US-based biotechs Tenaya Therapeutics, Inc. (Nasdaq: TNYA) and Alnylam Pharmaceuticals (Nasdaq: ALNY) entered into a research collaboration agreement to discover and validate novel genetic targets for the development of RNA interference cardiovascular disease therapeutics.
Under the deal, Tenaya will apply its proprietary target identification and validation platform to validate up to 15 gene targets over a two-year term, after which Alnylam will hold responsibility for all development and commercialization of RNAi-based therapeutics against validated targets. No drug candidates or specific molecular targets were disclosed within the announcement.
Tenaya will receive up to USD 10 million in upfront payments along with reimbursement for research costs incurred during the two-year validation period. Alnylam will be responsible for funding all downstream development and commercialization. Tenaya is eligible for up to USD 1.13 billion in development and commercial milestone payments contingent on all 15 validated genetic targets heart disease programs reaching approval.
Deal context
The collaboration leverages complementary capabilities from each company. Tenaya contributes a suite of integrated tools for modality-agnostic target identification and validation, including human induced pluripotent stem cell-derived cardiomyocytes, high-throughput in vitro screening, imaging analysis, machine learning algorithms, and engineered heart tissue models. The company states these capabilities have generated more than 150 genetic targets to date and contributed to its clinical-stage pipeline, which includes TN-201, a gene therapy for MYBPC3-associated hypertrophic cardiomyopathy; TN-401, a gene therapy for PKP2-associated arrhythmogenic right ventricular cardiomyopathy; and TN-301, a small molecule HDAC6 inhibitor for heart failure.
Alnylam brings its established RNAi therapeutics platform, which uses small interfering RNAs conjugated to GalNAc delivery systems to silence disease-driving genes at the mRNA level in hepatocytes. The collaboration expands the firm’s cardiovascular disease pipeline by sourcing novel genetic targets, and follows Alynlam’s August 2025 collaboration with Scenic Biotech for novel drug discovery, and a 2021 deal with PeptiDream for the discovery and development of peptide-siRNA conjugates. Alnylam has previously applied its RNAi platform to cardiovascular targets including PCSK9, leading to the discovery of inclisiran, licensed to The Medicines Company and subsequently acquired by Novartis in January 2020 as part of a USD 9.7 billion acquisition.