SCN1A Upregulation for Dravet Syndrome

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Dravet syndrome

Phase 1/2Clinical entryAntisense oligonucleotideEpilepsySupportive· high conf3 evidence-linked claims

Overview

Disease area: Epilepsy
Indication: Dravet syndromeMONDO:0100135
Mechanism of action: TANGO splice-modulating ASO upregulating SCN1A expression
Development stage: Phase 1/2
Institution: Capitol Movement Disorders Center
Principal investigators: S. Nakamura, R. Halvorsen
Targets: SCN1A
Pathways: Ion channel regulation
Trial registry: —

Scientific assessment

1 claims

Target ValidationStrong
Dravet results from SCN1A haploinsufficiency in interneurons; TANGO splice-modulating ASOs upregulate the healthy allele.
datasetMONDO:0100135

Translational assessment

1 claims

EndpointsModerate
Convulsive seizure frequency is the established efficacy endpoint; overnight EEG supports pharmacodynamic confirmation.
publicationPMID:32937104

Regulatory assessment

1 claims

Orphan / Rare DiseaseContext
Pediatric orphan epilepsy supports orphan designation and rare-pediatric-disease incentives.
regulatoryorphan-dravet

Commercial assessment

0 claims

No evidence linked yet.

Execution assessment

0 claims

No evidence linked yet.

Structured reviews

1 reviews

Supportive· high confExternalEpilepsy KOL2025-03-28

Strengths

Precise haploinsufficiency rationale; clear seizure endpoint.

Weaknesses

ASO redosing burden in pediatric population.

Improvement areas

Optimize dosing interval and delivery.

Internal rubric input (reviewer scoring — not a success probability)

Scientific validity

Translational readiness

Regulatory pathway

Commercial potential

Execution strength

Outcome

Clinical entry
Phase 1/2 in pediatric Dravet.2025-03-28

AI brief

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