Cardiovascular biotech Tenaya Therapeutics has signed a research collaboration agreement with RNAi specialist Alnylam Pharmaceuticals, in a deal that includes a $10 million upfront payment and much more in potential milestone payments for Tenaya if the partnership bears fruit.
The collaboration will focus on novel human genetic targets for cardiovascular disease, with Tenaya validating up to 15 potential candidates and Alnylam assuming responsibility for development and commercialization of any viable therapies. According to the March 5 release, Tenaya could earn up to $1.13 billion if all of the identified targets lead to approved drugs for cardiovascular disease.
A recent SEC filing describes the deal in more detail. Tenaya and Alnylam will jointly identify and nominate targets to advance and will work on them for at least two years while conducting in vitro and in vivo validation activities. As part of the accord, Alnylam will hold an exclusive worldwide license to any commercial candidates.
The pressure is on Tenaya to nominate high-quality targets. The filing states that if any nominated targets do not meet the agreed-upon standards, the upfront fee may be reduced by $500,000 per failed candidate, up to a maximum eight candidates.
Tenaya leverages “human induced pluripotent stem cell-derived cardiomyocytes (iPSC-CMs) with high-throughput in vitro screening, imaging analysis, and machine learning algorithms” to identify new genetic targets, according to the press release.
The company also uses heart tissue and in vivo models to validate potential candidates.
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The process has generated more than 150 genetic targets and has yielded the company’s current clinical-stage programs, including multiple gene therapies and a small-molecule candidate in phase 1.
It has been an up-and-down couple of years for Tenaya. In 2024, the company’s TN-401 gene therapy for arrhythmogenic right ventricular cardiomyopathy generated positive preclinical results. A couple of months later, the company laid off a fifth of its staff to focus on TN-401 and TN-201, another gene therapy targeting MYBPC3-associated hypertrophic cardiomyopathy.
Last year, the FDA placed a clinical hold on a TN-201 phase 1b/2a trial and instructed the biotech to standardize activities related to the immunosuppression regimen across trial sites.
Meanwhile, Alnylam has been pursuing other avenues to identify new targets and today announced a multiyear agreement with genomics company Helix to access Helix’s clinical data and medical and pharmacy claims datasets to identify new opportunities for RNAi therapeutics.
