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.
The group’s lead next-gen exon skipper beats a low bar set by Exondys, but adverse events loom large.
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.
Despite the recent flurry of gene therapy deals there is still a fairly extensive list of affordable companies out there.
Demand for new issues remains sluggish - only eight private biotechs managed to pull off an IPO in the first quarter of 2017.