Larimar Therapeutics, Inc.

Larimar Therapeutics, Inc. is a clinical-stage biotechnology company dedicated to discovering and developing treatments for complex rare diseases. Headquartered in Bala Cynwyd, Pennsylvania, the company focuses on addressing significant unmet medical needs for patients suffering from debilitating conditions.

The company's primary product candidate is nomlabofusp (formerly CTI-1601), an investigational recombinant fusion protein designed to deliver frataxin (FXN) to the mitochondria of patients with Friedreich's ataxia (FA). Friedreich's ataxia is a rare, progressive genetic disease characterized by insufficient frataxin production. Larimar Therapeutics utilizes a proprietary protein replacement therapy platform, based on novel cell-penetrating peptide technology, to deliver missing proteins inside cells.

Under the leadership of President and CEO Dr. Carole Ben-Maimon, Larimar Therapeutics recently received Breakthrough Therapy Designation from the FDA for nomlabofusp in Friedreich's ataxia. The company plans to submit a Biologics License Application (BLA) seeking accelerated approval in June 2026, with topline open-label study data expected in Q2 2026 to support this submission. A U.S. launch is targeted for the first half of 2027, if approved. Larimar's strategy is highly concentrated on the development of nomlabofusp, with its economics driven by research and development progress, regulatory execution, and access to capital.