SeekR Compound Optimization Engine: Establishing Data-Driven Heuristics for Next-Generation Extrahepatic RNAi Therapeutics

Principal Entity

Oligon Thérapeutiques inc.


Project of $749,350 over 1 year

  • Supported by CQDM through:
    Ministère de l’Économie, de l’Innovation et de l’Énergie du Québec (MEIE)
  • And by co-funding partner:
    – Oligon Thérapeutiques inc.

The project in detail:

Challenge: 

While GalNAc-siRNA conjugates (small molecule-based ligands that recognize and bind to specific receptor proteins on liver cells), have revolutionized hepatic RNA interference (RNAi), delivering siRNA to extrahepatic tissues remains the industry’s critical bottleneck. Emerging solutions like Antibody-siRNA conjugates show promise but suffer from complex biologic manufacturing, poor solid tumor penetration, and inefficient endosomal escape. There is currently no mature platform that combines the manufacturing simplicity of chemically synthesized RNA with the cell-specific delivery power of antibodies.

Solution:

The Oligon’s SeekR Platform, a proprietary, fully synthetic RNA therapeutic platform addresses this gap. A SeekR molecule is a bivalent chimera consisting of two high affinity aptamers flanking a double-stranded RNA “bridge” that encodes two specific siRNA. The result is a self-delivering RNA drug that can penetrate specific cell types and precisely silence multiple genes without the need for lipid nanoparticles or viral vectors.

Through this project, Oligon will employ systematic Design of Experiments (DoE) to map the multidimensional relationship between chemical architecture and functional performance of SeekR therapeutic compounds. This data will be synthesized into SeekR Optimization Heuristics (a proprietary set of algorithmic design rules). These heuristics will serve as a predictive engine, allowing Oligon to compute the optimal architecture for new SeekR candidates in silico before synthesis, transforming drug discovery from a trial-and-error process into a scalable engineering discipline.

Expected Achievements /Impacts:

By successfully establishing these Optimization Heuristics, Oligon will not only maximize the probability of clinical success for its lead assets, but this innovation in digital drug engineering will also accelerate the efficiency and success of translating new multitargeting concepts into pharmacologically optimized SeekR drugs. Furthermore, the Algorithmic Design Framework developed here will directly enable the design of aptamer siRNA SeekR combinations, generating a reusable and scalable innovation asset that will strengthen Oligon’s commercial competitiveness and further catalyze the growth of Québec’s emerging RNA therapeutics ecosystem.

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