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Category Archives: Genetic Therapy

Gene editing may be a path to restore partial hearing – Harvard Gazette

Jeffrey Holt, professor of otolaryngology and neurology at the Harvard Medical School and an author of the paper, successfully treated TMC1-related deafness with gene therapy by situating cells with healthy versions of the gene among the unhealthy to counteract the disease-causing mutation. But Volha Olga Shubina-Aleinik, a postdoctoral fellow in the Holt lab, said gene therapy may have a limited duration. That is why we need more advanced techniques, such as gene editing, which may last a lifetime.

Yeh spent years designing a base editor that could find and erase the disease-causing mutation and replace it with the correct DNA code. But even after she demonstrated good results in vitro, there was a problem: Base editors are too large to fit in the traditional delivery vehicle, adeno-associated virus, or AAV. To solve this problem, the team split the base editor in half, sending each piece in with its own viral vehicle. Once inside, both viruses needed to infect the same cells so the two base editor halves could rejoin and head off to find their target. Despite the labyrinthine entry, the editor proved to be efficient, causing only a minimum of undesired deletions or insertions.

We saw very little evidence of off-target editing, Liu said. And we noticed that the edited animals had much-preserved hair-cell morphology and signal transduction, meaning the hair cells, the critical cells that convert sound waves to neuronal signals, appeared more normal and behaved more normally.

After the treatment, Yeh performed an informal test: She clapped her hands. Mice that had previously lost all hearing ability jumped and turned to look. Formal tests revealed the base editor worked, at least in part: Treated mice had partially restored hearing and could respond to loud and even some medium sounds, Yeh said.

Of course, more work needs to be done before the treatment can be used in humans. Unedited cells continued to die, causing deafness to return even after the base editor restored function to others.

But the study also proved that the clandestine AAV delivery method works. Already, Liu is using AAV to tackle other genetic diseases, including progeria (premature aging), sickle cell anemia, and degenerative motor diseases. Were actually going after quite a few genetic diseases now, including some prominent ones that have caused a lot of suffering and energized pretty passionate communities of patients and patient families to do anything to find a treatment, Liu said. For progeria, theres no cure. The best treatments extend a childs average lifespan from about 14 to 14.5 years.

For Yeh, whose friend is still living with hearing loss, genetic deafness remains her primary target. Theres still a lot to explore, she said. Theres so much unknown.

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How COVID-19 is Affecting Clinical Trials and Drug Supplies from Biotech Bay Companies – BioSpace

Much of the news has been focused on the novel coronavirus and the rush to develop vaccines and treatments for the disease it causes (called COVID-19). But what is happening with all the other companies not directly involved in COVID-19 research? And how is COVID-19 affecting their clinical trials and drug supplies?

To answer these questions, BioSpace looked at COVID-19s impact on each of the Hotbeds.

Biotech Bay, the bustling biotech industry around San Francisco in California, is home to lots of biopharma companies, many of whom are trying to keep business as close to usual while adapting to these unique times.

Check out the table below for the impacts that Biotech Bay companies are experiencing due to COVID-19.

(For information about what Biotech Bay companies have joined the fight against COVID-19 and how they are working towards vaccines and treatments, check out this article.)

The information in this table was up to date as of May 18, 2020.

4D Molecular Therapeutics

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Allakos, Inc.

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Ambys Medicines

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Apollomics, Inc.

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Assembly Biosciences

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BioMarin Pharmaceutical Inc.

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BridgeBio

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DNA Script

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FibroGen

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Global Blood Therapeutics

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Horizon Therapeutics

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Intabio

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Iovance Biotherapeutics

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JOINN Biologics

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Maze Therapeutics

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MyoKardia

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How COVID-19 is Affecting Clinical Trials and Drug Supplies from Biotech Bay Companies - BioSpace

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Global Gene Therapy for Inherited Genetic Disorders Market 2020- Impact of COVID-19, Future Growth Analysis and Challenges | BioMarin Pharmaceutical…

In the Gene Therapy for Inherited Genetic Disorders statistical surveying study, 2019 is considered as the base year, and 2020-2027 is considered as the estimate time frame to anticipate the market size. Significant districts stressed in the report incorporate North America, South America, Europe, Asia, Pacific region Middle East & Africa

The report on the Gene Therapy for Inherited Genetic Disorders Market gives a foot perspective on the present continuing inside the Gene Therapy for Inherited Genetic Disorders market. Further, the report likewise considers the effect of the novel COVID-19 pandemic on the Gene Therapy for Inherited Genetic Disorders market and offers an away from of the anticipated market variances during the estimate time frame.

The global Gene Therapy for Inherited Genetic Disorders market report covers major market players such as

BioMarin Pharmaceutical Inc.bluebird bio Inc.Novartis AGOrchard Therapeutics PlcSpark Therapeutics Inc.

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In 2027, the Gene Therapy for Inherited Genetic Disorders market is spectated to outperform ~US$ xx Mn/Bn with a CAGR of xx% over the estimate time frame. The Gene Therapy for Inherited Genetic Disorders market clicked an estimation of ~US$ xx Mn/Bn in 2019. Region is required to represent a critical piece of the overall industry, where the Gene Therapy for Inherited Genetic Disorders market size is anticipated to blow up with a CAGR of xx% during the estimate time frame.

