The FDA has approved 'Otarmeni', the first gene therapy for congenital hearing loss (DFNB9) caused by OTOF gene mutations.It ...

Optogenetics, recognized by the 2026 medicine Nobel, makes cells respond to light, including eye cells that normally relay ...

Optogenetic therapy, based on Nobel prize-winning science, involves turning nerve cells on and off using light ...

A gene therapy based on Nobel prize-winning science has partially restored sight in some blind patients, with a clinical ...

The underlying field was recognized this week when the 2026 Nobel Prize in Physiology or Medicine went to Karl Deisseroth, MD ...

Perceive Biotherapeutics, Inc. ("Perceive Bio" or the "Company"), a biotechnology company advancing novel gene therapies in ophthalmology and adjacent indications, today announced the completion of ...

At EURETINA 2026, Arshad Khanani, MD, FASRS, reviews sura-vec, 4D-150 and ixo-vec, 3 nAMD gene therapies in phase 3 with ...

The U.S. Food and Drug Administration today approved Fayuvi (rebisufligene etisparvovec-hopf), the first treatment for pediatric patients with mucopolysaccharidosis type IIIA (MPS IIIA), also known as ...

Ultragenyx Pharmaceutical RARE announced that the European Medicines Agency (EMA) has validated its Marketing Authorization ...

Jason is a general assignment reporter, with particular focus on genetic medicine and rare disease. Confidential tips can be sent on Signal at JasonMast.77. Pfizer has abandoned development of a ...

Record global approvals, CRISPR breakthroughs, and expanding clinical and commercial activity are accelerating demand for cell and gene therapy tools and reagents.Dublin, Oct. 08, 2026 (GLOBE NEWSWIRE ...

Forbes contributors publish independent expert analyses and insights. A patient in a late-stage gene editing trial for a rare heart condition died from fatal liver complications after receiving an ...