Cure Rare Disease Collaborates With SickKids on Three-Year CRISPR Research Project


$500,000 initiative with Toronto children’s hospital aims to develop genome editing therapies for Duchenne muscular dystrophy patients

BOSTON (Feb. 22, 2022) – Cure Rare Disease, a nonprofit biotechnology research organization on a mission to develop precision medicine for rare diseases, today announced it will fund research at The Hospital for Sick Children (SickKids) in Toronto, that will seek to develop CRISPR-based genome editing therapies for Duchenne muscular dystrophy (DMD) patients. 

Over the course of three years, and supported by over $500,000 (USD) in funding from Cure Rare Disease, SickKids will build upon existing research and aims to develop a CRISPR/Cas9-based treatment to correct gene duplications that cause DMD by restoring full-length dystrophin, a protein critical to muscle strength and function.  DMD is a rare, fatal muscle-wasting disease caused by the body’s inability to produce dystrophin. 

“We are excited to have SickKids, Canada’s premier pediatric hospital, join our collaborative research efforts to develop CRISPR-based therapies for Duchenne muscular dystrophy,” said Rich Horgan, founder and president of Cure Rare Disease.  “This collaboration is a major milestone in our work to hopefully expand our drug development pipeline and continue building our pioneering model to create and dose customized therapeutics for rare diseases like DMD.”

The project will test the feasibility of repairing a variety of DMD-causing duplications utilizing cells from four Cure Rare Disease patients.  

Cure Rare Disease is currently funding an array of projects to develop treatments to correct DMD gene deletions, and will dose its first patient with a CRISPR-based treatment in 2022.

“Duplications in the genetic code are thought to be the direct cause for almost 10% of rare inherited disorders, yet they are incredibly difficult to model and study, which has slowed the development of precision medicine therapies for these patients,” said Zhenya Ivakine, PhD, Scientist in the Genetics & Genome Biology program at SickKids and a principal investigator in the project.  “This funding and collaboration represent an important step forward in our work to individualize therapies for patients with rare diseases, such as DMD.”

“We’re grateful for this opportunity and to these four patients and their families for enabling us to build upon our previous research and continue seeking new treatment avenues for patients where none existed before,” said Ronald Cohn, MD, Senior Scientist, Pediatrician, Geneticist, President and CEO of SickKids and a principal investigator in the project.


About Cure Rare Disease

Cure Rare Disease, a 501(c)(3) nonprofit based in Boston, is transforming possibilities for people with rare diseases by developing customized therapeutics in time to save lives.  Through a collaborative community of forward-thinking families, patients, scientists and supporters, Cure Rare Disease has enabled an ecosystem of innovation and discovery to overcome the obstacles inherent in existing models of medicine and to expedite individualized genetic corrections from clinical research to cure.  Learn more at www.cureraredisease.org.

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