For how we live.
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.
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.