Gene therapy
Adeno-associated viruses have long been go-to vectors for gene therapies. How AAVs are improving will be among the cell and gene therapy topics to be covered in Baltimore this week.
Follow News Editor Greg Slabodkin and Managing Editor Jef Akst as they travel with some 8,000 others for discussions of cell and gene therapy advances, challenges, regulations and more.
Ahead of the FDA’s June target action date for Sarepta’s Duchenne muscular dystrophy gene therapy, ICER Chief Medical Officer David Rind blasted the regulator’s accelerated pathway in a JAMA viewpoint article.
Pfizer will go toe-to-toe with CSL Behring following the FDA’s Friday approval of its hemophilia B gene therapy Beqvez and will launch a warranty program based on the durability of response.
From gene-corrected cell therapies to a new CAR-T, the cell and gene therapy space looks to expand its reach into the market.
Nanoscope Therapeutics nabbed a victory on the changed primary endpoint of its Phase IIb vision loss gene therapy trial Tuesday, teeing the biotech up to file for FDA approval of the candidate this year.
With several recent approvals in the space and more on the horizon, BioSpace looks at some of the key decisions and their larger significance both for patients and science.
Brazil’s Ministry of Health and nonprofit Caring Cross announced a collaboration Tuesday aimed at local manufacturing of CAR-T cell and stem cell gene therapies at a much lower cost than Europe and the U.S.
Two days after winning FDA approval, Orchard Therapeutics on Wednesday provided its U.S. launch plans for metachromatic leukodystrophy gene therapy Lenmeldy, which has a wholesale acquisition cost of $4.25 million for the one-time treatment.
To help support the launch of Lyfgenia, bluebird bio on Monday entered into a five-year term loan deal with Hercules Capital that will extend the biotech’s cash runway through the first quarter of 2026.
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