An analysis of company disclosures reveals a sharp rise in US FDA-imposed halts on clinical work involving gene therapies.
Celgene’s registrational Transcend NHL-001 study will be a big story at Ash, but the early focus falls on what has not been presented.
Novo Nordisk took an early step towards genome editing with yesterday’s Bluebird tie-up, and isn’t ruling out a move into conventional gene therapies.
Having succeeded with cystic fibrosis Vertex is now chasing a cure for type 1 diabetes.
More data with voxelotor shouldn’t hurt the project’s bid for accelerated approval, but there are still doubts about its effect on a harder patient outcome.
The company insists that its sickle cell disease therapy Endari still has a future, despite questionable efficacy and the approach of new treatments.
The cystic fibrosis player does deals with Crispr and Exonics to launch itself into a highly competitive rare disease area.
Protagonist Therapeutics and Pfizer will soon get an idea of their respective chances in beta-thalassaemia and Duchenne muscular dystrophy.
The first sickle cell and beta thalassaemia patients have been enrolled into a Crispr/Cas9 trial, heating up a race with Bluebird, Sanofi, Sangamo and Editas.