Tag: #rarediseases

After One Baby’s Custom CRISPR Rescue, a Startup Bets on Made-to-Order Gene Editing

A one-off CRISPR therapy saved a baby with a lethal mutation. Now a Doudna-backed startup and a new FDA pathway aim to scale bespoke gene medicines.

#crispr, #geneediting, #rarediseases, #fda, #biotech

First Successful Personalized CRISPR Gene Therapy Offers New Hope for Rare Genetic Diseases

Nine-month-old receives groundbreaking CRISPR therapy for rare genetic disorder, marking a milestone in precision medicine.

#CRISPR, #genetherapy, #rarediseases, #precisionmedicine