The report inspects each Gene Therapy for Inherited Genetic Disorders market player as per its piece of the pie, creation impression, and development rate. SWOT examination of the players (qualities, weaknesses, opportunities and threats) has been covered in this report. Further, the Gene Therapy for Inherited Genetic Disorders market study portrays the ongoing dispatches, understandings, R&D undertakings, and business systems of the market players including.

High Points of the Global Gene Therapy for Inherited Genetic Disorders Report:

The research report includes specific segments by Type and by Application. This study provides information about the sales and revenue during the historic and forecasted period of 2015 to 2027.

Global Gene Therapy for Inherited Genetic Disorders Market Segmentation By Type:

Eye DisordersHematological DisordersCentral Nervous System DisordersMuscular DisordersOthers

Global Gene Therapy for Inherited Genetic Disorders Market Segmentation By Applications:

HospitalClinicResearch InstituteOthers

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Global Gene Therapy for Inherited Genetic Disorders Market: Regional Analysis

The Gene Therapy for Inherited Genetic Disorders market is examined and showcase size data is given by region. The report incorporates nation astute and region-wise market size for the period 2015-2027. It additionally incorporates showcase size and conjecture by Type and by Application fragment as far as deals and income for the period 2015-2027.

The key regions covered in the Gene Therapy for Inherited Genetic Disorders market report are:

The examination report on the global Gene Therapy for Inherited Genetic Disorders market offers a treasury of economic situations and strategies wherein the market has been acting in various circumstances. Additionally, SWOT investigation and Porters Five Forces examination are utilized to speak to the positive and negative factors that are affecting the market development. Additionally, this report covers the inside and out factual examination and the market elements and requests which give an entire situation of the business.

Global Gene Therapy for Inherited Genetic Disorders Market: Competitive Analysis

This area of the report recognizes different key makers of the market. It enables the reader to comprehend the systems and coordinated efforts that players are concentrating on battle rivalry in the market. The extensive report gives a critical infinitesimal gander at the market. The reader can distinguish the impressions of the producers by thinking about the worldwide income of makers, the worldwide cost of manufacturer, and deals by makers during the conjecture time of 2015 to 2027.

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Global Gene Therapy for Inherited Genetic Disorders Market 2020- Impact of COVID-19, Future Growth Analysis and Challenges | BioMarin Pharmaceutical...

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Strategic Analysis to Understand the Competitive Outlook of Gene Therapy Market – Cole of Duty

Prophecy Market Insights Gene Therapy market research report provides a comprehensive, 360-degree analysis of the targeted market which helps stakeholders to identify the opportunities as well as challenges. The research report study offers keen competitive landscape analysis including key development trends, accurate quantitative and in-depth commentary insights, market dynamics, and key regional development status forecast 2020-2029. It incorporates market evolution study, involving the current scenario, growth rate, and capacity inflation prospects, based on Porters Five Forces and DROT analyses.

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An executive summary provides the markets definition, application, overview, classifications, product specifications, manufacturing processes; raw materials, and cost structures.

Market Dynamics offers drivers, restraints, challenges, trends, and opportunities of the Gene Therapy market

Segment Level Analysis in terms of types, product, geography, demography, etc. along with market size forecast

Regional and Country- level Analysis different geographical areas are studied deeply and an economical scenario has been offered to support new entrants, leading market players, and investors to regulate emerging economies. The top producers and consumers focus on production, product capacity, value, consumption, growth opportunity, and market share in these key regions, covering

The comprehensive list of Key Market Players along with their market overview, product protocol, key highlights, key financial issues, SWOT analysis, and business strategies. The report dedicatedly offers helpful solutions for players to increase their clients on a global scale and expand their favour significantly over the forecast period. The report also serves strategic decision-making solutions for the clients.

Competitive landscape Analysis provides mergers and acquisitions, collaborations along with new product launches, heat map analysis, and market presence and specificity analysis.

Segmentation Overview:

Gene TherapyMarket Key Companies:

GlaxoSmithKline plc, Bluebird Bio, Inc., Adaptimmune Therapeutics plc, Celgene Corporation, Shanghai Sunway Biotech Co. Ltd., Merck KGaA, Transgene SA, and OncoGenex Pharmaceuticals, Inc.

The Gene Therapy research study comprises 100+ market data Tables, Graphs & Figures, Pie Chat to understand detailed analysis of the market. The predictions estimated in the market report have been resulted in using proven research techniques, methodologies, and assumptions. This Gene Therapy market report states the market overview, historical data along with size, growth, share, demand, and revenue of the global industry.

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The study analyses the manufacturing and processing requirements, project funding, project cost, project economics, profit margins, predicted returns on investment, etc. This report is a must-read for investors, entrepreneurs, consultants, researchers, business strategists, and all those who have any kind of stake or are planning to foray into the Gene Therapy industry in any manner.

