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Rett Syndrome Program

Rett Syndrome

DiscoveryActive

Key Facts

Indication

Rett Syndrome

Phase

Discovery

Status

Active

Company

Herophilus

About Herophilus

Herophilus is a private, pre-clinical stage biotech leveraging a proprietary platform of human brain organoids, high-throughput biology, and AI/ML to de-risk and accelerate CNS drug discovery. The company has established a pipeline targeting high-need neurological and psychiatric conditions, including Rett syndrome, Alzheimer's disease, and schizophrenia, starting from a patient-derived biobank. By generating deep phenotypic data from physiologically relevant human models, Herophilus aims to identify novel drug candidates with a greater probability of clinical success.

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Therapeutic Areas

Neurology Psychiatry

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
RVL-001 (vorinostat)	Unravel Biosciences	Clinical Trial Initiated	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