Dr. Swee Lay Thein and Dr. Stuart Orkin won the $3 million Breakthrough Prize in Life Sciences for their work toward a ...

Philadelphia—A preclinical study uncovered a new gene therapy that targets pain centers in the brain while eliminating the risk of addiction from narcotics treatments, a breakthrough which could ...

Researchers who developed a gene therapy for inherited retinal diseases, along with five other teams, have received this year ...

The Breakthrough Prize Foundation awarded researchers involved in developing gene therapies for blindness and sickle cell ...

The US Food and Drug Administration (FDA) has released draft guidance on how sponsors can utilize next-generation sequencing ...

A new study has brought scientists one step further in the direction of developing a cure for a brutal group of rare brain disorders known as SYNGAP1-related disorders, or SRDs. Researchers were able ...

Can humans regrow limbs? Researchers find SP6 and SP8 genes are vital for regeneration in axolotls and mice, creating a viral ...

Peripheral nerve injuries, often caused by traumatic events such as car accidents, falls or battlefield injuries, can leave patients with long-term weakness, numbness or loss of function. Despite ...

For the second time, Astellas is walking away from Taysha Gene Therapies, this time choosing not to license the biotech’s adeno-associated-virus-based therapy designed to treat a rare ...

A few months after launching its lead gene therapy into the clinic, Arbor Biotechnologies has found a European partner to come along for the ride. Italian pharma Chiesi is fertilizing Arbor’s rare ...

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 ...

The treatment targets a specific type of inherited deafness caused by mutations in a gene called OTOF.