SMA Research Platform

Evidence graph for Spinal Muscular Atrophy

Biology-first target discovery
Christian Fischer / Bryzant Labs
14,766Targets
453Trials
84Drugs
7Datasets
6,987Sources
64,683Claims
72,052Evidence
29,649Hypotheses

Pre-registered Predictions

v2 LOCKED · 2026-05-06
3 on the clock · OSF

Public commitment

Result published within 30 days of settle date, regardless of outcome — confirmed, refuted, or null.

Almost no AI-DD platform pre-registers, and most quietly bury refuted predictions. Making the commitment explicit and timed — confirmed, refuted, or null published within 30 days regardless of outcome — is the cheapest credibility move in biotech. Sourced from Claude Opus’s cross-review feedback during the plan lock.

Q3-2026OPEN→ Top-10 #1
Settles by 2026-08-04 (in 83d)

DOK7-MuSK-Agrin-LRP4 NMJ ternary will reach cross-model CONFIRMED on at least one canonical sub-pair by 2026-08-04

Background

The DOK7-MuSK-Agrin-LRP4 ternary complex is the Top-10 #1 flagship NMJ-rescue mechanism in the platform. Per Phase 2 of the breakthrough plan, AF3 + Boltz-2 + Chai-1 + NeuralPLexer-3 consensus on the complex (not just pairs) is the platform's technical moat. We commit to a falsifiable, calendar-bound prediction settled entirely on internal compute — no wet-lab dependency.

Prediction

By 2026-08-04 (90 days from plan lock), the platform's 4-model cross-consensus engine (Boltz-2 + AF3 + Chai-1 + NeuralPLexer-3) will produce a CONFIRMED status (>= 3 of 4 models, all iPTM >= 0.65, spread < 0.30) for AT LEAST ONE of these canonical NMJ sub-pairs: DOK7 x MuSK_kinase, AGRN_LG3 x LRP4_beta12, or MuSK_frizzled x AGRN.

Method

Each sub-pair is queued through the Spark #2 AF3 lane, Dell Boltz-2 lane, NIM Chai-1 lane, and Modal NeuralPLexer-3 lane (after weights download completes). Results land in pair_consensus table on sma_platform; consensus_status is computed by the existing consensus_builder.py cron.

Confirm criterion

At least one sub-pair has consensus_status = 'CONFIRMED' or 'STRONG' in pair_consensus by 2026-08-04 23:59 UTC.

Refute criterion

NO sub-pair reaches CONFIRMED or STRONG by 2026-08-04 — i.e., every attempt remains at SINGLE_MODEL, NEEDS_REVIEW, DISPUTED, or LOW.

Null / inconclusive

Compute infrastructure failure prevents any model from completing on any sub-pair by 2026-08-04 (e.g., Spark #2 lost beyond hard-drain, Dell tunnel persistently down, NPLexer Modal weights never downloaded). Treated as null/inconclusive — re-prediction filed Q4.

Result will be published within 30 days of 2026-08-04 regardless of outcome.
Q3-2026-MUSCLEOPEN→ Top-10 #3
Settles by 2026-08-04 (in 83d)

A myostatin x ActRIIB x small-molecule antagonist ternary will reach cross-model CONFIRMED status on at least one canonical sub-pair by 2026-08-04

Background

Third of three Q3-2026 quarterly registrations. Tests whether the platform can produce a CONFIRMED ligand pose against the myostatin x ActRIIB receptor complex - the apitegromab-class axis with an open small-molecule opportunity.

Prediction

By 2026-08-04 23:59 UTC, the 4-model cross-consensus engine will produce CONFIRMED status on at least one canonical sub-pair: Myostatin prodomain x small-molecule antagonist, ActRIIB ECD x myostatin x small-molecule blocker, or Follistatin x myostatin x stabilizing small molecule.

Method

Each sub-pair queued through Boltz-2 (ligand), AF3 ligand-aware, Chai-1, NPLexer-3. Aggregated by consensus_builder.py.

Confirm criterion

At least one sub-pair has consensus_status IN (STRONG, CONFIRMED) by 2026-08-04 23:59 UTC. STRONG: 4/4 models, all iPTM>=0.65, spread<0.30. CONFIRMED: 3/4 models, all iPTM>=0.65, spread<0.30.

Refute criterion

No sub-pair reaches STRONG or CONFIRMED.

Null / inconclusive

Compute capacity loss prevents evaluation; re-prediction filed for Q4-2026.

Result will be published within 30 days of 2026-08-04 regardless of outcome.
Q3-2026-SPLICEOPEN→ Top-10 #5
Settles by 2026-08-04 (in 83d)

SMN2 splicing modulator binding will reach cross-model CONFIRMED status on at least one canonical RNA-protein-ligand ternary sub-pair by 2026-08-04

Background

Second of three Q3-2026 quarterly registrations. Tests the platforms RNA-protein-ligand ternary capability moat against the risdiplam-class SMN2 splicing modulator interface.

Prediction

By 2026-08-04 23:59 UTC, the 4-model cross-consensus engine will produce CONFIRMED status on at least one canonical sub-pair: SMN2 pre-mRNA x U2AF2 x risdiplam-class, SMN2 ISS-N1 x U1A snRNP x small molecule, or SMN2 exon-7 x SRSF1 x splice-modifier.

Method

Each sub-pair queued through Boltz-2 (ligand+RNA), AF3 ternary mode, Chai-1, NPLexer-3. Aggregated by consensus_builder.py.

Confirm criterion

At least one sub-pair has consensus_status IN (STRONG, CONFIRMED) by 2026-08-04 23:59 UTC. STRONG: 4/4 models, all iPTM>=0.55, spread<0.30. CONFIRMED: 3/4 models, all iPTM>=0.55, spread<0.30.

Refute criterion

No sub-pair reaches STRONG or CONFIRMED.

Null / inconclusive

Compute capacity loss prevents evaluation; re-prediction filed for Q4-2026.

Result will be published within 30 days of 2026-08-04 regardless of outcome.
Falsification log: Every refuted or null prediction stays on this page with the same prominence as confirmed ones. There is no “quiet retraction”. Sourced from the 3-LLM cross-review during plan lock 2026-05-06.
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