Gene therapy
Pfizer’s investigational Duchenne muscular dystrophy gene therapy, fordadistrogene movaparvovec, failed in a late-stage study to significantly improve motor function in patients versus placebo.
Given their seven-figure price tags, it’s not clear how accessible the would-be cures will be to U.S. patients on public or private insurance.
BioSpace’s Lori Ellis discusses the risks and challenges of cell and gene therapy combination products with DIA speakers James Wabby, AbbVie and Rob Schulz, Suttons Creek, Inc.
While these technologies are now a therapeutic reality, the ASGCT 2024 annual meeting this week was a reminder of just how far we are from widespread use.
With 15 patients started across its three gene therapies, bluebird bio claims a 138% year-over-year revenue growth and aims to initiate up to around 100 new patients in the current year.
At a Thursday ASGCT 2024 session, CBER Director Peter Marks made the case for a better, “more convergent” global framework on cell and gene therapies, especially for rare diseases.
Launched in 2021, the public-private consortium on Wednesday updated ASGCT attendees on its efforts to bring adeno-associated virus gene therapies to more rare disease patients.
We are in an unprecedented time in neurotherapeutics. Medicines that address the causative disease biology underlying central nervous system
In a fireside chat at the American Society of Gene & Cell Therapy conference, CBER Director Peter Marks spoke with Takeda’s Kristin Van Goor about how the regulator is approaching the exploding gene therapy space.
Regeneron Pharmaceuticals on Wednesday revealed that its investigational gene therapy DB-OTO restored hearing in two young children, according to an oral presentation at the American Society of Gene & Cell Therapy annual meeting.
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