Challenge trials help researchers study immune responses. Skeptics still doubt the approach is worth the risks ...

The French firm will use the proceeds to advance its ALIGATER platform for delivering genetic payloads and move its preclinical assets toward the clinic.

Financing co-led by Novo Holdings A/S and Bpifrance (as part of the InnoBio investment strategy) and joined by leading US and EU investors Invus ...

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

A single amino acid change in a neuronal ion channel called KCNQ2 blocks ion flow, prevents protein localization on axons, and results in severe epilepsy and slowed neurological development.