Clinical and commercial quality controls are eased as the FDA looks to expedite therapy approvals in areas of unmet need.

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

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

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

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 ...

Osteoarthritis is a highly prevalent joint disease that leads to cartilage breakdown, pain and disability, yet there are ...

U.K. biotech Ikarovec has teamed up with VectorBuilder to work on an eye disease gene therapy that could be administered in a ...

Taysha Gene Therapies Inc. (NASDAQ:TSHA) is one of the best penny stocks to buy for 2026. On January 6, the company confirmed ...

The new method is designed to focus specifically on pain-related signals, without interfering with normal activity in other ...

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

Lexeo Therapeutics Inc.’s phase I/II data – characterized by Oppenheimer analyst Leland Gershell as “heroic” – this week with ...