Growing demand for innovative, user-friendly drug delivery devices, driven by decentralized healthcare, advanced digital integration, and strategic regulatory collaboration. Cell & Gene Therapy Drug ...

Forbes contributors publish independent expert analyses and insights. A patient in a late-stage gene editing trial for a rare heart condition died from fatal liver complications after receiving an ...

Thanks to advances in imaging and diagnostic technologies, clinicians can now detect many genetic disorders in the womb, ...

Dustin Vidrine's sight is disappearing — shrinking, really. He has retinitis pigmentosa, a rare, inherited eye disease that runs in his family. By the time he was in his 20s, he noticed that his ...

Onasemnogene abeparvovec gene therapy post-nusinersen or risdiplam showed meaningful motor improvements in children with SMA, with a manageable safety profile. The study cohort included older, heavier ...

In a stunning medical breakthrough, scientists used customized CRISPR gene editing to save a baby’s life from a rare, previously incurable liver disorder—CPS1 deficiency—just six months after birth.

February 25, 2026 marks one year since KJ, an infant born with severe carbamoyl phosphate synthetase 1 (CPS1) deficiency, became the world's first person to receive a personalized CRISPR-based gene ...