ESGCT 2021 – Sio strengthens its case in GM1 gangliosidosis
A high dose of AXO-AAV-GM1 impresses, but small patient numbers provide a reason for caution.
Smaller companies set for third-quarter catalysts
The third quarter will see important data for Travere, Radius and Sio Gene, among other small developers.
Jury still out on Sarepta’s next generation
The group’s lead next-gen exon skipper beats a low bar set by Exondys, but adverse events loom large.
A three way battle beckons in GM1 gangliosidosis
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.
Plenty of choice remains for buyers of gene therapy
Despite the recent flurry of gene therapy deals there is still a fairly extensive list of affordable companies out there.
First-quarter floats see haves pulling away from the have-nots
Demand for new issues remains sluggish - only eight private biotechs managed to pull off an IPO in the first quarter of 2017.