Vancouver, BC – July 31, 2025 — Variational AI is proud to announce its participation in a new open science drug discovery initiative led by Agora Open Science Trust. The project is focused on optimizing the potency and selectivity of lead inhibitors targeting PRMT6, a Type I protein arginine methyltransferase implicated in the pathogenesis of Spinal Bulbar Muscular Atrophy (SBMA), a rare neuromuscular disorder.
As part of the multi-institutional effort, the project aims to evolve the current PRMT6 chemical probe (SGC6870, IC₅₀ = 77 nM) into a preclinical lead compound. Variational AI will support the endeavor by leveraging its Enki™ generative AI platform to propose structurally diverse backup series for the project target product profile (TPP).
“This collaboration is a testament to Variational AI’s commitment to extending our Enki™ platform to tackling unmet medical needs — particularly in rare diseases with no approved treatments.”
– Peter Guzzo, PhD, Executive Vice President, Drug Discovery at Variational AI.
The initiative combines expertise in medicinal chemistry, structural biology, machine learning, pharmacology and preclinical development across a global network of partners, including the Structural Genomics Consortium (SGC), University Health Network (UHN), University of Oxford, Venetian Institute of Molecular Medicine (VIMM), and Charles River. In keeping with the principles of open science, all data — including molecular structures, assay results, progress reports, and meeting recordings will be made publicly available.
“This partnership reflects a shift we are proud to lead, where open science is no longer seen as just an academic exercise. Contributions from industry partners, who provide access to cutting-edge technology like Variational AI, are critical to our mission to discover treatments for rare diseases like SBMA.”
– Peter Sampson, Vice President of Drug Discovery, Agora Open Science Trust.
As two Canadian organizations, Variational AI and Agora are proud to advance a model of science that prioritizes transparency, collaboration, and global impact. The open science framework not only accelerates discovery but also ensures that promising medicines reach underserved populations more affordably.
About Agora Open Science Trust
Agora Open Science Trust is a Canadian charity whose mission is to accelerate the discovery and development of affordable new medicines through open science. Agora’s first initiative, M4K Pharma (‘Medicines for Kids’), is advancing a novel ALK2 inhibitor for the treatment of Diffuse Intrinsic Pontine Glioma. Agora’s pipeline of collaborative open science drug discovery programs has recently expanded to include Spinal Bulbar Muscular Atrophy, and Primary Sclerosing Cholangitis – both of which currently have no approved treatment. The SBMA/PRMT6 program is supported by funding from the Krembil Foundation and Conscience’s Developing Medicines through Open Science program.
More information: https://www.agoraopensciencetrust.org
About Variational AI
Variational AI is redefining the unit economics of drug discovery for better patient outcomes.
Founded in 2019, the company has built a proprietary generative AI foundation model purpose-built for small molecule discovery. Its Enki™ platform leverages this model to generate novel molecular structures de novo, optimized to meet a defined Target Product Profile.
From Hit ID to Lead Optimization, Enki™ accelerates early discovery by generating synthesizable compounds without relying on high-throughput screening or traditional libraries.
Variational AI is growing – check out our open positions to join the team!
More information: https://variational.ai