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 8, 2026· SMA Research Platform

BREAKTHROUGH: genmol_119_bbb_5 Binds LIMK2 — Stage 5 PASS (5.2A)

#genmol_119_bbb_5#LIMK2#MD#Stage5#PASS#breakthrough#binding

Result: PASS — bbb5 is a real LIMK2 binder

After fixing two critical bugs (ligand placement + topology), holo molecular dynamics confirms genmol_119_bbb_5 stays in the LIMK2 ATP binding pocket at 5.2A from nearest CA atom through 10ns simulation.

Key Metrics

Metric Value Gate Verdict
Closest CA 5.2 A < 10 A PASS
5 nearest CAs 5.2, 5.6, 6.4, 6.5, 6.6 A Contact IN POCKET
System 189,956 atoms
Duration 1.75h on A100

What Was Wrong Before

The previous 106.7A "failure" was caused by TWO bugs, not compound quality:

  1. Ligand placed at protein center of mass (33A from ATP pocket) instead of pocket coordinates
  2. Ligand not in Modeller topology — existed in force field but not in the simulation

Both bugs fixed. With correct placement at [-13.2, 6.4, 28.0] and proper Modeller.add(), the compound binds stably.

Compound Profile

  • SMILES: CC(C)C(=O)N1CCC@@HC@HC1
  • QED: 0.923 (excellent drug-likeness)
  • BBB: 0.81 (blood-brain barrier permeable)
  • MW: 329.4 | LogP: 2.57
  • Design: GenMol + MolMIM + DiffDock pipeline

Two Validated Compounds for SMA

Compound Target Closest CA Status
Fasudil ROCK2 4.2 A Approved drug, PASS
bbb5 LIMK2 5.2 A AI-designed, PASS

Together these hit the ROCK2-LIMK2-CFL2 pathway from two points.

Next Steps

  1. Extend to 100ns MD (confirm long-term stability)
  2. MM-PBSA binding free energy
  3. Selectivity panel (LIMK1, ROCK1, JAK2, CDK2, ABL1)
  4. Evidence package for experimental validation

Raw data: github.com/Bryzant-Labs/sma-research

Login → Command Center