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 ...

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

The Nobel Prize committee says that optogenetics will lay the foundations for a new era in neuroscience, while pharma ...

Andelyn Biosciences ("Andelyn"), a leading US-based patient-focused cell and gene therapy Contract Development and ...

An experimental treatment combining gene therapy with specialized goggles has helped some people with advanced blindness ...

Ultragenyx Pharmaceutical (RARE) is back in focus after the European Medicines Agency validated its Marketing Authorisation ...

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 ...

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

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 ...

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 ...

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 ...