Only a few patients have undergone an intensive gene therapy cure for sickle cell disease since it was approved by the FDA due to it's high cost and limited access. NBC News' Yamiche Alcindor spoke ...

A phase 3 trial highlights betibeglogene autotemcel as a potentially curative gene therapy for severe transfusion-dependent β ...

Capricor Therapeutics has completed its US marketing application for deramiocel, a cell therapy the US biotech hopes will ...

Novartis reported that its intrathecal (IT) formulation of onasemnogene abeparvovec (OAV101 IT) reached its primary endpoint in a Phase III study in paediatric patients aged two to 17 with type II ...

Shares of Precigen PGEN have soared nearly 43% in the past week after the company provided an encouraging regulatory update ...

The pharma giant pulled out of its partnership with Sangamo, signalling unsteady waters for the latter company and the future ...

Two Philadelphia biotech companies scored approval in 2024 for first-of-their-kind gene therapy treatments for solid cancer tumors and melanoma. Those achievements by Iovance Biotherapeutics and ...

Muscular Dystrophy News Today covered the latest studies, treatment advances, and clinical trials for different types of the ...

Learn about pediatric low-grade glioma symptoms, causes, diagnosis, and treatment. Understand risk factors and prevention to ...

Pfizer gave Sangamo Therapeutics coal for Christmas, terminating a hemophilia A gene therapy pact to deprive the ...

Dec. 10, 2024 — In a single IV injection, a gene therapy targeting cBIN1 can reverse the effects of heart failure and restore heart function in a large animal model. The therapy increases the ...