News

CRISPR and Cancer: How Gene Editing Is Revolutionizing Oncology
Discover how CRISPR gene editing is creating personalized cancer therapies, enhancing immunotherapy, and offering new hope ...
STAT6h
For many Duchenne families, halt to gene therapy is heartbreak upon heartbreak
As gene therapy maker Sarepta Therapeutics tangles with FDA over its Duchenne treatment, patients and their families are ...
Roche Pauses Contested Gene Therapy’s Shipments Outside US
Roche Holding AG paused some shipments of the controversial gene therapy Elevidys following a similar action by the embattled ...
STAT7h
A mother’s perspective on Sarepta’s gene therapy halt
This week on "The Readout LOUD" podcast, a mother whose son has Duchenne muscular dystrophy shares her perspective on Sarepta ...
Researchers reduce cardiac arrhythmia after heart attacks using gene therapy
During a severe heart attack many heart muscle cells die and are replaced by scar tissue to stabilize the heart wall.
Sarepta shares slide again as drugmaker bows to FDA pressure to pause gene therapy
Sarepta Therapeutics is pausing shipments of its gene therapy for muscular dystrophy following several patient deaths that ...
MedPage Today3d
Drugmaker Refuses FDA's Request to Pull Gene Therapy Tied to Patient Deaths
The agency also revoked Sarepta's platform technology designation for AAVrh74 Friday and issued a safety communication saying the agency was investigating the three deaths due to acute liver failure ...
Fierce Pharma3d
Sarepta stands behind Elevidys after FDA requests gene therapy be pulled from market
As the controversy over the safety of Sarepta Therapeutics’ gene therapies comes to a head, the biotech is standing firm ...
Managed Healthcare Executive1d
Roctavian Gene Therapy Controls Bleeding for Five Years in Severe Hemophilia A
BioMarin's Roctavian gene therapy shows long-term effectiveness in treating severe hemophilia A, reducing bleeding and ...
Gene therapy maker Sarepta tells FDA it won’t halt shipments despite patient deaths
Drugmaker Sarepta Therapeutics says it won’t comply with a request from U.S. regulators to halt all shipments of its gene ...
Sarepta's Shocking Stand: Company Rejects FDA Request To Pull Gene Therapy Elevidys
FDA places clinical hold on Sarepta's LGMD gene therapy trials after three deaths and revokes platform tech designation due to safety concerns.
Roche pauses shipments of Elevidys gene therapy outside US
Roche Holding AG said on Tuesday it has paused shipments of muscular disorder gene therapy Elevidys in some countries outside ...