ASGCT
FDA Platform Designation Program Could Ease Path to Market for Gene-Editing Therapies
In a draft guidance, the agency outlined how the well-understood and reproducible components of genetic medicines could qualify as platform technologies.
Cell and Gene Therapy Pricing Model Remains Sticking Point Despite CMS Efforts to Defray Costs
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Despite some CMS policies designed to improve patient access to cell and gene therapies, the reliance on a value-based pricing model drew sharp criticism in an ASGCT session.
Regeneron Gene Therapy Restores Hearing in Infant With Genetic Form of Deafness, Data at ASGCT Show
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The 10-month-old patient was treated with DB-OTO, a gene therapy developed by Decibel Therapeutics, which Regeneron acquired last year.
Cancer Cell Therapies at ASGCT24
At the meeting, companies and academic centers presented early and long-term follow-up data on investigational cell therapies in several tumor types.
ASGCT24: Regenxbio, Rocket Pharmaceuticals, Orchard Therapeutics, and More
At the meeting, companies and academic centers presented early and follow-up data on investigational gene therapies for nMPS II, PKD, MLD, and more.