Early 2021 will see rare diseases dominate for biotech, with Sarepta revealing key data on its gene therapy play.
An $880m buyout by Lilly shows that some gene therapy companies have not yet priced themselves out of the market.
IGM’s snowballing share price belies some other major Ash winners, and will make Christmas unhappy for funds that had shorted the stock.
And both companies’ pyruvate kinase R activators could have Global Blood looking over its shoulder, though pain crises sow doubts.
Crispr Therapeutics might have stolen the Ash headlines, but a similar approach from Bluebird is also making progress.
Data in three more patients support Crispr and Vertex’s hope of developing a functional cure for beta-thalassaemia and sickle cell disease.
Among the upcoming haematology meeting’s non-oncology presentations Uniqure’s haemophilia B gene therapy scores a valuable late-breaker.