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Regulatory approvals: rare disease gene and biologic milestones
The FDA cleared Ultragenyx’s one-time gene therapy Genglycos (pariglasgene brecaparvovec; DTX-401) for glycogen storage disease type Ia (GSDIa), marking the treatment’s accelerated approval and...
Oncology: personalized mRNA neoantigen vaccine clears late-stage melanoma hurdle
Merck and Moderna reported that their personalized mRNA neoantigen vaccine, intismeran autogene, met key goals in a phase 3 melanoma trial when combined with Keytruda in the adjuvant setting. The...
Rare bone disease M&A: BioMarin expands mineralization pipeline with Alesta buy
BioMarin agreed to acquire Alesta Therapeutics in a deal valued at $275 million upfront, with up to an additional $215 million tied to development and regulatory milestones. The transaction brings...
Cell therapy: Chimeric pushes in vivo pivot after CAR T toxicities
Chimeric Therapeutics halted development of its cadherin-17-targeted autologous CAR T program CHM-2101 after a second patient at the highest dose level experienced dose-limiting toxicities. The...
Oncology therapeutics: sunvozertinib beats chemotherapy in phase 3 first-line EGFR exon 20 insertion NSCLC
Dizal Pharmaceuticals’ sunvozertinib outperformed chemotherapy in a phase 3 first-line trial for advanced non–small-cell lung cancer with EGFR exon 20 insertion mutations. In NEJM-published...
Healthcare policy and reimbursement: Teva IRA litigation revived by US appeals court
A federal appeals court revived part of Teva’s challenge to the Inflation Reduction Act’s Medicare price negotiation program, according to the report. The DC Circuit ruling reopened part of the...
Precision diagnostics and platform validation: benchmarking NGS assay concordance
Diaceutics and the Precision Cancer Consortium (PCC) launched the NGS Concordance Study to benchmark how consistently variant calls match across next-generation sequencing panels and platforms...
Genetic testing access: Medicare NGS coverage still leaves practical gaps
A new analysis of Medicare claims data found that even after CMS expanded national coverage for certain FDA-approved next-generation sequencing tests in advanced cancers, uptake and access...
AI in bioprocessing: Deepcell and Gilead co-develop a CHO cell morphology foundation model
Deepcell and Gilead Sciences entered a codevelopment partnership to build a custom AI foundation model for Chinese hamster ovary (CHO) cell line development using single-cell morphology. The...
Cell and gene therapy manufacturing: AAV vector supply constraints spur purification focus
Researchers highlighted that viral vector supply and downstream purification remain major bottlenecks for gene therapy, with purification materials struggling to separate full AAV capsids from...
Personalized melanoma mRNA vaccine clears Phase 3 milestone
Merck and Moderna announced that their personalized mRNA neoantigen vaccine, intismeran autogene, met a key Phase 3 endpoint in high-risk melanoma, supporting the combination with Keytruda...
FDA clears Regeneron’s garetosmab (Pasatru) for ultra-rare FOP
The FDA cleared Regeneron’s garetosmab, now marketed as Pasatru, for adults with fibrodysplasia ossificans progressiva (FOP), an ultra-rare genetic disorder that drives soft-tissue to bone...
FDA accelerates Ultragenyx’s first gene therapy for GSDIa
Ultragenyx received FDA accelerated approval for Genglycos (pariglasgene brecaparvovec; DTX-401), an AAV8 gene therapy for glycogen storage disease type Ia (GSDIa) in patients age 8 and older. The...
Regulatory oversight gaps for digital health AI devices
A new review found that most AI-enabled medical devices authorized by the FDA had not been tested for patient outcome improvements. Researchers identified 1,357 AI and machine-learning devices...
Gene therapy and trial risk in China’s fast investigator-initiated model
China’s streamlined clinical trial system for investigator-initiated trials is facing scrutiny after the deaths of three participants in investigator-initiated trials (IITs). The reporting centers...
Diagnostics and reimbursement: access gaps persist for NGS cancer testing
A JAMA Network Open analysis using Medicare claims data found that next-generation sequencing (NGS) uptake for cancer testing rose after Medicare expanded national coverage in 2018, but access...
Rare-disease dealmaking: BioMarin acquires Alesta to expand oral PPi-pathway therapy focus
BioMarin agreed to acquire Alesta Therapeutics in a deal valued at $275 million upfront, plus up to $215 million in development and regulatory milestone payments, to bring ALE1 into its pipeline....
Deep-tech meets drug development: Deepcell and Gilead build CHO morphology foundation model
Deepcell and Gilead Sciences announced a partnership to co-develop a custom AI foundation model for Chinese hamster ovary (CHO) cell line development using single-cell morphology data. The...
CAR T program setback drives pivot toward in vivo platform
Chimeric Therapeutics halted its Phase I/II study of CHM-2101, a cadherin-17-targeted autologous CAR T for gastrointestinal cancers, after a second patient at the highest dose level experienced...
Space and life sciences: NASA flags possible microbial survival on the Moon
NASA researchers reported in Science Advances that several microbial types that could hitchhike on upcoming Artemis missions may survive in the Moon’s permanently shadowed regions near the south...