Cure Rare Disease is focused on developing next-generation therapeutics specifically aimed at treating rare and ultra-rare neuromuscular diseases. Their approach involves a collaborative framework that leverages partnerships with leading academics and clinicians to accelerate the drug development process. This innovative model addresses the significant challenges posed by traditional drug development timelines and costs, which can exceed $2 billion and take over a decade. By reimagining the pathway for creating life-saving therapies, Cure Rare Disease aims to provide tailored solutions for patients who have been historically overlooked by conventional pharmaceutical efforts.

The company employs a structured six-stage process to develop its therapeutics, beginning with the characterization of genetic mutations in patients and culminating in clinical trials. This process includes prototyping and optimizing therapeutic candidates, testing their efficacy through tissue modeling and mutation-specific mouse models, and ensuring safety through rigorous manufacturing and testing protocols. Cure Rare Disease is currently working on multiple indications, including Duchenne and Becker muscular dystrophies, various forms of limb-girdle muscular dystrophy, and other rare genetic conditions. Their commitment to advancing research and development in this field is underscored by their extensive network of collaborations and the potential to impact over 200,000 lives.

United States

Cure Rare Disease™ is developing advanced therapeutics for those who have been diagnosed with rare, genetic diseases that have no treatment or cures. Through partnerships and collaboration, our world-renowned researchers build life-saving therapeutics that are intended to stop or reverse the progression of disease.

Cure Rare Disease

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