Evidence Release 2026.08DMD context observedDMD candidate-conditioned prediction locked

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Evidence freeze: 3 August 2026 P59 hold · scorer failure retained · no target score NAR working package · 6 main + 31 supplementary figures Resource: v1.2.0-measured-dmd-evidence Schema: 1.1 Open platform state → Open Trust Center → Open evidence dashboard → Open claim registry → Open release status → Detail level

Experiment OS · local V201 candidate

Convert the next experiment into gates a machine can refuse.

This plan is derived from the canonical Research Answer and the existing ZNF133 Study Card. It exposes readiness without mistaking a design scaffold for a registered, powered or authorized experiment.

Inspect execution gates Open registry JSON

STOP — DRAFT BLOCKED OPEN NUMERIC AND AUTHORITY GATES. The plan has 6 open and 2 partial gates. No execution authority, immutable registration, powered sample size or measured DMD outcome exists.

Bound object chain

Research Answer → Study Card → Experiment Plan → Outcome slot.

EXPERIMENT-PLAN:DMD:ZNF133:CRISPRI:48H:FUSION_AND_MOLECULAR_RESPONSE:V1

Question: Q2:DMD:PROLIFERATING_MYOBLAST:CRISPRI:ZNF133:48H:FUSION_AND_MOLECULAR_RESPONSE:SINGLE
Source answer: ANSWER:Q2:DMD:PROLIFERATING_MYOBLAST:CRISPRI:ZNF133:48H:FUSION_AND_MOLECULAR_RESPONSE:V1
Source Study Card: SC:v1.2.0-measured-dmd-evidence:ZNF133:1.5-DRAFT
Lifecycle track: TRUTH GENERATION · prediction required: no

Execution gate ledger

6 ready · 2 partial · 6 open.

“Ready” means usable as a draft input. Registration still requires every partial and open item to be frozen under experimental authority.

Open JSON Schema →

READYGATE:QUESTION_SCOPE

Question scope

READY REGISTERED ANSWER

Preserve the exact disease, cell state, perturbation, target, time and endpoint identity.

Source: ANSWER:Q2:DMD:PROLIFERATING_MYOBLAST:CRISPRI:ZNF133:48H:FUSION_AND_MOLECULAR_RESPONSE:V1

READYGATE:BIOLOGICAL_SCOPE

Disease, cell, target, time and endpoint

READY AS DRAFT

Freeze the query-selected scope at registration; do not generalize beyond it.

Source: Q2:DMD:PROLIFERATING_MYOBLAST:CRISPRI:ZNF133:48H:FUSION_AND_MOLECULAR_RESPONSE:SINGLE

PARTIALGATE:PRIMARY_ESTIMAND

Primary estimand

PARTIAL REQUIRES ENDPOINT SCALE AND NUMERIC EFFECT

Choose the primary endpoint scale and freeze the minimally important numeric effect with units.

Source: SC:v1.2.0-measured-dmd-evidence:ZNF133:1.5-DRAFT

READYGATE:INFERENTIAL_UNIT

Inferential unit

READY AS DRAFT

Independent biological replicate or independently generated perturbation unit; cells within one aggregate are not inferential replicates.

Source: SC:v1.2.0-measured-dmd-evidence:ZNF133:1.5-DRAFT

READYGATE:CONTROLS

Comparator and controls

READY AS DRAFT

Non-targeting control. Positive assay control. Mock delivery control when delivery itself can affect phenotype. Target-engagement and viability controls.

Source: SC:v1.2.0-measured-dmd-evidence:ZNF133:1.5-DRAFT

OPENGATE:REPLICATION

Replication and sample size

OPEN POWER CALCULATION

Use the 3–6 replicate range only for a variance pilot; freeze final n after assay variance and minimally important effect are available.

Source: SC:v1.2.0-measured-dmd-evidence:ZNF133:1.5-DRAFT

PARTIALGATE:RANDOMIZATION

Randomization and blocking

PARTIAL SEQUENCE NOT GENERATED

Randomize Independent culture well or biological replicate, not individual cells.; retain blocks for Donor/isogenic pair. Differentiation batch. Plate and reagent identity.; generate and freeze the allocation sequence.

Source: SC:v1.2.0-measured-dmd-evidence:ZNF133:1.5-DRAFT

OPENGATE:BLINDING

Blinding

OPEN NOT SPECIFIED

Declare who is blinded during acquisition, QC, endpoint extraction and primary analysis.

Source: SC:v1.2.0-measured-dmd-evidence:ZNF133:1.5-DRAFT

OPENGATE:NUMERIC_QC

Numeric assay QC

OPEN NUMERIC THRESHOLDS

Target-engagement threshold must be numeric and frozen. Viability floor and image-quality thresholds must be numeric and frozen.

Source: SC:v1.2.0-measured-dmd-evidence:ZNF133:1.5-DRAFT

OPENGATE:EFFECT_MARGINS

Effect and equivalence margins

OPEN NUMERIC VALUES AND UNITS

Required before registration; derive from assay biology or a justified pilot and store the numeric value with units. Required before interpreting a null result; store a symmetric or asymmetric numeric margin with units.

Source: SC:v1.2.0-measured-dmd-evidence:ZNF133:1.5-DRAFT

READYGATE:ANALYSIS

Analysis, multiplicity and missingness

READY AS DRAFT

Mixed-effects model with treatment fixed effect and donor/batch/reagent blocking; report reagent-specific estimates. Primary endpoint across the two reagents; secondary endpoints form a separate multiplicity family. Define exclusions before unblinding; report all missing units and reasons; do not single-impute primary outcomes without a prespecified sensitivity analysis.

Source: SC:v1.2.0-measured-dmd-evidence:ZNF133:1.5-DRAFT

READYGATE:STOP_AND_RELEASE

Stopping and complete release

READY AS DRAFT

Stop or classify as infeasible if a required numeric QC threshold fails. Do not interpret a nonsignificant result as no material effect without a negligible-effect interval. Do not change canonical candidate status automatically; require governed review. Release the frozen card, protocol identifiers, analysis code, complete denominators and results irrespective of direction; never overwrite the registered card.

Source: SC:v1.2.0-measured-dmd-evidence:ZNF133:1.5-DRAFT

OPENGATE:REGISTRATION

Immutable registration

OPEN NOT REGISTERED

Resolve authority decisions, freeze the plan and deposit an immutable registered Study before execution.

Source: /resource/api/v1.1/registered_studies.json

OPENGATE:OUTCOME_SLOT

Outcome return slot

OPEN EMPTY LEDGER

Append every qualifying outcome, including null, toxic, discordant, QC-failed and inconclusive results; never overwrite registration.

Source: /resource/api/v2/outcome_ledger.json

Required decisions

Seven items must be resolved before immutable registration.

These are authority or numeric choices. The website cannot infer them from candidate scores, the qualitative information-gain label or a pilot planning range.

Freeze the perturbation direction and its causal, compensatory or accompanying rationale.
Freeze the primary endpoint scale, minimally important effect and units.
Freeze the negligible-effect margin and units before interpreting a null result.
Estimate assay variance and complete the final sample-size calculation.
Freeze numeric target-engagement, viability and image-quality thresholds.
Generate the allocation sequence and declare blinding responsibilities.
Name the experimental authority and create an immutable registration receipt.

Export the design object

The JSON and YAML share one checksummed plan identity.

Exporting does not authorize execution, calculate power or register a Study.

Download JSON Download YAML Open Outcome Ledger