Rare Daily Staff

Cure Rare Disease, a nonprofit biotechnology research organization, said it has formed a research partnership with Seattle-based Curi Bio to further develop its CRISPR-based gene therapy for Duchenne muscular dystrophy.

Rich Horgan, whose brother Terry has Duchenne muscular dystrophy, founded the organization to develop precision therapies for rare diseases. The organization’s lead experimental therapy is a CRISPR therapy to treat DMD patients with an exon 1 deletion.

Duchenne muscular dystrophy (DMD) is a rare, fatal neuromuscular genetic disease that occurs in approximately one in every 3,500-5,000 males worldwide. DMD is caused by a change or mutation in the gene that encodes instructions for dystrophin. Symptoms of DMD usually appear in infants and toddlers. Affected children may experience developmental delays such as difficulty in walking, climbing stairs or standing from a sitting position. As the disease progresses, muscle weakness in the lower limbs spreads to the arms, neck and other areas. Most patients require full-time use of a wheelchair in their early teens, and then progressively lose the ability to independently perform activities of daily living such as using the restroom, bathing and feeding. Eventually, increasing difficulty in breathing due to respiratory muscle dysfunction requires ventilation support, and cardiac dysfunction can lead to heart failure. The condition is universally fatal, and patients usually succumb to the disease in their twenties.

The 32-week project evaluates gene therapy candidates on human-relevant cardiac and skeletal muscle models. These cardiac and skeletal muscle models are derived from human induced pluripotent stem cells (hiPSC) that allow for patient-specific modeling of diseases, including Duchenne muscular dystrophy. 

The collaboration will utilize Curi Bio’s Mantarray platform, an innovative biosystem that leverages hiPSC-derived 3D engineered tissues. The Mantarray platform improves the physiological relevance of cell cultures in the preclinical stage of therapeutic discovery. These hiPSC-derived tissue models will then be used with Cure Rare Disease’s CRISPR-based gene therapy candidates to measure their safety and efficacy on restoring cell and tissue function in patients.

“There are many obstacles for individuals and families seeking a cure for rare diseases like Duchenne muscular dystrophy and the existing testing modalities for potential therapies are a significant limitation,” said Horgan.  “Through our partnership with Curi Bio, we will be able to study how our gene therapy will work on a given patient’s cells in a manner that was impossible just a few years ago.  This collaboration is a game changer and gets us one step closer to having an effective therapy that will successfully treat Duchenne muscular dystrophy.”

Photo: Rich Horgan, founder and president of Cure Rare Disease

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