Gene therapy clinical holds take centre stage
An analysis of company disclosures reveals a sharp rise in US FDA-imposed halts on clinical work involving gene therapies.
Ash 2019 preview – the first winner and losers emerge
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 goes early to leapfrog haemophilia gene therapies
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.
Vertex spends nearly $1bn on Semma's unproven diabetes cell tech
Having succeeded with cystic fibrosis Vertex is now chasing a cure for type 1 diabetes.
EHA 2019 – Global Blood faces sickle cell questions
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.
Emmaus tries to move forwards by reversing
The company insists that its sickle cell disease therapy Endari still has a future, despite questionable efficacy and the approach of new treatments.
Vertex’s second transformation: Duchenne time
The cystic fibrosis player does deals with Crispr and Exonics to launch itself into a highly competitive rare disease area.
Upcoming events – Protagonist and Pfizer target rare diseases
Protagonist Therapeutics and Pfizer will soon get an idea of their respective chances in beta-thalassaemia and Duchenne muscular dystrophy.
Vertex and Crispr up the ante in gene therapy chase
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.
