Floatz Rating
CCC48/100
Confidence
Indicative
Sponsor
University of California, San Diego
Modality
nucleic_acid
Development Phase
Phase 1/2
Status
Active
Evidence ledger · v0.2
Clinical Evidence
Clinical track record: trial progression, reported outcomes, safety signals, and endpoint quality.
33Indicative
Detailed axis rationale is planned and will be published soon.
| Trial | Phase | Status | N | Primary endpoint | Readout |
|---|---|---|---|---|---|
NCT06314490CT.gov Personalized Antisense Oligonucleotide Therapy for Rare Pediatric Genetic Disease: SCN2A | — | Active Not Recruiting | — | — | — |
Competitive Position
Competitive setting: how crowded the indication is, class-level failures, and timing against rivals.
95Indicative
Detailed axis rationale is planned and will be published soon.
Same indication · Hereditary Disease
| Asset | Sponsor | Phase | Rating |
|---|---|---|---|
| Nl-Scn2A-002 (this asset) | University of California, San Diego | P1/2 | CCC · 48 |
| Dexasone | Intergroupe Francophone du Myelome | P3 | BB |
| Aoc 1044 | Avidity Biosciences, Inc. | P3 | B |
| Nexiguran Ziclumeran | Intellia Therapeutics | P3 | B |
| Zeleciment Rostudirsen | Dyne Therapeutics | P3 | B |
| Cyclophosphamide | Children's Hospital Los Angeles | P2 | CCC |
| Pomalidomide | Intergroupe Francophone du Myelome | P2 | CCC |
| Fulvestrant | Borstkanker Onderzoek Groep | P2 | CCC |
| Alpelisib | Borstkanker Onderzoek Groep | P2 | CCC |
+7 more in the Hereditary Disease cohort
Scientific Foundation
Strength of the underlying biology: target validation, tractability, modality fit, and how related mechanisms have fared.
NR
Planned for methodology v0.2.
Development Feasibility
How realistically the program can be executed, drawing on modality precedent, enrollment dynamics, and sponsor delivery.
NR
Planned for methodology v0.2.
Commercial Opportunity
Commercial prize: addressable population, unmet need, and the value case for the indication.
NR
Planned for methodology v0.2.
IP & Exclusivity
Exclusivity position, covering patent protection and freedom-to-operate runway.
NR
Planned for methodology v0.2.
Manufacturing & Supply
Manufacturing and supply readiness, driven by modality process and scale-up risk.
NR
Planned for methodology v0.2.
Related assets
Citation
Floatz Terminal. Nl-Scn2A-002 in Hereditary Disease. Methodology v0.2. Rated under v0.2 effective July 8, 2026. Last refreshed July 8, 2026. Accessed July 14, 2026. https://terminal.floatz.ai/assets/nl-scn2a-002-hereditary-disease
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