Tag: gene therapy

June 15, 2021

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 […]

June 15, 2021

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 […]

June 7, 2021

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. […]

May 21, 2021

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 […]

May 19, 2021

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 […]

May 14, 2021

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 […]

May 13, 2021

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 […]

May 12, 2021

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 […]

May 6, 2021

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 […]

May 4, 2021

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 […]

April 30, 2021

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 […]

April 29, 2021

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 […]