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 ...

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

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

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

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

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

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

Sangamo is on course to run out of money within months and has now lost access to up to $220 million in milestone payments ...

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 ...

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 ...