A high dose of AXO-AAV-GM1 impresses, but small patient numbers provide a reason for caution.
The third quarter will see important data for Travere, Radius and Sio Gene, among other small developers.
After revealing plans to buy one of its holdings out of a Spac, Roivant itself moves towards a blank-cheque company listing.
Immunovant, the poster child for the hot new biotech flotation vehicle, might soon go private again.
As several big pharma companies drop Parkinson’s projects, attention turns to gene therapies and repurposing diabetes drugs.
Early data could give clues about whether gene therapies from Sio, Passage Bio or Lysogene will come out on top in this rare inherited disease.