Novartis has recently announced that its intrathecal (IT) formulation of onasemnogene abeparvovec (OAV101 IT) successfully ...

JCR Pharma & Modalis validate initial proof of concept in a joint research programme for development of a novel gene therapy for a CNS disease: Hyogo, Japan Tuesday, January 7, 20 ...

Vertex is paying Orna $65 million upfront, in part via convertible note, according to a Jan. 7 release. Orna—which acquired ...

The science has proven that gene therapies are effective treatments, and they have given hope to patients and their families ...

Only a few patients have undergone an intensive gene therapy cure for sickle cell disease since it was approved by the FDA ...

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 ...

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

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 ...