Sarepta loses its Momentum
A clinical hold for the company’s Exondys 51 follow-on adds further pressure on the forthcoming gene therapy readouts.
EHA 2022 – Crispr still looks bloody good
Updated data with exa-cel in sickle cell disease and beta-thalassaemia could put the fear into competitors.
Go or no go? The FDA’s Amylyx dilemma
June will see adcoms aplenty, with Bluebird, Acadia, and the Covid vaccine makers Novavax, Pfizer/Biontech and Moderna all facing FDA committees.
Genetic medicine: the next generation
A new report from Evaluate Vantage looks at new approaches aiming to solve problems facing the current generation of genetic medicines.
Intellia’s in vivo Crispr stays in play, clinically at least
Though broad questions intensify, NTLA-2001 continues showing clinically what it is meant to.
