Programs and outcomes

We have designed key molecules that advanced into clinical development and validated critical pharmacology, ADME, pharmacokinetic, and pharmacodynamic assumptions that shaped program decisions. Across 30+ programs, 6+ candidates reached clinical or advanced preclinical stages and 35+ patents were co-invented.

Advanced candidate

Splicing Modulators — from MoA to advanced candidate

The program began with an unclear mechanism of action. Our team established the structural basis for splicing modulation, then built models that predicted selectivity over related splice sites — the key liability that had stalled earlier efforts.

Over multiple synthesis rounds, we ran multi-parameter optimization simultaneously addressing potency, selectivity, metabolic stability, and oral bioavailability. Each round produced fewer than 30 compounds, guided by free energy calculations and ML-predicted ADMET profiles.

The result was ATM5292: an advanced candidate with a clean selectivity profile, confirmed mechanism, and favorable PK in multiple species.

Advanced candidate

RNA Binding Protein Inhibitor

A novel target with no prior clinical precedent. We ran iterative computational-experimental cycles addressing the classic optimization triangle: potency against the target, selectivity over related RNA-binding proteins, and ADMET properties suitable for oral dosing.

Structure-guided design using homology models (no crystal structure available) combined with ligand-based SAR to navigate chemical space efficiently. The program reached candidate quality in fewer rounds than the client's internal estimate.

More programs

Oncology First-in-Class Covalent PARG Inhibitors Designed the first covalent inhibitor for PARG. Synthetic lethality enabled by computational prediction of covalent binding modes. Patent filed + AACR 2024
GPCR / Metabolic GPCR Agonist for Metabolic Syndrome Complete pipeline from hit design through optimization. Computational screening identified agonist scaffolds. Optimized lead delivered
Radiopharmaceutical Radio Ligand Lead Optimization Optimization under challenging property constraints for imaging. Balanced binding affinity with radiolabeling feasibility. Optimized lead delivered
Kinase Kinase Selectivity by Design Structural basis of selectivity across a kinase family. Rational selectivity-driven design confirmed experimentally. Selective compounds confirmed
Peptide Macrocyclic Peptides Explainable AI models elucidated mechanism of action. Structure-guided hit-to-lead for this challenging modality. Hit-to-lead advanced
Genomics Allele-Specific Target Validation Target selection at scale for gain-of-function mutations. Computational assessment of mutational impact on dimerization. Novel targets validated
Oncology Ferroptosis Target Inhibitors First-in-class hit finding for ferroptosis targets. Complete hit-to-lead in novel chemical space. Hit-to-lead completed
RNA RNA G-Quadruplex Modulators G-quadruplex structure modeling and MoA postulation for RNA-targeted therapeutic intervention. Mechanism postulated

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