initiating this next portion of the studies at Duke should help us further validate this approach in Parkinson's."

Seelos has also begun work on designing a vector capable of inducing and suppressing PD-related phenotypes by carrying a unit to overexpress the alpha-synuclein (-synuclein) protein and a unit to mediate inducible suppression of -synuclein. The process aims to create a next-generation suppressive unit, carrying a more effective effector molecule than previously used, in the form of DNA methyltransferase 3A and 3L (DNMT3A & L). This research may help to advance a potential novel, effective and precise tool for reversing SNCA pathologies that can provide a valuable new therapeutic strategy for treating PD.

About SLS-004

SLS-004 is a novel epigenome-editing approach to modulate expression of SNCA gene mediated by modification of DNA-methylation. SLS-004 utilizes an all-in-one lentiviral vector harboring dCas9-DNA methyltransferase 3A (DNMT3A) to enrich DNA-methylation within CpGs island at the SNCA intron 1 region. The system resulted in a precise and fine-tuned downregulation (30%) of SNCA overexpression in hiPSC-derived dopaminergic neurons from a PD patient with the triplication of the SNCA locus (SNCA-Tri). Most importantly, the reduction of SNCA expression mediated by the developed system was sufficient to ameliorate disease related cellular phenotypes. The in vitro studies achieved several key millstones including the establishment that DNA hypermethylation at SNCA intron 1 allows an effective and sufficient tight downregulation of SNCA expression levels and suggests the potential of this target sequence combined with the CRISPR-dCas9 technology as a novel epigenetic-based therapeutic approach for PD.

Forward Looking Statements

Statements made in this press release, which are not historical in nature, constitute forward-looking statements for purposes of the safe harbor provided by the Private Securities Litigation Reform Act of 1995. These statements include, among others, those regarding the initiation of a proof-of-concept study to demonstrate whether administration of the LV-dCas9-DNMT3A virus can prevent and/or delay PD, the potential for the LV-dCas9-DNMT3A virus to prevent and/or delay PD, the efficacy and safety of SLS-004, the potential use of other pre-clinical models to validate SLS-004, the potential for the in vivo proof-of-concept study to validate the design of a vector capable of inducing and suppressing PD-related phenotypes, and the potential for Seelos' research to advance a novel, effective and precise tool for reversing SNCA pathologies. These statements are based on Seelos' current expectations and beliefs and are subject to a number of factors and uncertainties that could cause actual results to differ materially from those described in the forward-looking statements. Risks associated with Seelos' business include, but are not limited to, the risk of not successfully executing its preclinical and clinical studies and not gaining marketing approvals for its product candidates, the risk that prior test results may not be replicated in future studies and trials, the risks that clinical study results may not meet any or all endpoints of a clinical study and that any data generated from such studies may not support a regulatory submission or approval, the risks associated with the implementation of a new business strategy, the risks related to raising capital to fund its development plans and ongoing operations, risks related to Seelos' current stock price, risks related to the global impact of COVID-19, as well as other factors expressed in Seelos' periodic filings with the U.S. Securities and Exchange Commission, including its Annual Report on Form 10-K and Quarterly Reports on Form 10-Q. Although we believe that the expectations reflected in our forward-looking statements are reasonable, we do not know whether our expectations will prove correct. You are cautioned not to place undue reliance on these forward-looking statements, which speak only as of the date hereof, even if subsequently made available by us on our website or otherwise. We do not undertake any obligation to update, amend or clarify these forward-looking statements, whether as a result of new information, future events or otherwise, except as may be required under applicable securities laws.

Contact Information:Anthony MarcianoHead of Corporate CommunicationsSeelos Therapeutics, Inc. (Nasdaq: SEEL)300 Park Ave., 12th FlNew York, NY 10022(646) 293-2136anthony.marciano@seelostx.comwww.seelostherapeutics.comhttps://twitter.com/seelostxhttps://www.linkedin.com/company/seelos

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Seelos Therapeutics Announces Sponsored Research Agreement with Duke University for Gene Therapy Studies of SLS-004 in Parkinson's Disease - WFMZ...

