Gene therapy data shake Solid
Better-looking safety might not be enough to get Solid back in the Duchenne muscular dystrophy game.
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.
Bluebird split looks premature
Today’s safety scare with Lentiglobin calls into question the logic behind Bluebird’s planned business separation.
World Symposium – Orchard leads the crop of Hurler syndrome hopefuls
Promising early data with the group’s gene therapy put it ahead of Regenxbio for now.
Bayer’s gene therapy juggling act
Two arm’s-length acquisitions and two licensing deals have given Bayer a cell and gene therapy unit; now to bring it all together.
Pivotal valrox data give Biomarin déjà vu
Waning factor VIII levels mean the durability question is still unanswered.
Gene therapy trial fails to rectify Sarepta’s sorry record
Sarepta’s first ever controlled study, of a Duchenne muscular dystrophy gene therapy, brings the markets down to earth.