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Strategic Analysis to Understand the Competitive Outlook of Gene Therapy Market - Cole of Duty

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Gene Therapy Market to Reach $4402 million by 2023 with Key Players Novartis, Kite Pharma, GlaxoSmithKline, Spark Therapeutics – Cole of Duty

The global gene therapy market was valued at $584 million in 2016, and is estimated to reach $4,402 million by 2023, registering a CAGR of 33.3% from 2017 to 2023. Gene therapy is a technique that involves the delivery of nucleic acid polymers into a patients cells as a drug to treat diseases. It fixes a genetic problem at its source. The process involves modifying the protein either to change the genetic expression or to correct a mutation. The emergence of this technology meets the rise in needs for better diagnostics and targeted therapy tools. For instance, genetic engineering can be used to modify physical appearance, metabolism, physical capabilities, and mental abilities such as memory and intelligence. In addition, it is also used for infertility treatment. Gene therapy offers a ray of hope for patients, who either have no treatment options or show no benefits with drugs currently available. The ongoing success has strongly supported upcoming researches and has carved ways for enhancement of gene therapy.

Top Companies Covered in this Report:, Novartis, Kite Pharma, Inc., GlaxoSmithKline PLC, Spark Therapeutics Inc., Bluebird bio Inc., Genethon, Transgene SA, Applied Genetic Technologies Corporation, Oxford BioMedica, NewLink Genetics Corp.

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The gene therapy market is a widely expanding field in the pharmaceutical industry with new opportunities. This has piqued the interests of venture capitalists to explore this market and its commercial potential. Major factors that drive the growth of this market include high demands for DNA vaccines to treat genetic diseases, targeted drug delivery, and high incidence of genetic disorders. However, the stringent regulatory approval process for gene therapy and the high costs of gene therapy drugs are expected to hinder the growth of the market.

The global gene therapy market is segmented based on vector type, gene type, application, and geography. Based on vector type, it is categorized into viral vector and non-viral vector. Viral vector is further segmented into retroviruses, lentiviruses, adenoviruses, adeno associated virus, herpes simplex virus, poxvirus, vaccinia virus, and others. Non-viral vector is further categorized into naked/plasmid vectors, gene gun, electroporation, lipofection, and others. Based on gene type, the market is classified into antigen, cytokine, tumor suppressor, suicide, deficiency, growth factors, receptors, and others. Based on application, the market is divided into oncological disorders, rare diseases, cardiovascular diseases, neurological disorders, infectious disease, and other diseases. Based on region, it is analyzed across North America, Europe, Asia-Pacific, and LAMEA.

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Table Of Content

CHAPTER 1: INTRODUCTION

CHAPTER 2: EXECUTIVE SUMMARY

CHAPTER 3: MARKET OVERVIEW

CHAPTER 4: GENE THERAPY MARKET, BY VECTOR TYPE

CHAPTER 5: GENE THERAPY MARKET, BY GENE TYPE

CHAPTER 6: GENE THERAPY MARKET, BY APPLICATION

CHAPTER 7: GENE THERAPY MARKET, BY REGION

CHAPTER 8: COMPANY PROFILE

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Gene Therapy Market to Reach $4402 million by 2023 with Key Players Novartis, Kite Pharma, GlaxoSmithKline, Spark Therapeutics - Cole of Duty

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Irish stars to play virtual concert for baby in need of life-saving treatment – Irish Mirror

A host of top Irish music stars including Daniel ODonnell and Nathan Carter will take part in a six hour virtual concert on Saturday to help raise funds for a baby girl in need of life-saving treatment.

Little Livie was born with a rare neuromuscular condition and needs more than 2 million for treatment in the US.

Livies parents, Keith Mulhern and his fiance Karen Vickers who live in Co Meath, are calling for all possible support in their quest to save their daughters life.

Livie (Olivia) is nine months old and recently diagnosed with a rare and serious genetic neuromuscular condition called spinal muscular atrophy (SMA) type 1.

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SMA is caused by a gene deletion which results in deterioration of Livies nerve cells connecting her brain and spinal cord to her bodys muscles. There is no cure and Livie has its most severe type.

Without any treatment, her condition would continue to deteriorate and it would be unlikely that she would live past her second birthday.

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Treatment options for Livie are very limited.

The familys best hope for a better life for Livie is a once-off gene therapy called Zolgensma, which is currently only available in USA. It costs approximately US$2,100,000.

Livies story has touched thousands of peoples hearts worldwide and sparked a major GoFundMe campaign to reach the goal and give her the best chance.

Keith and Karen are against the clock as Zolgensma is only available to children under the age of two years.

Over 428,000 has been raised so far, with the couple saying that they are on a long road but have got off to a fantastic start.

They are now hoping todays concert, which is being organised by Newtowncunningham musician Alister McQuilken, will give them a further financial boost in their quest to get enough money for Livies treatment.

She is our world, and we want to do everything we possibly can to give her a better quality of life and a longer life, Karen said.

Click here to visit the GoFundMe page: https://www.gofundme.com/f/a-better-life-for-livie

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Irish stars to play virtual concert for baby in need of life-saving treatment - Irish Mirror

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