Regeneron gene therapy improves hearing in two children
Bio Pharma Dive
MAY 8, 2024
The results add to an early but growing body of evidence suggesting gene therapy may improve outcomes in young kids with a genetic form of deafness.
Wed.May 08, 2024
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ASGCT2024: FDA’s Marks on Accelerated Approval, Gene Therapy Costs and More
BioSpace
MAY 8, 2024
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.
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Regeneron gene therapy restores hearing in deaf child
pharmaphorum
MAY 8, 2024
Regeneron’s gene therapy for otoferlin-related hearing loss restored hearing to normal levels in one profoundly deaf child within just 24 weeks
ASGCT24: Regeneron’s Gene Therapy Shows ‘Dramatic’ Hearing Improvement in Dea.
BioSpace
MAY 8, 2024
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.
Making the Case for Case Management for Cell and Gene Therapies
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As demand for advanced therapies increases, so does the need for more specialized supply chain support, as these products have strict transportation and handling requirements. It is crucial to ensure supply chain risks are mitigated and have proactive strategies in place to address unforeseen challenges before they become an issue.
For every million-dollar gene therapy, Peter Marks gets an email from FDA chief Robert Califf
Fierce Pharma
MAY 8, 2024
After Orchard Therapeutics’ gene therapy for a rare genetic disease was approved in March, Peter Marks, M.D., received a simple email from Commissioner Robert Califf, M.D. | After Orchard Therapeutics’ gene therapy for a rare genetic disease was approved in March, Peter Marks, M.D.,
Sarepta Seeks Label Expansion, Bigger Market for DMD Gene Therapy
BioSpace
MAY 8, 2024
As Sarepta Therapeutics moves closer to full approval and an expanded label for its gene therapy, some experts push back on clinical efficacy and cost while others note the hope it provides patients with Duchenne muscular dystrophy.
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