Developing transformative gene repair therapies for devastating genetic neuromuscular diseases

Exonics Therapeutics is developing transformative SingleCut CRISPR gene editing therapies to treat genetic neuromuscular diseases with significant unmet need. Our initial focus is on repairing mutations that cause Duchenne muscular dystrophy, a serious disease with no cure.

SingleCut CRISPR gene editing is a revolutionary technology that can be used to repair genetic mutations.

gene editing

Exonics’ lead program with SingleCut CRISPR gene editing is focused on the most frequent mutations that cause Duchenne, a serious disease that is currently underserved by available treatments. Our technology has the potential to be applied to a wide range of other neuromuscular diseases.

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The Duchenne community has a legacy of playing an important role in search of effective treatments to address this devastating disease.


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