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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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Read our takes on the biggest stories happening in the industry.
If cell and gene therapy makers are going to achieve their mission to improve patients’ lives, the industry must come together to share information across stakeholders, from regulators to manufacturers to payers.
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Psychedelic and psychedelic-assisted therapies in the neuropsychiatric space have stalled for decades.
Madrigal’s NASH drug hits the mark in late-stage trial.
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Ocugen gets the go-ahead from the FDA to trial its late-stage regenerative cell therapy.
Astellas’ zolbetuximab hit the mark in a second late-stage trial in Claudin-18.2-positive gastric cancer.
Covalent biologic company Enlaza launches with $61M in seed financing.
Lilly digs deeper into metabolic diseases via GPCR partnership with Sosei.
FDA
BioCryst Pharmaceuticals is discontinuing the development of its Factor D inhibitor BCX9930 in paroxysmal nocturnal hemoglobinuria due to competitive factors.