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Personalized mRNA cancer vaccine delivers Phase 3 success
Merck and Moderna announced Phase 3 success for their personalized neoantigen mRNA vaccine intismeran autogene in adjuvant melanoma, reporting a statistically significant improvement in reducing...
FDA approvals expand rare bone disease options
The FDA cleared Regeneron’s activin A-targeting antibody garetosmab, branded Pasatru, for fibrodysplasia ossificans progressiva (FOP). The approval follows Phase 3 results showing reductions in...
Ultragenyx secures first gene therapy approval for ultra-rare GSDIa
Ultragenyx won accelerated FDA approval for Genglycos (pariglasgene brecaparvovec; DTX-401), marking the first approved treatment for glycogen storage disease type Ia (GSDIa). The AAV8 gene...
Silence Therapeutics advances siRNA drug for polycythemia vera
Silence Therapeutics reported positive Phase II results for divesiran, an siRNA targeting TMPRSS6 for polycythemia vera (PV). In the SANRECO trial (NCT05499013), the program met its primary...
Abcuro funds registrational study for inclusion body myositis antibody
Abcuro raised $66 million in a Series D financing to support a registrational clinical study for ulviprubart, its monoclonal antibody targeting cytotoxic T cells in inclusion body myositis (IBM)....
BioMarin buys Alesta for oral bone disease therapy
BioMarin agreed to acquire Alesta Therapeutics for $275 million upfront, plus up to $215 million in development and regulatory milestones, expanding its rare bone-disease pipeline. The deal brings...
Network Bio launches with $50M and Nvidia partnership for cfRNA foundation models
Network Bio launched with $50 million in financing and a collaboration valued at $30 million with Nvidia to develop cell-free RNA (cfRNA) foundation models. The startup says it is assembling...
Deepcell and Gilead co-develop CHO cell morphology AI foundation model
Deepcell and Gilead Sciences entered a partnership to co-develop a custom AI foundation model for Chinese hamster ovary (CHO) cell line development using morphology data. Deepcell will image...
FDA, diagnostic and oncology systems: benchmarking and coverage issues
Two parallel developments spotlight the operational pressure building around next-generation sequencing (NGS) in oncology. First, Diaceutics and the Precision Cancer Consortium launched an NGS...
Phase I safety pause in Boehringer program
Boehringer Ingelheim suspended a Phase 1 trial of an investigational small molecule after what it described as a safety event. The German pharma said it is pausing the study while it seeks to...
Personalized cancer mRNA immunotherapy scores Phase 3 win
Merck and Moderna said their personalized neoantigen mRNA vaccine, intismeran autogene, achieved a Phase 3 success in adjuvant melanoma, adding meaningful competition to the neoantigen-vaccine...
FDA clears Ultragenyx’s first marketed gene therapy
The FDA cleared Ultragenyx’s one-time AAV gene therapy Genglycos (pariglasgene brecaparvovec; DTX-401) for glycogen storage disease type Ia (GSDIa), giving the company its first approved gene...
Regeneron’s activin A antibody wins FDA approval for FOP
Regeneron’s garetosmab, branded Pasatru, won FDA approval for fibrodysplasia ossificans progressiva (FOP), adding a second approved therapy for the ultra-rare bone-forming disorder. The antibody...
Rare bone disease M&A—BioMarin buys Alesta’s oral HPP candidate
BioMarin struck a deal to acquire Alesta Therapeutics for up to $490 million, taking on ALE1, a clinical-stage oral candidate for hypophosphatasia (HPP). The transaction includes $275 million up...
Immuno-oncology AI tooling gets new industry backing
Deepcell and Gilead agreed to co-develop an AI foundation model for Chinese hamster ovary (CHO) cell line development using single-cell morphology data. Under the collaboration, Deepcell will...
Cell-free RNA AI and Nvidia collaboration
Network Bio launched with $50 million in financing and signed a collaboration valued at roughly $30 million with Nvidia to build a cell-free RNA foundation model. The program targets an “AI-ready”...
Rare blood cancer siRNA hits Phase 2 primary endpoint
Silence Therapeutics reported positive Phase II results for divesiran, an siRNA therapy targeting TMPRSS6 in polycythemia vera (PV). In the SANRECO trial (NCT05499013), divesiran met its primary...
Startup funding—Abcuro raises $66m to expand inclusion body myositis study
Abcuro raised $66 million in a Series D to advance ulviprubart for inclusion body myositis (IBM), a progressive muscle disorder with no FDA-approved therapies. The company said the funding will...
AI for diagnostics quality—TRUECAM aims to flag uncertainty
PolyU researchers unveiled TRUECAM, a framework designed to show when pathology AI predictions are reliable versus uncertain, and when a human pathologist should take over. The system is built...
Innovation in genomic medicine manufacturing—Fulcrum reverse merger closes public-market path
Fulcrum Therapeutics and Slate Medicines agreed to combine via a $245 million reverse merger, creating a faster route to public markets for Fulcrum’s and Slate’s migraine programs. The merged...