SMA Research Platform

Evidence graph for Spinal Muscular Atrophy

Biology-first target discovery
Christian Fischer / Bryzant Labs
Targets
Trials
Drugs
Datasets
Sources
Claims
Evidence
Hypotheses

Top Drug Candidates

COMPUTATIONAL

This is a unified, computationally ranked list of candidate compounds drawn from ChEMBL bioactivity data, cross-disease repurposing libraries, and DiffDock virtual binding against SMA target pockets. Every entry is a computational prediction — a compound that passed in-silico filters and scored well on an integrated ranking. None of these candidates has been tested in a wet lab; ranking high here is a hypothesis to test, not a measured result.

Each candidate passes through a 6-stage computational filtering pipeline: (1) Drug-likeness — Lipinski, QED, PAINS substructure filters, BBB/CNS-MPO estimates; (2) Structural — DiffDock pose prediction against SMA target binding pockets; (3) Analog search — ChEMBL SAR neighbours; (4) ADMET prediction — rule-based absorption/distribution/metabolism/excretion/toxicity estimates; (5) Literature scan — automated PubMed retrieval; (6) Suggested assays — proposed experimental designs for any future wet-lab follow-up. All six stages are in-silico; none constitutes experimental validation.

Note: ADMET predictions use rule-based heuristics (Lipinski Rule of 5, TPSA-based BBB estimate, QED score, PAINS substructure filters). These are computational filtering tools, not validated pharmacokinetic or toxicology models.

How does Candidate Scoring work?

Candidates are scored 0–1 (integrated computational score) and binned into tiers: Tier A (≥ 0.6) — highest computational ranking, the first compounds a wet lab would screen if/when experimental work begins; Tier B (0.4–0.6) — moderate computational signal; Tier C (< 0.4) — weak / computational-only signal.

The integrated score combines target-convergence score, pChEMBL activity, ADMET profile, PAINS/hERG/AMES risk flags, and repurposing evidence where available. These are computational rankings, not measured activities.

Login → Command Center