Science ❯ Biotechnology ❯ Genetic Engineering

Therapeutic Applications

Clinical Trials Viralgen Gene Editing Gene Therapy Safety Animal Models

One Infusion Cuts LDL by Up to 62% in Early Gene‑Editing Trial

Interim NEJM data show dose‑dependent PCSK9 knockdown with an initially favorable safety signal prompting Fast Track status.

Sarepta Defies FDA, Keeps Duchenne Gene Therapy on the Market