Gene therapies are raising hopes for patients with rare disorders, various cancers, and complex neurological diseases. But there are ethical and policy challenges on the path from research to ...

Cas9 in germline editing presents opportunities for genetic disease correction, alongside risks of off-target effects and ...

Scientists from Spirovant Sciences describe SP-101, a novel adeno-associated virus (AAV) gene therapy for the treatment of cystic fibrosis (CF) in the peer-reviewed journal Human Gene Therapy.

Showcased 7 presentations at ISSCR 2025, including a featured oral presentation on first-in-human gene therapy for heart failure, powered by their proprietary human mini-Heart technology. FDA ...

Researchers at the University of Minnesota have completed a first-in-human clinical trial testing a CRISPR/Cas9 gene-editing technique to help the immune system fight advanced gastrointestinal (GI ...

Medera Presented Updated Results from First-In-Human Gene Therapy Trial for Heart Failure with Preserved Ejection Fraction at 2025 HFpEF Summit Provided by GlobeNewswire Apr 1, 2025, 12:00:00 PM ...

Jayden Wilsey, 15, of Falmouth, is undergoing human gene therapy for sickle cell disease at Boston Children's Hospital. The hospital's sickle cell program currently has 450 patients under the age ...