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.
An analysis looking at forecast discrepancies for the top pipeline projects reveals vast differences of opinion about Vertex's cystic fibrosis triplet and Novo…
Rivipansel needs to help sickle cell patients leave hospital sooner and BCX7353 faces established hereditary angioedema agents.
After Monday’s celebrations the sector gets down to discussing the difficult business of actually selling and making money from drugs.
Autolus leads winners in spite of scepticism over its CAR-T, and gene therapies disappoint.
The latest Lentiglobin data impress, but launch worries could clip Bluebird’s wings.
Investors had been awaiting an update on voxelotor with trepidation, but a lenient FDA has lit up the company’s Ash.
Swedish Orphan Biovitrum made waves yesterday by getting its rare disease asset emapalumab into the Ash late-breakers – and bagging approval soon afterwards.