Better-looking safety might not be enough to get Solid back in the Duchenne muscular dystrophy game.
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.
Today’s safety scare with Lentiglobin calls into question the logic behind Bluebird’s planned business separation.
Promising early data with the group’s gene therapy put it ahead of Regenxbio for now.
Two arm’s-length acquisitions and two licensing deals have given Bayer a cell and gene therapy unit; now to bring it all together.
Waning factor VIII levels mean the durability question is still unanswered.
Sarepta’s first ever controlled study, of a Duchenne muscular dystrophy gene therapy, brings the markets down to earth.