Gene therapies go skin deep to tackle epidermolysis bullosa
Three gene therapy contenders, Abeona, Fibrocell and Krystal, are taking different approaches in the quest to cure epidermolysis bullosa.
Auris acquisition could prompt more mega VC rounds
The J&J-Auris tie-up will yield a huge return for investors who went in big.
Another knock back for urea cycle disorders
Curevac’s decision to exit work on an mRNA approach to OTC deficiency follows a clinical hold on Translate Bio’s similar project, and raises broader concerns.
Novartis and Gilead’s multiple myeloma CARs diverge
As Gilead bows out of BCMA, Novartis pins its hopes on a BCMA/CD19 combo.
The list of shame no biopharma wants to join
An upcoming Icer report will put the spotlight on unjustified price increases even as signs of moderation emerge.
In sticking to the knitting has Array given up too much?
At a time when most of biotech seems to want to become “the next Genentech” Array Biopharma has stuck to a partnering model and, in hindsight, lost out in the process.
Gene therapy: how much will it cost patients?
Bluebird Bio has pushed forward the conversation on how to make million-dollar treatments affordable for insurers, but biopharma and payers are ominously quiet about…
Round two for Axsome
Fresh from a mid-stage success in depression Axsome offers an unusual way to trade its upcoming phase III result.
Smaller groups await Bristol-Myers Squelgene fallout
Celgene had many fingers in many pies – so what will happen now to its smaller collaborators?
Kickstarting ideas
Independent, data-driven daily news and analysis on pharma, biotech and medtech.