Larimar is dedicated to improving the ability of patients with rare diseases to pursue their dreams

About Larimar

Clinical-stage biotechnology company focused on developing treatments for complex rare diseases.

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Our Platform

Proprietary intracellular delivery platform intended to deliver proteins and other molecules inside cells.

Our Platform >

Friedreich’s ataxia Clinical Trial

Currently in a Phase 1 trial, CTI-1601, is intended to deliver human frataxin to patients with Friedreich’s ataxia.

Clinical Trial >

Intracellular delivery to treat rare diseases

Larimar Therapeutics is a clinical-stage biotechnology company focused on developing treatments for complex rare diseases. The company’s lead compound, CTI-1601, is currently being evaluated in a Phase 1 clinical program as a potential treatment for Friedreich’s ataxia, a rare and progressive genetic disease. Larimar Therapeutics also plans to use its intracellular delivery platform to design other fusion proteins to target additional rare diseases characterized by deficiencies in intracellular bioactive compounds.

News and Events


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