A five-year clinical cohort study shows that an optogenetic therapy combining gene injection and light-stimulating goggles is safe and enabled several blind patients with advanced retinitis pigmentosa ...

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 ...

To see a 3-year-old boy who was born with profound deafness simply smile and point to the sounds coming from a nearby speaker felt like nothing short of a medical miracle to everyone in the room.

An international trial led by University of Pittsburgh scientists found that an optogenetic gene therapy combined with ...

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

A patient has died in a clinical trial evaluating a Rocket Pharmaceuticals gene therapy shortly after complications emerged that led the FDA to pause the study. An inquiry into the fatality is ...

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

A gene therapy designed to replace the missing protein that causes fragile X syndrome restored several disease-relevant traits in a mouse model, according to a new study published in Gene Therapy.

Meiragtx Holdings ((MGTX)) announced an update on their ongoing clinical study. Take advantage of TipRanks Premium at 50% off! Unlock powerful investing tools, advanced data, and expert analyst ...

Robust, muscle-specific activation of LAMA1 following a single administration of muscle-tropic AAV vectors encoding the GNDM activator Marked improvement in survival and muscle function in DyW mouse ...

Results from the largest cohort of children who received a gene therapy for a rare immunodeficiency condition have shown the long-term safety and efficacy of the curative treatment, in a study led by ...