SMA Research Platform

Evidence graph for Spinal Muscular Atrophy

Biology-first target discovery
Christian Fischer / Bryzant Labs
13,058Targets
453Trials
84Drugs
7Datasets
42,617Sources
48,024Claims
55,019Evidence
29,646Hypotheses

Targets

VALIDATED DATA

Genes, proteins, and pathways in the SMA Research Platform. Filter by role: SMA Targets covers the Simon-priority list (primary) plus pathway-adjacent legacy targets. Off-Target Substrate shows the proteome-scan kinases, GPCRs, and ion channels used for selectivity screening. All Targets returns the complete catalogue. Role is stored on targets.metadata.target_role — all filtering happens server-side.

How does Target Role Classification work?

Every target row carries a target_role set by the backend classifier. Priority order on collisions:

  • primary_sma — canonical Simon/SMA axis genes (SMN1/2, MuSK-AGRN-LRP4-DOK7-RAPSN-AChR, ROCK-LIMK-CFL, PERP, PLS3, NCALD, UBA1, TP53 …)
  • sma_adjacent — Bryzant legacy research targets (ingested pre-2026-04-20, not on the primary list)
  • offtarget_substrate — proteome-scan kinases, GPCRs, ion channels used for Boltz-2 / Chai-1 selectivity screening
  • other — miscellaneous ingest without a clear flag

Classifier source: autonomous-jobs/scripts/classify_targets_role.sql. Filtering: /api/v2/targets?role=….

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