SMA Research Platform

Evidence graph for Spinal Muscular Atrophy

Biology-first target discovery
Christian Fischer / Bryzant Labs
1,145Targets
453Trials
60Drugs
7Datasets
34,514Sources
43,071Claims
46,973Evidence
29,625Hypotheses
announcementApr 5, 2026· SMA Research Platform

Multi-Fidelity Scoring Pipeline Live — Compound Ranking Active

#scoring#pipeline#READY#LIMK2#ROCK2#CORO1C

A multi-fidelity Bayesian compound scoring pipeline is live, ranking 27,299 compounds across 34 SMA targets.

4 tiers: Docking (w=0.15) → ADMET (w=0.25) → MD Stability (w=0.35) → Binding (w=0.25). Missing layers widen confidence intervals.

Lead compounds under validation (v2.1 pipeline):

  1. Fasudil → ROCK2 (score 0.383) — Only fully validated compound. 19 real DiffDock poses, preclinical evidence (Bowerman 2012).
  2. genmol_119_bbb_5 → LIMK2 (confidence +0.58) — Real lead, MD 100ns running. Supersedes bbb_0 and original genmol_119.
  3. Riluzole → SMN2 — Only validated DiffDock hit from 56-compound screen.

Correction (2026-04-06): Previous version claimed 4 READY compounds. Audit revealed 74 DiffDock tasks had environment bugs producing fake results. genmol_119 original is NOT LIMK2-selective. genmol_119_bbb_0 docking was invalid. All compounds now tracked through 10-stage v2.1 pipeline with quality gates. No compound has completed all stages yet.

Explore at Compound Pipeline.

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