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Muscular dystrophy epigenetic therapy funding
Epicrispr Biotechnologies secured $90 million in a Series C round after sharing early data for EPI-321, an epigenetic editing approach targeting facioscapulohumeral muscular dystrophy (FSHD). The...
Eczema antibody race heats up with new financing
Infinimmune launched its eczema push with a $75 million Series A, aiming to deliver next-generation biologics for atopic dermatitis that could improve on Dupixent’s pace and injection burden. The...
Cystic fibrosis setback reshapes Vertex competition
Sionna Therapeutics reported that its Phase 2 cystic fibrosis add-on pill, SION-719, failed to show benefit when added to Vertex’s Trikafta, prompting the biotech to stop advancing the program and...
Gene therapy trial startup launches in deafness
Skylar Bio dosed its first patient in a new gene therapy effort for childhood hearing loss linked to GJB2 mutations, building on the field’s momentum after FDA approval of Regeneron’s...
Radiopharma manufacturing rejection impacts competitive landscape
The FDA rejected ITM Isotope Technologies Munich’s radiopharmaceutical candidate intended for neuroendocrine tumors, citing manufacturing problems that delayed a potential challenger to Novartis’...
Venture-backed base editing off-target testing platform
Broken String Biosciences launched BaseMap ABE, a platform meant to standardize genome-wide off-target characterization for adenine base editing (ABE) therapeutic development. The company said the...
Oncology companion diagnostic cleared by FDA
Labcorp received FDA approval for PGDx elio tissue complete as a companion diagnostic for detecting BRAF V600E and V600K mutations in melanoma, supporting patient selection for targeted therapies...
Regulatory framework for medical devices—CMS RAPID pathway advances
CMS issued proposed details for the RAPID pathway, an effort to reduce the gap between FDA breakthrough device approvals and Medicare coverage. The program is designed to address “coverage...
China tightens clinical trial rules affecting speed model
China tightened controls on clinical trials via a regulatory shift that is reshaping the country’s previously fast translation model. The reporting focuses on investigator-initiated trials (ITTs),...
Ecosystem manufacturing: BMS picks Houston for $2.3B campus
Bristol Myers Squibb selected Houston as the location for a $2.3 billion multi-modal manufacturing campus supporting drug product and finished goods manufacturing from late development through...
Regulatory approval – mRNA flu vaccine
The FDA approved Moderna’s seasonal influenza mRNA vaccine mRNA-1010 (mFLUSIVA), marking the first approved flu shot built on an mRNA platform. The decision followed a unanimous VRBPAC vote,...
Biopharma reshoring – BMS manufacturing campus
Bristol Myers Squibb selected Houston for a new $2.3 billion multi-modal manufacturing campus designed to support drug product and finished goods from late development through launch. The site...
Clinical setback – cystic fibrosis add-on failure
Sionna Therapeutics said its cystic fibrosis strategy hit a wall in a Phase 2 trial, reporting that SION-719 did not provide additive benefit when combined with Vertex’s Trikafta. The company now...
Rare disease M&A – Jazz acquires Actio
Jazz Pharmaceuticals agreed to acquire Actio Biosciences in a deal valued at just over $1.3 billion, consisting of $820 million upfront plus up to $500 million in milestone payments tied to...
Rare blood cancer trial win – Silence Phase 2
Silence Therapeutics reported Phase 2 success for its siRNA therapy divesiran in polycythemia vera, hitting the trial’s primary endpoint in the 48-person study. Silence said 88% of patients...
Cell therapy decision support – AML transplant model
A new gene-expression risk model is being used to personalize allogeneic stem cell transplant decisions for children with AML, aiming to refine who benefits most from allo-HSCT. The approach...
Companion diagnostics – Labcorp FDA approval
The FDA approved Labcorp’s PGDx elio tissue complete as a companion diagnostic for detecting BRAF V600E and V600K mutations in melanoma. Labcorp said the approval enables patient selection for...
Weight management access – Medicare GLP-1 Bridge pricing
CMS’s Medicare GLP-1 Bridge program is reshaping weight management pricing by setting a flat $50 monthly copay point for eligible beneficiaries through December 31, 2027. The program runs outside...
Oncology transplant/cancer diagnostics – Natera MRD and early cancer push
Natera said it is expanding beyond its existing MRD footprint after a series of regulatory milestones for Signatera. The company received FDA approval for companion diagnostic use in...
Drug safety and clinical trial operations – ITTs under China tightening
China is tightening oversight of investigator-initiated trials (ITTs), a model that has helped accelerate trial starts but has increasingly drawn scrutiny. A STAT+ report said new enforcement is...