News

Science Daily1h
CRISPR uncovers gene that supercharges vitamin D—and stops tumors in their tracks
A gene called SDR42E1 has been identified as a key player in how our bodies absorb and process vitamin D. Researchers found that disabling this gene in colorectal cancer cells not only crippled their ...
BioSpace1d
Sarepta Tags DMD Gene Therapy Elevidys With Black Box Warning, Axes 500 Staff
Following the death of two teenage patients with Duchenne muscular dystrophy following Elevidys treatment, Sarepta ...
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., ...
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 ...
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 ...
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.
Live Science6d
In a first, congenital deafness in teens and adults treated with new gene therapy
This is the first time such results have been achieved in both children and adult patients born with a specific type of ...
InvestorsHub on MSN20h
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 ...