Unlike existing treatments for Alzheimer’s that target unhealthy protein deposits, the new approach developed at UC San Diego ...

Leaders from the world of cell and gene therapy shared their personal stories and policy recommendations in an extraordinary roundtable conducted by the FDA in front of its recently-appointed top ...

In the first part of this series, we explored how early genetic screening and gene therapy transform the lives of newborns ...

A Falmouth teen, Jayden Wilsey, is the first to receive gene therapy to treat sickle cell at Boston Children's Hospital.

RegenXBio has shared updated phase 1/2 data on its Duchenne muscular dystrophy (DMD) gene therapy, providing evidence that ...

The company is one of the first to receive a “platform technology designation,” which could speed the review of certain gene ...

As newborn screening and rapid DNA sequencing become routine, we are poised to catch and treat inherited diseases at their ...

New gene therapy improves the life of 18-year-old with Sickle Cell Disease which affects 5,000 New Jersey residents living ...

Scientists at the San Raffaele Telethon Institute for Gene Therapy (SR-Tiget), Milan, have found that gene editing using ...