Here is a roundup of the most important news in biotech and medicine this morning.
The need-to-know this morning
- Beam Therapeutics announced that its CRISPR therapy risto‑cel met the trial’s primary endpoint, providing freedom from severe pain crises for at least 12 months. The company will submit an FDA marketing application by year‑end, though detailed efficacy data have not yet been released.
PepGen shows promising data for muscular dystrophy
PepGen reported that its investigational therapy delivered the most encouraging early results to date for a prevalent form of muscular dystrophy, based on a key efficacy metric.
Also Read
- AMA Enters the Fray to Combat Medical Misinformation
- STAT+: PepGen says early trial for muscular dystrophy drug looks promising
- When a Gun Owner Shows Early Signs of Dementia
- Japanese Man’s Severe Head Tremor and Cognitive Decline Linked to Rare Aqueductal Web, Resolved by Ventriculoperitoneal Shunt