Sept. 28, 2020 11:00 UTC

CAMBRIDGE, Mass.--(BUSINESS WIRE)-- AVROBIO, Inc. (Nasdaq: AVRO), a leading clinical-stage gene therapy company with a mission to free people from a lifetime of genetic disease, today announced that the European Commission (EC) has granted orphan drug designation for AVR-RD-02, the companys investigational gene therapy for the treatment of Gaucher disease. AVR-RD-02 consists of the patients own hematopoietic stem cells, genetically modified to express glucocerebrosidase (GCase), the enzyme that is deficient in Gaucher disease. AVROBIO recently dosed the first patient in the GuardOne Phase 1/2 clinical trial to evaluate the safety and efficacy of AVR-RD-02.

Like many lysosomal disorders, Gaucher disease can lead to debilitating complications throughout the body and brain. The standard of care does not address all these symptoms and may not be able to halt progression of the disease, said Geoff MacKay, AVROBIOs president and CEO. Our investigational gene therapy is designed to address the head-to-toe manifestations of Gaucher disease with a single dose. Were pleased to receive orphan drug designation, which recognizes the potential of our approach to transform the standard of care and, we hope, the quality of life for people living with this rare genetic disorder.

The EC grants orphan drug designation to drugs and biologics intended for the safe and effective treatment, diagnosis or prevention of rare diseases or conditions that impact fewer than 5 in 10,000 patients in the European Union. Orphan drug designation gives companies certain benefits, including reduced regulatory fees, clinical protocol assistance, research grants and 10 years of market exclusivity following regulatory approval.

AVR-RD-02 has also received orphan drug designation from the U.S. Food and Drug Administration.

About Gaucher Disease

Gaucher disease is a rare, inherited lysosomal storage disorder characterized by the toxic accumulation of glucosylceramide (GlcCer) and glucosylsphingosine (GlcSph) in macrophages. Macrophages bloated with these fatty substances are called Gaucher cells which amass primarily in the spleen, liver and bone marrow. This results in a variety of potential symptoms, including grossly enlarged liver and spleen, bone issues, fatigue, low hemoglobin levels and platelet counts and an adjusted lifetime relative risk of developing Parkinson's disease that may be more than 20 times greater than the general population. Even on enzyme replacement therapy (ERT) the current standard of care people with Gaucher disease type 1 typically have a shortened life expectancy and may experience debilitating symptoms that significantly reduce their quality of life. An estimated 1 in 44,000 people are diagnosed with Gaucher disease.

About AVR-RD-02

AVR-RD-02 is an investigational lentiviral gene therapy designed to provide a durable therapeutic benefit for people living with Gaucher disease. The therapy starts with the patients own hematopoietic stem cells, which are genetically modified to express functional glucocerebrosidase (GCase). Functional GCase reduces levels of glucosylceramide and glucosylsphingosine, the accumulated substances which cause the symptoms of Gaucher disease. AVROBIO is currently evaluating AVR-RD-02 in GuardOne, a Phase 1/2 clinical trial.

About lentiviral gene therapy

Lentiviral vectors are differentiated from other delivery mechanisms because of their large cargo capacity and their ability to integrate the therapeutic gene directly into the patients chromosomes. This integration is designed to maintain the therapeutic genes presence as the patients cells divide, which potentially enables dosing of pediatric patients, whose cells divide rapidly as they grow. Because the therapeutic gene is integrated into the patients own stem cells, patients are not excluded from receiving the investigational therapy due to pre-existing antibodies to the vector.

