News

The American Journal of Managed Care2d
Progress and Challenges With Gene Therapy in Hereditary Hearing Loss
Gene therapy appears to be a promising approach for a subset of genetic deafness, although challenges remain with development ...
Fierce Pharma2d
Vinay Prasad's exit signals FDA's probable slip toward more permissive regulation of cell and gene therapy: analyst
But this time, the unknown is hitting the industry in a different way from when Prasad was tapped to lead CBER less than ...
FDA Says Sarepta Can Resume Gene Therapy in Some Patients
In a major win for Sarepta Therapeutics Inc., US regulators are recommending that patients who can walk be allowed to take ...
Fierce Pharma1d
Untangling Sarepta’s gene therapy fallout and a growing trust deficit
Over a whirlwind three weeks, Sarepta Therapeutics has faced tough safety questions around its commercial gene therapy ...
Progress in gene therapy offers hope for long-term knee pain relief
For nearly three decades, Mayo Clinic researcher Christopher Evans, Ph.D., has pushed to expand gene therapy beyond its ...
Sarepta will resume gene therapy shipments after FDA review of recent patient death
Shares of beleaguered drugmaker Sarepta Therapeutics jumped in afterhours trading Monday after the company said it would ...
GEN - Genetic Engineering and Biotechnology News1d
Gene Therapy Companies Set Sights on Cardiovascular Disease
The primary safety risks associated with gene therapy include immune reactions and off-target effects in unintended organs, such as the liver, lungs, and spleen. Nevertheless, Ali and Townsend note ...
Two boys died after a gene therapy. This family won’t give up hope.
The Revell family hoped a gene therapy could buy time for their sons, who have a rare and fatal disease. After two patients ...
X-SCID gene therapy gives scientists a rare glimpse into early immune system
Findings from St. Jude Children's Research Hospital demonstrate that virtual memory T cells, a specialized group of immune ...
Managed Healthcare Executive1d
Gene Therapy for Wet AMD May Replace Anti-VEGF Injections | ASRS 2025
Ixo-vec reduced anti-VEGF injection frequency and was well tolerated in patients with wet AMD. A phase 3 pivotal trial ...
FDA’s Reversal on Gene Therapy Helps My Son
Parents of boys with Duchenne muscular dystrophy weigh in on drug innovation and medical regulation.
Healthcare Dive18d
33 states pick up CMS program to pay for sickle cell gene therapies
The CMS said on Wednesday that 33 states, plus the District of Columbia and Puerto Rico, will participate in the “Cell and Gene Therapy Access Model” to centrally coordinate insurance coverage ...