Bayer's High Hopes For Huntington's Gene Therapy As Novartis’s Branaplam Stumbles
France Gives Thumbs-Up To Trial
When the German major bought AskBio a couple of years ago, one of its assets was a gene therapy for Huntington’s, a devastating disease that leads to complete physical and mental deterioration. That asset, BV-101, is about to enter the clinic.
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Hopes rise as first four evaluable patients experienced no serious adverse events, and a sustained decline in level of key Huntington’s disease protein with the novel gene therapy product.
The company now turns to its next-generation Huntington drug, WVE-003.
Roche’s Phase III of tominersen in Huntington's disease has been stopped after an independent interim review, but with no new or emerging safety signals reported, analysts presume efficacy was the problem.