About AVROBIO

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

Forward-Looking Statements

This press release contains forward-looking statements, including statements made pursuant to the safe harbor provisions of the Private Securities Litigation Reform Act of 1995. These statements may be identified by words and phrases such as aims, anticipates, believes, could, designed to, estimates, expects, forecasts, goal, intends, may, plans, possible, potential, seeks, will, and variations of these words and phrases or similar expressions that are intended to identify forward-looking statements. These forward-looking statements include, without limitation, statements regarding our business strategy for and the potential therapeutic benefits of our prospective product candidates, including AVR-RD-02 for the treatment of Gaucher disease; the anticipated benefits of the European Commissions grant of orphan drug designation for AVR-RD-02; the design, commencement, enrollment and timing of ongoing or planned clinical trials and regulatory pathways; the timing of patient recruitment and enrollment activities, clinical trial results, and product approvals; the anticipated benefits of our gene therapy platform including the potential impact on our commercialization activities, timing and likelihood of success; the expected benefits and results of our implementation of the plato platform in our clinical trials and gene therapy programs; and the expected safety profile of our investigational gene therapies. Any such statements in this press release that are not statements of historical fact may be deemed to be forward-looking statements. Results in pre-clinical or early-stage clinical trials may not be indicative of results from later stage or larger scale clinical trials and do not ensure regulatory approval. You should not place undue reliance on these statements, or the scientific data presented.

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

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AVROBIO Receives Orphan Drug Designation from the European Medicines Agency for AVR-RD-02, an Investigational Gene Therapy for Gaucher Disease -...

NEWARK, Del., Sept. 24, 2020 /PRNewswire/ --Neurophth Therapeutics, Inc., (hereinafter referred to as "Neurophth") today announced that its leading candidate, NR082 (rAAV2-ND4, NFS-01 project), was granted an orphan drug designation (ODD) by the U.S. FDA for the treatment of Leber's Hereditary Optic Neuropathy associated with ND4 mutation.

Leber's Hereditary Optic Neuropathy (LHON) is a maternally inherited mitochondrial disease, characterized by acute or subacute, painless vision loss or even loss simultaneously or sequentially, accompanied by central visual field defect and color vision impairment with poor prognosis. It was first reported by German scholar Leber in 1871. It affects about 1-9:100,000 people worldwide. LHON is one of the blinding diseases. The disease mainly occurs in young- and middle-aged men. Currently, there is no effective treatment for LHON. About 70% - 90% of LHON is caused by ND4 mutation of harboring a point mutation at nucleotide 11778 associated with a G-to-A transition. With the development of NR082, AAV-based gene therapy of LHON becomes possible.

"Due to the lack of effective treatment, the quality of life of LHON patients associated with ND4 mutation is very poor, and a huge unmet medical needs have not been fulfilled," said Dr. Alvin Luk, Chief Executive Officer at Neurophth. "NR082 is the first candidate drug developed by Neurophth. It uses recombinant adeno-associated virus serotype 2 to deliver the genetically modified ND4 gene (rAAV2-ND4). After a single intravitreal injection, the gene is translated and expressed in cells, which effectively supplements the function loss caused by endogenous mutation. Through this gene therapy, the electron transport function of mitochondrial respiratory chain was maintained, and the increase of ATP synthesis restored the normal function of mitochondria, which in turn improved the sensory function of the retinal ganglion cells and improved the visual acuity of LHON patients."

Luk added, "the significance of orphan drug designation is that regulators recognize the unmet medical needs of rare diseases like LHON. The recognition of NR082 will reduce the R&D investment to a certain extent and accelerate the progress of clinical trials and marketing registration. Furthermore, Neurophth is committed to fundamentally solve the causes and change the quality of life of patients through a single treatment of gene therapy."

Professor Bin Li, Founder, Chairman and Chief Scientific Officer at Neurophth, said: "NR082 has been granted as orphan drug by U.S. FDA, which further strengthens our focus on gene therapy for rare ophthalmic diseases, and develops more drugs for treatment of ocular genetic diseases, bringing hope to patients with ocular genetic diseases in the world".

