LRMR
- Larimar Therapeutics, Inc.
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Overview
Company Summary
Larimar Therapeutics, Inc. is a biopharmaceutical company focused on the development of novel treatments for a group of rare genetic diseases known as Friedreich's ataxia (FA) and other related disorders. FA is a degenerative neuromuscular disorder that primarily affects coordination, mobility, and heart function.
Larimar Therapeutics aims to address the underlying cause of FA by developing small molecule therapeutics that can increase the production of frataxin, a protein that is deficient in FA patients. The company's lead investigational product candidate is known as CTI-1601, which is designed to increase frataxin levels within the mitochondria (the energy-producing structures in cells) of FA patients.
CTI-1601 is being developed as a potential one-time gene therapy, delivered directly to the central nervous system or peripheral tissues, to provide long-term benefits for FA patients. The company has been conducting preclinical studies and has plans to initiate clinical trials to evaluate the safety and efficacy of CTI-1601.
In addition to its lead program, Larimar Therapeutics is also exploring other potential therapeutic candidates and technologies that may have applications in treating FA and related disorders. The company's goal is to address the significant unmet medical needs of patients with FA and potentially extend its research to other neurodegenerative diseases.
Overall, Larimar Therapeutics is dedicated to developing innovative therapies that have the potential to improve the lives of individuals suffering from FA, ultimately aiming to significantly impact the course of the disease and provide hope for patients and their families.