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A recent FDA reversal sparked new hope for patients with Huntington’s disease. Flying under the radar, Skyhawk Therapeutics revealed 12-month functional data from a midstage trial of its own candidate showing improvements on a key disease measurement scale.
Recent approvals for Corcept Therapeutics and Merck have injected momentum into the space, where GSK, Allarity Therapeutics, OSE Immunotherapies and others are advancing their own candidates.
The FDA plans to hold an advisory committee meeting to discuss Capricor Therapeutics’ application for deramiocel, which the agency rejected last July. The news surprised CEO Linda Marbán, who told BioSpace the FDA has not communicated any issues of concern with the company’s resubmitted application.
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THE LATEST
Neurocrine Biosciences and Voyager Therapeutics inked a CNS-targeted collaboration potentially worth a potential $4.4 billion.
Twist Bioscience announced a partnership with Astellas Pharma on Monday, seeking to discover antibodies against several targets to hit on curative therapies for hard-to-treat diseases.
Takeda and Arrowhead Pharmaceuticals’ Phase II trial studying fazirsiran, an investigational RNA therapeutic developed for AATD-LD, was effective in Phase II–but so was the placebo.
Day One Biopharmaceutical reported positive topline data from a Phase II trial in pediatric low-grade glioma, confirming earlier results.
Italian biopharma Chiesi Farmaceutici SpA announced Sunday it was acquiring rare disease-focused Amryt Pharma in a deal that could reach up to $1.48 billion in value.
The pharma titan adds an intriguing blood pressure medication to its line up with a buyout of CinCor.
Ipsen bolsters rare disease portfolio with acquisition of liver disease specialist Albireo.
The FDA approved Eisai and Biogen’s lecanemab (Leqembi) Friday afternoon. It is the second anti-amyloid antibody to be approved for Alzheimer’s disease in two years.
PCN-101, a ketamine-based candidate for treatment-resistant depression being developed by Atai’s Perception Neuroscience, fell short in a Phase II trial.
Thursday, Fate Therapeutics announced it terminated the 2020 collaboration agreement with Johnson & Johnson’s biopharma subsidiary Janssen.