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FDA Grants Priority Review to Chimerix for Dordaviprone in H3 K27M-Mutant Diffuse Glioma
In a Phase III trial, 28 percent of newly diagnosed patients with H3 K27M-mutant diffuse glioma responded to treatment with dordaviprone.
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Merck's Welireg Approved in Europe for Von Hippel-Lindau-Associated Cancers
The European Commission granted approval for von Hippel-Lindau-associated renal cell carcinoma, CNS hemangioblastomas, or pancreatic neuroendocrine tumors.
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FDA Grants Priority Review to Ultragenyx's BLA for Sanfilippo Syndrome Type A Gene Therapy
The firm is seeking accelerated approval for UX111 using reductions in levels of heparan sulfate in cerebrospinal fluid as a surrogate endpoint.
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BC Cancer Foundation Launches C$6.8M Campaign to Advance CAR T-Cell Therapies
The foundation hopes to put the funds toward two clinical programs in blood cancers and one preclinical project in solid tumors.
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Restore Vision Doses First Retinitis Pigmentosa Patient in Phase I/II Gene Therapy Trial
The Tokyo-based biotech firm expects RV-001 to be able to alter light sensitivity for patients with limited or no vision regardless of genotype.
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Aptose Biosciences Enters $25M Common Shares Purchase Agreement
NEW YORK – Aptose Biosciences on Thursday said it has entered into a common share purchase agreement with an institutional investor, which it expects will bring in around $2.1 million in gross ...
precisionmedicineonline5d
More Precise Response Markers Needed for Enhertu Amid HER2 Biomarker Disruption, Researchers Say
By analyzing ERBB2 mRNA expression data, researchers identified a group of HER2-negative patients with poor outcomes and potential markers of treatment resistance.