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Alnylam announced AHA and ISA presentations covering vutrisiran in transthyretin amyloidosis, including five-year HELIOS-B follow-up with two years of open-label extension data. Planned analyses also examine exercise capacity, cardiopulmonary measures, and real-world treatment patterns. The announcement previews forthcoming presentations; open-label and post hoc analyses require interpretation distinct from randomized comparisons.
Precision BioSciences updated its ARCUS in vivo editing portfolio in its third-quarter report. The company described ongoing ELIMINATE-B evaluation of PBGENE-HBV and the Phase 1/2 FUNCTION-DMD study of PBGENE-DMD, which uses AAV delivery. Planned data releases and management expectations should be distinguished from completed clinical outcomes.
Nurix said Sanofi will stop the first-in-human Phase 1 trial of oral STAT6 degrader SAR448272 and end the program. Nurix no longer expects future milestone payments or royalties from this asset, while other licensed programs remain eligible for such payments. The November 3 announcement did not disclose the reason for discontinuation.
PAQ Therapeutics plans to present the first clinical findings for selective KRAS G12D degrader PT0253 at ENA 2026 in Barcelona on November 20. The oral presentation will draw on an ongoing Phase 1 study in advanced solid tumors; a separate poster covers preclinical work. The announcement did not disclose quantitative efficacy results.
Arvinas said it will prioritize a Phase 1b study of its LRRK2 degrader ARV-102 in progressive supranuclear palsy in the European Union and United Kingdom. Trial applications and regulatory authorization remain pending. The company continues discussions with US and Japanese regulators, while the US investigational new drug application remains on hold.
Researchers used the properties of more than 100 central nervous system drugs to design brain-directed ionizable lipids. HX1 nanoparticles delivered RNA and enabled gene editing in mouse brains, delivered reporter mRNA in a nonhuman primate, and reduced radiation-related brain injury in mice carrying Il10 mRNA. These are preclinical findings.
Lonza signed an agreement to acquire five US CDMO sites from Merck KGaA, adding bioconjugation, payload-linker, viral vector, and advanced delivery capabilities. Closing is expected in mid-2027, subject to customary conditions. Until then, the businesses will continue operating independently.
An AAPS Open perspective surveys conjugation strategies for antisense oligonucleotides and siRNAs, including small-molecule ligands, cell-penetrating peptides, antibodies, and aptamers. The abstract contrasts clinically established GalNAc delivery with emerging approaches to extrahepatic tissues and discusses translation challenges. This is a synthesis of delivery platforms and clinical progress, rather than a new experimental or clinical efficacy study.
An AAPS Open study used a compaction simulator to track reversible density changes in urea between 600 and 900 MPa, consistent with its known form I-to-III transformation. The original abstract proposes in-die density monitoring as a material-sparing screen for pressure-induced phase changes. Applicability depends on a sufficiently large density difference; urea is the demonstrated model compound.
Samsung Biologics reported that 97.66% of PolyPeptide's issued shares had been tendered by October 30, with a provisional participation rate of 97.99% including concert parties. Its November 2 notice postponed settlement and anticipated completion in late 2026 or the first quarter of 2027, subject to remaining offer conditions and regulatory approvals.
Fresenius signed an exclusive development agreement with WuXi Biologics providing access to up to 37 biosimilar candidates over the next decade. Initial work is expected to focus on polatuzumab vedotin and teprotumumab biosimilars. Fresenius retains selection flexibility and commercialization economics; the candidate count is an option-based opportunity, not 37 approved products.
FDA announced final guidance on potency assurance for cellular and gene therapies on November 2. Its recommendations integrate manufacturing-process design, material and process controls, in-process testing, and lot-release potency assays. The agency emphasizes product-specific, risk-based strategies across development and commercialization; this is final guidance, not approval of any individual therapy.
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