Industry Partnership to Replace Trial and Error in RNA Drug Discovery

Designing new medicines has traditionally relied on an expensive cycle of trial and error, testing thousands of molecules before finding the one with the perfect balance of effectiveness and safeness. A new collaboration between Alnylam Pharmaceuticals and AI firm Inceptive Nucleics aims to change that by using generative AI to predict which drug candidates are most likely to succeed before they ever enter the lab.

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The partnership combines Alnylam’s 20 years of RNA interference expertise, which has already produced 6 approved medicines, with Inceptive’s “foundation models of life”, AI systems trained to recognise complex biological patterns within genetic sequences. By analysing mRNA targets and exploring millions of possible small interfering RNA designs, these models can identify molecules predicted to have greater potency, improved efficacy and stronger therapeutic potential. Researchers can then focus their lab testing on only the most promising candidates instead of broad trial and error screening.

Early collaboration has been promising, with Inceptive’s models generating meaningful biological insights from rather small datasets within only a matter of weeks, demonstrating an ability to characterise small interfering RNA molecules and adapt across different research projects without needing to retrain.

This collaboration is worth up to $2 billion, reflecting the magnitude of this shift in pharmaceutical research. AI is moving beyond supportive admin workflows and into creative scientific discovery processes, helping researchers ask better questions, generate novel hypotheses and accelerate early-stage development.

Foundation models trained on biological data could eventually help design entirely new classes of medicine, reducing development times and improving efficiency, but also giving pharmacovigilance professionals a new responsibility to validate these discoveries. AI is becoming a powerful partner in helping to decide which experiments are worth performing, but we humans retain responsibility to do the final test and check these experiments can be approved and marketed.

“Together, we’re not just accelerating drug discovery; we’re changing the way we understand and improve life.” Jakob Uszkoreit, Co-Founder and CEO of Inceptive

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