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Adeno-Associated Virus (AAV) Vector-Based Gene Therapy Market Expected to Grow at a Significant CAGR of 43 4% During the Study Period 2019-30

Share this article Share this article LAS VEGAS, Jan. 25, 2021 /PRNewswire/ The AAV Vector-Based Gene Therapy market size is anticipated to shoot up exponentially attributing to an increase in the approval of a growing number of gene therapies and readily adoption on approval, ability to treat a broad array of conditions, increasing prevalence of diseases, convenient one-time dosing approach and curative treatment options. DelveInsight s  report offers a clear picture of the market position of the AAV vectors in the Gene therapy, emerging pipeline therapies, AAV vector-based Gene Therapy market share occupied by individual diseases, current and forecasted market share in the 7MM (the US, EU5 (the UK, Germany, France, Italy and Spain) and Japan) for the study period 2017-30. 

BIOMARIN INVESTIGATION INITIATED by Former Louisiana Attorney General: Kahn Swick & Foti, LLC Invest

NEW ORLEANS (BUSINESS WIRE) Former Attorney General of Louisiana, Charles C. Foti, Jr., Esq., a partner at the law firm of Kahn Swick & Foti, LLC (“KSF”), announces that KSF has commenced an investigation into BioMarin Pharmaceutical Inc. (NasdaqGS: BMRN).On August 19, 2020, the Company disclosed that it received.

5 questions facing the FDA in 2021

Editor’s note: This story is part of a series on the trends that will shape the industry in 2021. You can find all the articles on our trendline. Few government agencies were as front and center in the public eye last year as the Food and Drug Administration, which came under immense pressure to authorize unproven drugs as the world grappled with the pandemic and conducted high-stakes reviews of two coronavirus vaccines. As COVID-19 cases continue to soar in the U.S., most of those pressures haven t gone away. But the inauguration next week of Joseph Biden as president will almost certainly bring new management to the agency, a change that can often result in policy shifts both subtle and far-reaching.

BioMarin Announces Positive Phase 3 Gene Therapy Trial Results in Adults with Severe Hemophilia A; Study Met All Primary and Secondary Efficacy Endpoints in One-Year Data Set

Share this article Share this article SAN RAFAEL, Calif., Jan. 10, 2021 /PRNewswire/  BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) today announced positive topline results from its ongoing global Phase 3 GENEr8-1 study of valoctocogene roxaparvovec, an investigational gene therapy for the treatment of adults with severe hemophilia A.  This is the largest global Phase 3 study to date for any gene therapy in any indication, with 134 participants.  All participants in the study received a single dose of valoctocogene roxaparvovec and completed a year or more of follow-up.  Figure 1: Box-and-Whiskers Plot of Factor VIII Activity in 4-Week Intervals, Subset Population (N=17 ) - Key: The boxes show the interquartile ranges with the lines in the boxes indicating medians. The ends of the whiskers represent the minimum and maximum values and diamonds indicate the means. - Includes only HIV-negative subjects dosed 2 or more years prior to Nov 2020 data cut date. One subject was lost t

Gene-Therapy Firm Bluebird Bio Is Spinning Off Its Cancer Programs

Shares of the gene-therapy biotech bluebird bio have tumbled in recent months, with the stock dropping 46.4% over the past year as the S&P 500 Health Care sector index has climbed 14.2%. Now, the company is trying something entirely different in an effort to stop the downward spiral. In a move announced Monday morning, bluebird (ticker: BLUE) said it planned to split into two public companies by the end of the year. One will focus on rare diseases and the other will work on treating cancer. “After careful strategic review, it is clear to us that the two businesses are best served by independent leadership and teams to drive distinct strategic and operational objectives,” said the company’s CEO, Nick Leschly, in a statement.

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