discoveryMay 4, 2026
AlphaFold-3 confirms ROCK-LIMK-Cofilin therapeutic axis in SMAHeadline AlphaFold-3 batch 04.05 returned high-confidence iPTM scores (0.80-0.83) for four key protein-pair interactions previously flagged as structurally unce...
Accelerating SMA Drug Discovery Through Computational Science
We combine molecular screening, evidence analysis, and computational biology to identify and validate new therapeutic candidates for SMA — featuring our ROCK-LIMK2-CFL2 axis discovery across 5/6 independent research streams.
Search the Evidence Graph
Data updated 37d ago · Last: pubmed (17 items)
Computational Discovery Pipeline
Stage 0 · Generation
GenMol · MolMIM
Stage 1 · ADMET
ADMET-AI GNN · 41 endpoints
Stage 1.5 · Retrosynthesis
AiZynthFinder
Stage 2 · Safety
PAINS · hERG · CYP
Stage 3a · Rigid Dock
DiffDock v2.2 · FlowDock
Stage 3b · Induced-Fit
NeuralPLexer3
Stage 4 · Selectivity
≥5 off-target panel
Stage 5 · MD 100 ns
OpenMM · GROMACS
Stage 6 · Free Energy
MM-PBSA + FEP
Stage 7 · Orthogonal
PMIDs · scRNA
Stage 8 · Lab Handoff
ALBF · Lab-in-the-Loop
Drug Screening Funnel
25,519ChEMBL CompoundsBioactivity data
▼ 2% filtered
25,042Drug-likeness FilterLipinski + QED44,059AI-Designed MoleculesGenMol + MolMIM▼ 14% filtered
37,779DockedDiffDock v2.2▼ 99% filtered
214Positive BindersConfidence > 0▼ 94% filtered
13Dual-Target HitsHit ≥2 SMA targets0Selective LeadsLow off-target0BBB-Permeable LeadsCNS-penetrantLatest Discoveries
gpu resultApr 30, 2026
AF3 Batch 02804: LIMK2×CFL2 Substrate Interface Validates Core SMA Therapeutic Axis (iPTM=0.80)TL;DR AlphaFold-3 batch `20260429_02804` (33 protein-protein interface jobs, 5 model seeds each) ingested into the platform. First structural validation of the ...
computationApr 27, 2026
v2 validator: 3 hits PASS, methodology calibration documented3-LLM consensus validator now PRODUCTION (v2) The Spark Chai-1 saturator surfaces protein-ligand pair predictions continuously, and a Modal serverless A100 back...
announcementApr 12, 2026
SMA Congress 2026: NMJ axis is the #1 unmet need — launching an in-silico rescue campaignWhat the 2026 SMA Congress told us The 5th International Scientific Congress on SMA (Budapest, 11–14 March 2026) reframed the agrin-LRP4-MuSK-DOK7-RAPSN NMJ axi...
hypothesisApr 12, 2026
Three-mechanism combo memo: Fasudil + ARGX-119-like + CDDO-Me as the next SMA conversationThe thesis Fasudil alone is a single-mechanism cytoskeletal rescue. The 2026 SMA Congress (Budapest) and the Cure SMA 2025 meeting together handed the field two...
data updateMay 2, 2026
New high-dose Spinraza regimen may offer added benefits in SMAA new high-dose regimen of Spinraza (nusinersen) was recently approved in the U.S. as a treatment for spinal muscular atrophy (SMA), with clinical trial data sh...
computationApr 27, 2026
Three-backend GPU fleet live: NIM + Dell + DGX Spark GB10Three-backend research compute fleet now live The SMA research platform now operates three independent GPU compute backends in parallel, each running pharma-rel...
discoveryApr 12, 2026
Swallowing delay persists in early-treated SMA infants — bulbar pathway is independent of motorThe finding A German cohort (n = 30+ SMA infants treated with nusinersen or onasemnogene abeparvovec) shows that swallowing development remains delayed compared...
data updateMay 5, 2026
U.K. consensus sets new guidance for hip care in children with SMAA panel of experts has developed the first U.K. national consensus recommendations for preventing and managing hip problems in children with spinal muscular atr...