Gene-editing kicked off 2024 with a bang in the medical sector but closed the year with a whimper. It started with the FDA ...

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

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

In a finding with implications for one of the most promising cutting-edge medical treatments, researchers evaluated several ...

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

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

Sangamo, a gene therapy pioneer, loses a financial lifeline as Pfizer drops a gene therapy program for hemophilia.

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

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 ...

Bryan Johnson, who’s spending hundreds of millions of dollars on treatments designed to extend his life, is many things to ...

GEMMA Biotherapeutics, founded by Jim Wilson earlier this year, will use the money to expand its operations and gene therapy ...

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 ...