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RVL-001 (vorinostat)

Rett Syndrome

Clinical Trial InitiatedActive

Key Facts

Indication

Rett Syndrome

Phase

Clinical Trial Initiated

Status

Active

Company

Unravel Biosciences

About Unravel Biosciences

Unravel Biosciences is a private, preclinical-stage biotech founded in 2019 and based in Cambridge, Massachusetts. The company has developed a proprietary AI-driven platform that analyzes patient RNA signatures to create digital twin models, enabling the discovery of new therapeutic mechanisms and the repurposing of existing drugs. This approach aims to accelerate clinical validation and derisk development, as evidenced by its lead program for Rett syndrome which has received FDA Orphan Drug Designation and entered clinical testing. Unravel operates a hybrid business model, both discovering its own therapeutics and partnering its platform for drug discovery collaborations.

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Therapeutic Areas

Neurology Rare Diseases Complex Disorders

Other Rett Syndrome Drugs

Drug	Company	Phase	Status
NTI164	Neurotech International	Phase 2	Active
RTT1	Grann Pharmaceuticals	Pre-clinical	Active
NEX-1 / NEX-2	Nexien BioPharma	Pre-clinical / Phase 1	Active
KIT-13	NeuroCores	Pre-clinical	Active
DPM-1003	DepYmed	Phase 1	Active
ExoEdit® for Rett Syndrome	Evox Therapeutics	Pre-clinical	Active
Rett Syndrome Program	Herophilus	Discovery	Active
Glutamate Modulator for Rett Syndrome	Numedicus	Licensed/Orphan Designation	Active
TTI-0102	Thiogenesis Therapeutics	Phase 1	Planned
DAYBUE® (trofinetide)	Neuren Pharmaceuticals	Approved	Commercial
TSHA-102	Taysha Gene Therapies	Phase 1/2	Active (Pivotal-Enabling)
Sarizotan	Newron Pharmaceuticals	Phase III	Discontinued