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CTI-1601 (Nomaxofusp)

Friedreich's Ataxia

Phase 2Active (Dose Exploration)NCT05579691

Key Facts

Indication
Friedreich's Ataxia
Phase
Phase 2
Status
Active (Dose Exploration)
Company

About Larimar Therapeutics

Larimar Therapeutics is dedicated to addressing rare diseases with significant unmet medical needs through its proprietary intracellular protein replacement platform. Its lead candidate, CTI-1601, is a recombinant fusion protein designed to deliver human frataxin to the mitochondria of patients with Friedreich's ataxia, a debilitating and life-shortening neurodegenerative disorder. The company is advancing CTI-1601 through clinical development while exploring its platform's potential for other rare diseases. Larimar's strategy centers on demonstrating proof-of-concept in Friedreich's ataxia to validate its technology and create value for patients and shareholders.

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Other Friedreich's Ataxia Drugs

DrugCompanyPhase
Vatiquinone (PTC743)PTC TherapeuticsPhase 3
CTI-1601Larimar TherapeuticsPhase 1