For how we live.
The agency issued the tradable voucher to Ultragenyx for FAYUVI, an AAV gene therapy for the neurologic features of MPS IIIA that it approved on 17 September 2026. Such vouchers can be redeemed or sold.
A European registry compared 52 transplanted patients with 79 untreated ones over up to 25 years. The benefit was clearest when transplant came before symptoms, and it did not stop progression entirely.
An independent 2026 meta-analysis of AAV micro-dystrophin trials found small, mostly uncontrolled functional benefits — largest in the youngest boys — set against serious harms including rare deaths.
Suppressor tRNAs have been a promising idea for nonsense mutations for years, held back by weak activity and delivery. A Science paper reports one chemical tweak and one redesigned nanoparticle.
GENGLYCOS treats the cause of glycogen storage disease type Ia, not just the symptoms cornstarch regimens manage — but it carries a long list of serious risks and an accelerated-approval asterisk.
Three of the four Yamanaka factors, delivered to one eye and switched on with an antibiotic for eight weeks. The mouse data are striking; the trial is a safety study and will not measure ageing.
In two industry-funded phase 3 studies, every child followed at least 16 months met the goal — but all had a grade 3 or 4 event, and one died of a conditioning-drug complication.
A modelling analysis puts 2.75 million children under five in sub-Saharan Africa living with sickle cell disease. Seven African countries have transplant programmes; approved gene therapies cost millions.
ETX101 doesn't replace the faulty gene behind Dravet syndrome — it turns up the healthy copy that's still there. New data from 21 children show the effect holding for a year.
27 of 28 patients had no severe pain crises after treatment with reni-cel. But the sponsor ended the study early, and the reported analysis was not the one it was designed to test.
The draft Plausible Mechanism Framework would let a well-supported mechanism of action carry weight where patient populations are too small for randomisation. It is open for comment for 60 days.
Nine children received a single infusion of AAV9 carrying the beta-galactosidase gene. Enzyme activity rose and substrate fell in every participant; some developmental measures held steady and others declined.
The CHMP's November meeting produced ten positive opinions. Two of them — teplizumab and a Telethon-sponsored gene therapy — address conditions the EU has had no authorised treatment for.