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FDA approvals—rare disease therapeutics
The FDA cleared Regeneron’s Pasatru (garetosmab) to treat fibrodysplasia ossificans progressiva (FOP), intensifying competition in an ultra-rare bone-loss market that already has Ipsen’s Sohonos....
Gene therapy—first approvals and commercialization path
Ultragenyx secured FDA approval for Glenglycos, its one-time gene therapy for glycogen storage disease type Ia (GSDIa), marking the company’s first marketable gene therapy. The liver-targeted...
Cancer vaccines—personalized neoantigen mRNA moves toward approval
Merck and Moderna announced that their personalized neoantigen mRNA vaccine, intismeran autogene, met a pivotal Phase 3 endpoint in high-risk melanoma, a result regulators and investors are now...
Phase 3 oncology—head-to-head competitiveness in targeted NSCLC
Dizal Pharmaceuticals’ sunvozertinib outperformed chemotherapy in a Phase 3 trial for first-line treatment of advanced NSCLC with EGFR exon 20 insertion mutations, extending progression-free...
Biotech dealmaking—reverse merger puts migraine antibody into public markets
Fulcrum Therapeutics agreed to combine with Slate Medicines through a $245 million reverse merger, creating a fast route to public markets for Slate’s migraine asset SLTE-1009. The program is a...
Diagnostics—NGS adoption uneven despite Medicare national coverage
A JAMA Network Open study using seven years of Medicare claims data found that uptake of next-generation sequencing (NGS) in cancer rose after CMS expanded national coverage in 2018, but access...
Digital health regulation—FDA’s TEMPO pilot advances for remote monitoring
The FDA is building out a more structured approach to evaluating digital health devices, with participation in its TEMPO pilot expanding across companies focused on remote monitoring and...
Biopharma partnerships—Deepcell and Gilead co-develop CHO cell-line morphology foundation model
Deepcell and Gilead Sciences announced a collaboration to co-develop a custom AI foundation model for Chinese hamster ovary (CHO) cell line development using single-cell morphology data. The...
Funding—Network Bio launches with $50M and Nvidia partnership
Network Bio launched with $50 million in financing and a collaboration valued at $30 million with Nvidia to develop a cell-free RNA (cfRNA) foundation model. The startup is assembling...
Regulatory and litigation—Parse Biosciences wins appeals over 10x patents
The U.S. Court of Appeals for the Federal Circuit upheld a Patent Trial and Appeal Board decision invalidating three 10x Genomics patents tied to single-cell nucleic-acid analysis. The ruling...
Personalized neoantigen mRNA vaccines deliver randomized Phase 3 signal in melanoma
Merck and Moderna reported success in a pivotal randomized late-stage trial testing intismeran autogene—a personalized mRNA neoantigen vaccine—added to Merck’s Keytruda (pembrolizumab) in resected...
FDA clears Regeneron’s activin A antibody for fibrodysplasia ossificans progressiva
The FDA approved Regeneron’s activin A-targeting monoclonal antibody garetosmab (Pasatru) for adults with fibrodysplasia ossificans progressiva (FOP), Regeneron’s second U.S. launch in the...
New Phase 3 win extends first-line benefit of EGFR exon 20 insertions drug
In a multinational Phase 3 trial, Dizal Pharmaceuticals’ sunvozertinib outperformed chemotherapy (carboplatin–pemetrexed) as first-line treatment for advanced NSCLC with EGFR exon 20 insertion...
Biotech dealmaking: BioMarin buys Alesta’s oral ALE1 for hypophosphatasia
BioMarin agreed to acquire Alesta Therapeutics in a transaction valued at $275 million upfront, plus up to $215 million in development and regulatory milestones, for Alesta’s lead candidate ALE1....
Cell and gene therapy operations: analytics automation targets cell therapy auditing at scale
BlueRock Therapeutics said it built an automated system for high-throughput auditing across cell therapy workflows, targeting a known gap between the rise of cell therapies and the availability of...
AI and data infrastructure: Network Bio launches with $50M and partners with NVIDIA
Network Bio launched with $50 million in financing and a collaboration valued at $30 million with NVIDIA to develop a cell-free RNA (cfRNA) foundation model. The startup said it will use...
Liquid biopsy expansion: Pillar Biosciences brings sequencing assay into Japan cfDNA/cfRNA screening
Pillar Biosciences said it will contribute its next-generation sequencing assay, oncoReveal Essential+, to a Japan partnership designed for liquid biopsy genomic screening. The program plans to...
Diagnostics evidence standardization: Diaceutics and PCC launch NGS concordance study
Diaceutics and the Precision Cancer Consortium (PCC) launched the NGS Concordance Study to benchmark variant detection across next-generation sequencing panels and platforms used in routine...
Regulatory and compliance scrutiny of AI medical devices
A broad review of artificial intelligence-enabled medical devices cleared by the FDA found that most were not tested to determine whether they improve patient outcomes. Researchers analyzed 1,357...
Epigenetic mechanism study: KDM2A loss restores antitumor immunity in liver cancer
A study in the British Journal of Cancer reports that removing KDM2A, a histone demethylase, can restore antitumor immunogenicity in liver cancer. The work places an epigenetic regulator at the...