News

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 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., ...
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 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 ...
US FDA declines to approve Ultragenyx's gene therapy for rare genetic disorder
The U.S. Food and Drug Administration has declined to approve Ultragenyx Pharmaceutical's experimental gene therapy to treat ...
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 ...
Science News1y
Most of today’s gene therapies rely on viruses - Science News
Since gene therapy was first proposed to treat such genetic diseases in the 1970s, it has had thrilling highs — including the first successful gene therapy, in 1990, in a 4-year-old born with ...
InvestorsHub on MSN18h
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 ...