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KIT-13

Rett Syndrome

Pre-clinicalActive

Key Facts

Indication
Rett Syndrome
Phase
Pre-clinical
Status
Active
Company

About NeuroCores

NeuroCores is a private, pre-clinical biotech company pioneering a novel approach to treating severe neurological conditions by targeting plasmalogen deficiency. The company's lead compound, KIT-13, is an oral small molecule for Rett syndrome that has demonstrated safety and efficacy in mouse models and has secured both FDA Orphan Drug and Rare Pediatric Disease designations. Founded in 2016 as a spin-out from Japanese research, NeuroCores is led by a team with deep neuroscience and biotech commercialization experience. The company operates at the intersection of high unmet medical need and a potentially transformative platform technology, positioning it for significant value creation if clinical translation is successful.

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Other Rett Syndrome Drugs

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DPM-1003DepYmedPhase 1
ExoEdit® for Rett SyndromeEvox TherapeuticsPre-clinical
RVL-001 (vorinostat)Unravel BiosciencesClinical Trial Initiated
Rett Syndrome ProgramHerophilusDiscovery
Glutamate Modulator for Rett SyndromeNumedicusLicensed/Orphan Designation
TTI-0102Thiogenesis TherapeuticsPhase 1
DAYBUE® (trofinetide)Neuren PharmaceuticalsApproved
TSHA-102Taysha Gene TherapiesPhase 1/2
SarizotanNewron PharmaceuticalsPhase III