crispr
4 stories tagged "crispr"
The FDA Cleared a CRISPR Cure for Two-Year-Olds Whose Own Trial Enrolled No One That Young
Casgevy's pediatric trials enrolled 11 children aged five and up, yet the FDA extended the $2.2 million therapy down to age two, where the conditioning chemo can permanently sterilize a child too young to bank eggs or sperm.
CancerThe Reason Immunotherapy Bounces Off Prostate Cancer: Tumors Chew Off a Genetic Tail to Blind the Immune System
Prostate tumors overproduce a protein that destroys the immune system's docking targets. A CRISPR tool that blocks the trick made checkpoint drugs work again, so far only in mice.
InvestigationsA Gene-Editing Trial Killed a Six-Year-Old. The Only Official Penalty Was a $3,500 Fine.
Xinhua Hospital paid about 24,000 yuan in September 2025. The death it was fined over stayed out of the medical literature for another ten months, and out of the Nature paper entirely.
Drugs & FDAThe FDA Cleared a Sickle-Cell Cure for Two-Year-Olds in 53 Days. It Starts With Chemo That Can Sterilize Them.
Casgevy is a real advance against a brutal disease. It is also a $2.2 million therapy that requires sterilizing chemotherapy, saw a child die in trials, and reached toddlers on a 53-day review Makary's FDA hands out as a favor.