*FDA grants orphan drug designation to drugs and biological products designed to safely and effectively treat, diagnose, or prevent rare diseases or conditions affecting less than 200,000 people in the US. According to the Orphan Drug Act of FDA, Orphan Drug Designation (ODD) provides opportunities for grant funding, fast approval channel, and some incentives, such as waiver of New Drug Application (NDA) fees, tax credits for clinical trial expenses and exemption for prescription drug users' fees as well as the products are entitled to a seven-year of market exclusivity and will not be affected by patents.

About NR082 (rAAV2-ND4; NFS-01 Project)

LHON disease is caused by mutations in mitochondrial DNA 11778, 14484 or 3460. ND4 gene of 11778 G>A mutation is the main pathological factor, which exists in 55-70% of European and American patients and 90% of Chinese patients. NR082 (NFS-01 project) is an innovative candidate drug for ophthalmic AAV-based gene therapy. It uses AAV2 vector to express human ND4 gene in the retinal ganglion cells to repair optic neuropathy caused by 11778 G>A mutation.

As early as 2011, Professor Bin Li's team started the world's first LHON gene therapy investigator-initiated trial (IIT) with this candidate drug. Nine subjects who participated in the clinical trial have been followed up for up to 8 years with no serious adverse reactions, and 5 of them have significant improvement in their vision. This result is the longest follow-up record of gene therapy in the world, which has already been published in the Scientific Report, EBioMedicine and Ophthalmology journals, and has fully proven the long-term safety, effectiveness, and durability of AAV gene therapy in clinical settings.

After the gratifying results of the first study, Professor Li's team conducted a more comprehensive IIT clinical study from 2017 to 2018, with 159 subjects (including 10 subjects from Argentina), which is the largest clinical trial in the entire gene therapy in the world. Among those, 143 of the patients has completed the 12-month follow-up and 56.6% showed a significant BCVA (best-corrected visual acuity improved by at least 0.3 LogMAR) improvement. No serious adverse reaction was found. In May 2020, at the 23rd online annual meeting of the American Society for Gene and Cell Therapy (ASGCT) and the online annual meeting of the Association for Research in Vision and Ophthalmology (ARVO), Neurophth presented these two clinical research data on the treatment of LHON with NR082 (NFS-01 project of rAAV2-ND4), demonstrating the international advanced level of this research in the field of gene therapy.

Following the positive results of these two IIT trials, Neurophth is actively preparing the China/U.S. IND (Investigational New Drug) applications, and plans to carry out the registration clinical Phase 1/2/3 registration trial to evaluate the safety, efficacy and durability of NR082.

About Neurophth

As a clinical-stage R & D company, Neurophth is committed to exploring and developing new therapies for global patients with ophthalmic diseases. With the help of the mature AAV ophthalmic gene therapy technology platform and the deep understanding of the ophthalmology field by the founding team for decades, Neurophth has established a rich, robust product pipeline, including more than 10 research projects for various ophthalmic diseases, such as dominant hereditary optic atrophy, optic nerve injury diseases, vascular retinopathy, etc., and gradually expanded from rare to common ophthalmic diseases. Additionally, the company is preparing to build a GMP commercial production platform for gene therapy drugs accordance with the international quality standards, and plans to build an ophthalmic gene therapy transformational excellence center, aiming to become a global leader in gene therapy in ophthalmology to benefit patients all over the world.

Prospect of Gene Therapy in Ophthalmology

Inherited retinal diseases (IRDs) have long been regarded as an ideal disease area for gene therapy, because most of the gene mutations leading to the disease have been identified (more than 200 gene defects are associated with the most common IRDS). The eye is, to some extent, an immune privilege. Clinical trials have shown that gene therapy using adeno-associated virus (AAV) or lentivirus (LV) vectors in the eye does not cause systemic side effects and does not cause significant immune responses. The most common IRDs were Retinitis Pigmentosa (RP), Achromatopsia color blindness (ACHM), Leber Hereditary Optic Neuropathy (LHON), Leber Congenital Amaurosis (LCA), Stargardt disease and X-linked Retinoschisis (XLRS).

