Solid Biosciences’ (SLDB) data this morning from the first six patients to take its experimental gene therapy for Duchenne muscular dystrophy, SGT-001, falls below the expectations of Wall Street analysts, Matthew Herper of STAT reports. lan Ganot, the co-founder and CEO of Solid, told him, “Look, as background, we are a company that was formed to discover, develop, and deliver meaningful therapies for patients with DMD. We believe SGT-001 has the potential to be a meaningful therapy for patients with DMD, and obviously are committed to move forward as fast as we can.” A rival gene therapy being developed by Sarepta Therapeutics (SRPT) has shown much more dramatic results in terms of the amount of modified dystrophin produced and apparent benefits to patients, Herper writes. Ganot, however, says Solid is encouraged simply that there are signs of expression even at the starting dose.
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