Tenaya Therapeutics and Alnylam partner on RNAi cardiovascular target discovery worth up to USD 1.13b.
Tenaya Therapeutics, Inc. (Nasdaq: TNYA), a clinical-stage biotechnology company headquartered in South San Francisco, California, and Alnylam Pharmaceuticals (Nasdaq: ALNY), based in Cambridge, Massachusetts, have 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. Financial terms were not disclosed in full, but the deal is not a traditional drug licensing arrangement; it is a preclinical research collaboration with no named drug candidates or specific molecular targets disclosed at 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. Royalty rates and territorial rights were not publicly disclosed, though Alnylam's assumption of full development and commercialization responsibilities suggests it holds broad, likely worldwide, rights to resulting programs. No equity component was disclosed.
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 Pharmaceuticals 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.
For Tenaya, the deal monetizes its target discovery platform in a therapeutic area adjacent to but distinct from its own gene therapy pipeline. The upfront payment and research reimbursement provide near-term capital, while the milestone structure offers long-term upside tied to Alnylam's development execution. This is the first disclosed business arrangement between the two companies.
For Alnylam, the collaboration expands its cardiovascular disease therapeutics pipeline by sourcing novel genetic targets from an external discovery engine. Alnylam has previously applied its RNAi platform to cardiovascular targets including PCSK9, the basis for inclisiran, which was originally licensed to The Medicines Company and subsequently acquired by Novartis in January 2020 as part of a USD 9.7 billion acquisition. Alnylam also entered a broad RNAi collaboration with Regeneron Pharmaceuticals in March 2021, reported at approximately USD 1 billion upfront with total potential value exceeding USD 2 billion, covering multiple therapeutic areas including cardiometabolic indications.
The RNAi cardiovascular space has seen sustained deal activity. Arrowhead Pharmaceuticals has advanced internal siRNA programs targeting APOC3 (plozasiran) and ANGPTL3, and entered a collaboration with Amgen in 2016 covering RNAi programs for cardiovascular and metabolic targets, from which the Lp(a)-targeting program olpasiran emerged. Other RNAi-based cardiovascular assets in clinical development include:
- Inclisiran (Novartis) — PCSK9 siRNA, approved and marketed
- Olpasiran (Amgen/Arrowhead) — Lp(a)-targeting siRNA, Phase III
- Plozasiran (Arrowhead) — APOC3 siRNA, Phase III
- Zerlasiran (Silence Therapeutics) — PCSK9 siRNA, Phase II
The Tenaya Alnylam collaboration is structured to feed Alnylam's pipeline with targets that may extend beyond these established pathways, though the absence of disclosed targets makes direct competitive comparisons premature at this stage.
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Summary
Tenaya Therapeutics, Inc. (Nasdaq: TNYA), a clinical-stage biotechnology company headquartered in South San Francisco, California, and Alnylam...