A high dose of AXO-AAV-GM1 impresses, but small patient numbers provide a reason for caution.
Data are due in the fourth quarter for Aldeyra, Aeglea and Radius, among other small developers.
The third quarter will see important data for Travere, Radius and Sio Gene, among other small developers.
The second quarter brings updates for Scholar Rock, Alnylam and Dicerna, among others.
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.
An $880m buyout by Lilly shows that some gene therapy companies have not yet priced themselves out of the market.
Venture-backed companies are floating earlier, but time to takeout stays stubbornly steady.