News

MedPage Today2h
Duchenne Gene Therapy Will Undergo Changes After Patient Deaths
At the FDA's request, delandistrogene moxeparvovec (Elevidys), the only approved gene therapy for Duchenne muscular dystrophy ...
InvestorsHub on MSN7h
Rocket Pharmaceuticals Shares Jump After FDA Grants Accelerated Review Status to Gene Therapy
Rocket Pharmaceuticals Inc. (NASDAQ:RCKT) surged 15% after receiving a key regulatory boost from the U.S. Food and Drug ...
Managed Healthcare Executive1d
CMS Launches Value-Based Gene Therapy Program for Sickle Cell Disease
CMS is brokering outcomes-based agreements on behalf of Medicaid programs. Thirty-three states, along with Washington, D.C., ...
Healio3h
FDA fast tracks one-time intravitreal gene therapy for geographic atrophy
The FDA granted fast track designation to SAR446597, an intravitreal gene therapy for the treatment of geographic atrophy due to age-related macular degeneration, according to a press release from ...
Fierce Healthcare2d
CMS officially names participants in Cell and Gene Therapy Access Model
The Centers for Medicare and Medicaid Services (CMS) has selected 35 participants for its new, voluntary Cell and Gene ...
Researchers used gene therapy to restore hearing in adults
Groundbreaking new research shows that it is possible to use gene therapy to restore hearing in both children and adults.
Fierce Pharma3d
Ultragenyx takes another hit as FDA snubs AAV gene therapy over manufacturing issues
The manufacturing-related rejection for UX111 is more than a setback for Ultragenyx, as it also delays a key test of the new ...
Managed Healthcare Executive10h
Applying Evidence Standards to Guide Gene Therapy Use in BCG-Unresponsive NMIBC
An expert discusses how high-cost gene therapies require evidence standards including Phase 3 trial data showing complete response rates and durability, real-world evidence and health economic ...