To date, only one ophthalmic AAV gene therapy product has been approved in the world, namely the first AAV2 gene therapy voretigene neparvovec-rzyl (LUXTURNA; Spark Therapeutics) approved by FDA in December 2017 to treat IRD caused by RPE65 double allele mutation in adult or pediatric patients. The approval of LUXTURNAhas brought confidence and hope to the global ophthalmic gene therapy field. Public information disclosed that at least 20-30 kinds of gene therapy for ophthalmic diseases are in the research and development stage, and the international representative companies include Applied Genetic Technologies Corporation and Meira GTX, and new companies represented by Neurophth have also begun to enter the international stage of ophthalmic gene therapy.

References

Contact:Dr. Alvin LukAlvin.Luk@neurophth.com

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Neurophth Therapeutics' Treatment of Leber's Hereditary Optic Neuropathy Gene Therapy NR082 was Granted Orphan Drug Designation by US FDA - BioSpace

SHANGHAI, Sept. 28, 2020 /PRNewswire/ -- On September 25, 2020, BIORAY LABORATORIES Inc. ("Bioraylab"), a company specialized in gene therapy and cell drug R&D, announced for the first time that clinical trials of non-viral PD1 specific targeted CAR T therapy in relapsed/refractory B-cell non-Hodgkin lymphoma, undertaken by the company in cooperation with East China Normal University and the First Affiliated Hospital, School of Medicine, Zhejiang University, has achieved significant breakthroughs. This is the world's first application of gene editing technology to realize PD1-knockin CAR T treatment, and is also the world's first clinical trial treating lymphoma with non-viral PD1 specific targeted CAR T cells.

The latest research results, finished by East China Normal University, the First Affiliated Hospital, School of Medicine, Zhejiang University and Bioraylab, were announced on the preprint platform medRxiv on September 23, 2020.

PD1 specific targeted CD19-CARTis Bioraylab's Quikin CART platform technology using its proprietary IP, precisely inserting CAR cassette into PD1 locus without using virus, thus generating the CAR T product in just one step. The product combines PD1 immune checkpoint inhibition with CART anti-tumor activity, generating effects of both anti-PD1 immunotherapy and CAR T therapy. Several ongoing clinical trials demonstrate the outstanding safety and effectiveness of this CAR T product.

Two patients showed complete remission following a three-month treatment

The clinical trial program enrolled 15 patients, among which, four patients who were able to be evaluated showed partial remission (PR) after receiving the one-month treatment, while two patients showed complete remission (CR) after three months of treatment.

There were no CAR T cell related high-grade (3) adverse eventsduring the entire treatment, including cytokine release syndrome (CRS)and neurologic toxicity. After infusion, the CAR T cells were well sustained in vivo.

The Quikin CART technology produces CAR T cells without using virus, greatly reducing high costs of producing CAR T products, while avoiding the cancer risk from random insertion. The regulation of T cell endogenous genes and the constant expression of CAR are realized in just one step. Compared with other CAR T technologies, Quikin CART has a variety of advantages, including a simpler process, fewer production links, shorter time preparation and higher product uniformity.

Liu Mingyao, chief scientist at Bioraylab and professor at East China Normal University, said, "Without using virus, the Quikin CART technology can realize the precise integration of CAR cassette into genome as well as regulation of T cell endogenous genes in just one step, delivering many unparalleled advantages compared with existing CAR T technologies. The technology offers a strong platform for the development of more diverse CAR T products in the future."

Huang He, the project investigator of this clinical trial and chairman of the First Affiliated Hospital, School of Medicine, Zhejiang University, commented, "The current results showed that the non-viral PD1-knockin CAR T cellsproduced by using the Quikin CART technology have great potential to treat patients. We are very delighted to see patients rapidly show CR after treatment, and are looking forward to safer and more durable long-term effects on relapsed/refractory patients through this novel CAR T technology."

In addition to the ongoingclinical trials of non-viral PD1 specific targeted CD19-CART, Bioraylab is conducting research on other non-viral specific targeted CART products for treating solid tumors, with the aim of achieving more breakthroughs in CAR T therapy.

