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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
Graphite Bio announced it is voluntarily pausing the Phase I/II study of its lead candidate nula-cel in sickle cell disease.
Moderna and CytomX ink a cancer partnership.
In another early high-value deal, Amgen inked a licensing agreement with Synaffix for the development of next-generation antibody-drug conjugates.
NovoCure’s novel cancer-fighting platform hits the mark in non-small cell lung cancer.
Metagenomi announced a Series B extension financing round led by Moderna, Leaps by Bayer and several others.
A letter published Wednesday in the NEJM links a stroke patient’s death to lecanemab. Eisai investigators respond.
Galmed announces positive late-stage results in biopsy-proven NASH patients.
In a recently published study, MD Anderson researchers achieved curative responses in pancreatic cancer models by targeting checkpoints in T cells and myeloid suppressor cells.
Geron announced Wednesday positive topline results from the Phase III IMerge trial evaluating the telomerase inhibitor, imetelstat, in lower-risk myelodysplastic syndromes patients.
Backed by Versant and Genentech, Belharra Therapeutics launched Wednesday with $130 million in total financing to advance its novel chemoproteomics platform.