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Fragile X Syndrome Program

Fragile X Syndrome

Discovery/Pre-clinicalActive

Key Facts

Indication

Fragile X Syndrome

Phase

Discovery/Pre-clinical

Status

Active

Company

Purposeful

About Purposeful

Purposeful is an early-stage biotech leveraging an AI platform to identify new therapeutic uses for existing, approved drugs, with a primary focus on rare genetic diseases. The company has established research collaborations with patient foundations and pharmaceutical companies to validate candidates for conditions like Fragile X Syndrome and Glioblastoma. Operating as a private, likely pre-revenue entity, Purposeful aims to significantly reduce the time and cost of drug development by bypassing early-stage safety testing.

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Therapeutic Areas

Rare Diseases Neurology Oncology Dermatology

Other Fragile X Syndrome Drugs

Drug	Company	Phase	Status
KER-0193	Kaerus Bioscience	Preclinical	Active
p70 S6 Kinase Inhibitor	Epigen Biosciences	Preclinical	Active
HLX-0206	Healx	AI Discovery	Active
ENT-3379	Enthorin Therapeutics	Phase 1	Active
AUT00206	Autifony Therapeutics	Clinical	Active
Fragile X Program	Sentinel Oncology	Preclinical	Active
Fragile X Syndrome Trial	Science 37	Phase 3	Completed/Highlighted
DAYBUE® (trofinetide)	Neuren Pharmaceuticals	Phase 2	Completed
Fragile X Syndrome Test	Genomics BioSci & Tech	Clinical	Active
Xanamem (emestedastat)	Actinogen Medical	Phase 2	Active