Larimar Therapeutics is a clinical-stage biotechnology company that focuses on developing innovative treatments for complex rare diseases. Central to their approach is a proprietary intracellular delivery platform designed to transport proteins and other therapeutic molecules directly into cells. This platform is exemplified by their lead product candidate, nomlabofusp, which is currently undergoing a Phase 2 clinical trial as a potential treatment for Friedreich’s ataxia, a rare genetic disorder characterized by low levels of frataxin, a crucial protein for mitochondrial function. Nomlabofusp is engineered to mimic human frataxin and is equipped with a Cell Penetrating Peptide (CPP) that facilitates its entry into cells and mitochondria, thereby addressing the deficiency of this vital protein in affected patients.

The technology behind Larimar's platform aims to provide disease-modifying therapies by delivering missing or deficient proteins within the cellular machinery. The process involves nomlabofusp crossing both the cell membrane and the mitochondrial membrane, where it is processed to release mature human frataxin. This innovative approach not only targets Friedreich’s ataxia but also holds the potential for developing additional fusion proteins aimed at treating other rare diseases associated with intracellular protein deficiencies. By leveraging their proprietary protein replacement therapy platform, Larimar Therapeutics is positioned to make significant advancements in the treatment of rare diseases that currently lack effective therapies.

United States

Larimar Therapeutics, Inc. (Nasdaq:LRMR), 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 in the U.S. as a potential treatment for Friedreich’s ataxia (FA). Larimar 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. 

Central Nervous System (CNS)

Genetic Diseases

Rare diseases

Larimar Therapeutics

Friedreich’s Ataxia

Rare genetic disease

<p class="font_7">CTI-1601 is a recombinant fusion protein intended to deliver human frataxin into the mitochondria of patients with Friedreich’s ataxia (FA or FRDA) who are unable to produce enough of this essential protein.</p>

Larimar Therapeutics

Company Details