We offer target selection/evaluation, drug design and early stage development to research organizations interested in exploring RNA targets either as strategic partnerships or services. The specialization of our platform is to generate highly specific and bioavailable small molecules targeting RNA/protein complexes, as well as systematically identifying safe and effective candidate RNA targets for a given disease indication. Costing can be tailored to means, from commercially available compounds to ultrahigh specificity novel entities requiring dedicated complex synthesis.
We have successfully adapted our platform to multimodal protein targets, described here.
Multiplex imaging system
Appropriate for zebrafish, other small animals and bioprinted constructs. High throughput (up to one object/min) liquid handling from 96-well plates with automated cellular precision positioning and imaging. Compatible with calcium imaging and automated laser ablation studies.
Custom disease model design available.
Established models include
- Fibrosis
- Angiogenesis
- Ablative brain/spine injury and stroke models
- Tumorigenic (NF1) with individual tumor tracking
- Neuroplasticity (including DAT, PET1 and GLUT subtypes)
- Neuroactivity (including subtypes). Suitable for agonist/re-uptake inhibitor differentiation
Established post-hoc pipelines include MS-based PK/PD/Tox and NGS.
Target selection
All RNA is targetable — though not always directly, selectively or safely. We employ an AI-guided systems biology target selection process, with target interactome analysis to score a candidate RNA drug target for intrinsic safety, targetability, conservation, distribution and efficacy. This methodology successfully identifies the safety outcomes of existent RNA target clinical trials. Low cost project feasibility services for target identification are available for any given disease indication.
AI-Guided Library Generation
Distilling a testable subset of RNA-compatible and synthesizable chemical space using discriminatory machine learning and graph theory. Typically a 1M compound library is selected for virtual screening within 48H. Acceptable target engagement is achieved for >⅔ of these compounds. Completely customizable to user needs such as tissue specificity or commercial availability.
RNA/protein virtual screening
Best-in class for RNA/protein interfaces, guided by the latest molecular dynamic parameters alongside novel gridscoring and solvation methods.