Orphan Drug Designation applies to approximately 50,000 Patients who predominantly suffer from severe chronic pain due to ...

A patient death in a CRISPR trial halts gene editing studies, raising critical safety questions. What this means for the ...

Sarepta Therapeutics Inc. fell Monday after reporting its embattled gene therapy is expected to miss fourth-quarter sales ...

"Regulatory flexibility must be tailored for cell and gene therapies," commented FDA Commissioner Marty Makary. "These are ...

For those born with certain types of congenital deafness, the cochlear implant has been a positive and enabling technology.

The FDA granted fast track status to VectorY’s VTx-002, an antibody gene therapy targeting TDP-43 protein aggregates in ALS.

Ocugen has announced the publication of positive results from its phase 1 GARDian1 trial of OCU410ST, a gene therapy for ...

GEN’s first virtual event of 2026—we present a group of outstanding researchers and thought-leaders to discuss the latest advances and challenges in delivering genetic therapies.

A five-year-old boy who received the world's most expensive drug as a baby has made "incredible progress" and can walk ...

This virtual event is part of the Transforming Pediatric Healthcare series from U.S. News & World Report, developed with ...

It was the priciest biotech startup sale in North Carolina history. Now five years under Bayer, AskBio advances more AAV gene therapies.

Revolutions in gene therapy are rapidly changing the landscape of modern medicine. Revolutions in gene therapy are rapidly changing the landscape of modern medicine, forcing society and science alike ...