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Tag: gene therapy


Cell and Gene Therapy, Industry

Germany approves legislation to improve cell & gene therapy availability

June 15, 2021

Via: EPM Magazine

The parliament advocated for an increase in the number of NUB inquiries that can take place each year. NUB inquiries are used by hospitals to gain short term reimbursement for innovative treatment and diagnostic methods, supplementing the standard Diagnosis Related […]


Cell and Gene Therapy, Industry

Biogen’s inherited eye disease gene therapy misses the mark in late-stage trial

June 15, 2021

Via: Pharma Times

The Phase III STAR study enrolled 169 adult males with a genetically confirmed diagnosis of choroideremia. It evaluated the safety and efficacy of a single subretinal injection of the investigational gene therapy. The primary endpoint was the proportion of patients […]


Cell and Gene Therapy, Industry

Quality considerations for a growing gene therapy field

June 7, 2021

Via: Biopharma Dive

The approvals of Roche’s Luxturna (2017) for inherited retinal disease and Novartis’ Zolgensma (2019) for spinal muscular atrophy are indicative of a resurgence in the gene therapy field after a decline due to the death of a patient in 1999. […]


Cell and Gene Therapy, Industry

Biogen takes a small gamble with Ginkgo manufacturing deal, hoping for big rewards in gene therapy

May 21, 2021

Via: FiercePharma

For a company that generated $13.4 billion last year, $5 million is hardly a make-or-break investment. So it goes with the $5 million gamble Biogen is taking on Ginkgo Bioworks’ cell programming platform and know-how. With an incentive-laden deal, Massachusetts-based […]


Cell and Gene Therapy, Industry

Sarepta Therapeutics’ DMD gene therapy shows early success

May 19, 2021

Via: PMLiVE

The 12-week results come from the first 11 participants enrolled in Sarepta’s Study SRP-9001-103 or ENDEAVOR study, which is being conducted in partnership with Swiss pharma company Roche. The study, which has so far enrolled 20 participants with DMD between […]


Cell and Gene Therapy, Industry

Biogen gene therapy misses goal in eye disease study

May 14, 2021

Via: Biopharma Dive

Biogen’s acquisition of Nightstar signaled the biotech company’s commitment to gene therapy research, an investment it’s expanded upon with subsequent deals like a January collaboration with Germany’s ViGeneron and another earlier this week with Capsigen. The Nightstar deal bought Biogen […]


Cell and Gene Therapy, Industry, News

Third Rock launches biotech targeting ‘notoriously difficult’ DNA regulators

May 13, 2021

Via: Biopharma Dive

The instructions for making a human are long and complicated. They involve over 3 billion sets of microscopic letters, so tightly coiled that, if unwound, they would be taller than the average person. The daunting task of reading these instructions […]


Cell and Gene Therapy, Industry

Gene therapy successfully treats rare immunodeficiency in paediatric patients

May 12, 2021

Via: European Pharmaceutical Review

An experimental gene therapy developed by researchers at the US’s University of California – Los Angeles (UCLA) and UK’s Great Ormond Street Hospital (GOSH) in London has successfully treated 48 of 50 children born with a rare and deadly inherited […]


Cell and Gene Therapy, Industry, Mergers and Acquisitions

CSL closes deal for UniQure gene therapy in hopeful sign for M&A

May 6, 2021

Via: Biopharma Dive

According to Paul Matteis, an analyst at Stifel, the closing of the deal removes a question mark hanging over UniQure’s stock. Regulators, especially those in the U.S., are taking harder looks at pharmaceutical acquisitions, suggesting that approval of the UniQure-CSL […]


Cell and Gene Therapy, Industry

Gene therapy in Alzheimer’s disease mouse model preserves learning and memory

May 4, 2021

Via: ScienceDaily

Researchers at University of California San Diego School of Medicine, with colleagues elsewhere, have used gene therapy to prevent learning and memory loss in a mouse model of Alzheimer’s disease (AD), a key step toward eventually testing the approach in […]


Cell and Gene Therapy, Industry

Resource Links Gene Variants with Immune System Regulation and Immune-Mediated Diseases

April 30, 2021

Via: Genetic Engineering and Biotechnology News

University of Tokyo researchers and colleagues at the RIKEN Research Institute have compiled a first-of-its-kind genetic database for autoimmune and autoinflammatory diseases. It’s hoped that the resource will offer up new insights into how immune disorders develop, and potentially aid […]


Cell and Gene Therapy, Industry, News

Gene Therapy CDMO Forges Ahead with $120M Series B Financing

April 29, 2021

Via: Genetic Engineering and Biotechnology News

Gene therapy-focused CDMO Forge Biologics has closed on a $120 million Series B financing, saying the proceeds will accelerate its planned expansion of adeno-associated virus (AAV) manufacturing capabilities. The company operates a 175,000-square-foot cGMP facility in Columbus, OH, dedicated to […]