About Bioraylab

Founded in Shanghai in 2013, BiorayLaboratories Inc.("Bioraylab")is already well positioned to become the world's leading gene cell pharmaceutical manufacturer, by way of innovation with gene editing and the development of breakthrough therapies that benefit all mankind. Bioraylab owns 108 patents, conductedIIT trials for fiveprojects in eightof the world's leadinghospitals, three of which having entered the IND application stage. Bioraylab has, over the past five years, published 12 high-level academic papers in internationally renownedjournals, including Nature Biotechnology and Nature Medicine. The firmhas built three technology platforms for gene editing, cell therapy and gene therapy, and has a 6,000-square-meter GMP pilot plant and an operating team comprising nearly 100 people, placing the firm in the position of being able to guarantee the rapid transformation and application of innovative research results.

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World's first clinical trial of non-viral PD1 specific targeted CAR T therapy achieves great breakthroughs - WFMZ Allentown

Timothy Ray Brown, the first person known to have been cured of HIV infection, says he is now terminally ill from a recurrence of the cancer that prompted his historic treatment 12 years ago.

Brown, dubbed "the Berlin patient" because of where he lived at the time, had a transplant from a donor with a rare, natural resistance to the AIDS virus. For years, that was thought to have cured his leukemia and his HIV infection, and he still shows no signs of HIV.

But in an interview with the Associated Press, Brown said his cancer returned last year and has spread widely. He's receiving hospice care where he now lives in Palm Springs, Calif.

"I'm still glad that I had it," Brown said of his transplant. "It opened up doors that weren't there before" and inspired scientists to work harder to find a cure, which many had begun to think was not possible, the 54-year-old said Sept. 24.

"Timothy proved that HIV can be cured, but that's not what inspires me about him," said Dr. Steven Deeks, an AIDS specialist at the University of California at San Francisco, who has worked with Brown to further research toward a cure.

"We took pieces of his gut, we took pieces of his lymph nodes. Every time he was asked to do something, he showed up with amazing grace," Deeks said.

Brown was an American working as a translator in Berlin in the 1990s when he learned he had HIV. In 2006, he was diagnosed with leukemia.

Dr. Gero Huetter, a blood cancer expert at the University of Berlin, believed that a marrow transplant was Brown's best chance of beating the leukemia. He wondered, could he also cure Brown's other life-threatening disease by using a donor with a gene mutation that provides natural resistance to the AIDS virus?

Donors like these are very rare and transplants are risky. Doctors have to destroy the patient's diseased immune system with chemotherapy and radiation, then transplant the donor's cells and hope they develop into a new immune system for the recipient.

Brown's first transplant in 2007 was only partly successful: His HIV seemed to be gone but his leukemia was not. He had a second transplant from the same donor in March 2008 and that one seemed to work.

Since then, Brown has repeatedly tested negative for HIV and has frequently appeared at AIDS conferences where cure research is discussed.

"He's been like an ambassador of hope," said Brown's partner, Tim Hoeffgen.

A second man, Adam Castillejocalled "the London patient" until he revealed his identity earlier this yearalso is believed to have been cured by a transplant similar to Brown's in 2016.

But donors like these are scarce and the procedure is too risky to be widely used.

Scientists have been testing gene therapy and other ways to try to get the effect of the favorable gene mutation without having to do a transplant. At an AIDS conference in July, researchers said they may have achieved a long-term remission in a Brazil man by using a powerful combination of drugs meant to flush dormant HIV from his body.

Mark King, a Baltimore man who writes a blog for people with HIV, said he spoke with Brown recently and is grateful for what Brown has contributed to AIDS research.

"It is unfathomable what value he has been to the world as a subject of science. And yet this is also a human being who is a kind, humble guy who certainly never asked for the spotlight," King said. "I think the world of him."

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First man cured of HIV infection now has terminal cancer - Modern Healthcare