Study supports favorable safety and tolerability profile and clinically meaningful functional and structural benefits in Stargardt disease ...

PHILADELPHIA, April 17, 2025 /PRNewswire/ -- Researchers at Children's Hospital of Philadelphia (CHOP) in collaboration with researchers at Royal Prince Alfred Hospital in Sydney, Australia announced ...

A new gene therapy can reverse the effects of heart failure and restore heart function in a large animal model. The therapy increases the amount of blood the heart can pump and dramatically improves ...

SGT-212 has received FDA Fast Track, Rare Pediatric Disease and Orphan Drug designations -- Dosing of the first participant in the Phase 1b ...

A retired HGV driver was the first person in the world to take part in a clinical trial to extend the lifespan of blood ...

MSK is the first hospital in New York City to offer gene therapy through its pediatric cancer program MSK Kids. Exagamglogene autotemcel (exa-cel) (Casgevy®) is a gene therapy for treating sickle cell ...

When Domenic Catrine was born, his heel swelled up from his vitamin K shot. His circumcision site wouldn't stop bleeding. At the Children's Hospital of Philadelphia, Catrine was diagnosed as having ...

Atsena Therapeutics, a clinical-stage gene therapy company focused on using the life-changing power of genetic medicine to reverse or prevent blindness, today announced that dosing is complete in ...

A significant milestone in Ocugen's development of a gene therapy for Stargardt disease has triggered a substantial rally in the company's stock. The catalyst was the publication of positive Phase 1 ...

Forbes contributors publish independent expert analyses and insights. Juergen Eckhardt leads Bayer’s impact investment unit, Leaps by Bayer. Some of the year's notable breakthroughs include the first ...

The gene therapy market is surging toward $36.55 billion by 2032, powered by rising demand for curative treatments that target genetic diseases at their source([1]) . The FDA approved three ...