Our Programs

Our initial focus is on Duchenne muscular dystrophy, which addresses a very significant unmet need.

In preclinical mouse models of Duchenne, Exonics’ SingleCut CRISPR gene editing technology has used adeno-associated virus (AAV) to deliver gene-editing enzymes CRISPR/Cas9 directed by highly specific single guide-strand RNA to repair exon mutations and restore the production of dystrophin, a key protein which helps stabilize and protect muscle fibers. The loss of dystrophin causes Duchenne.

 

Exonics is initially editing exon 51, which will address the largest group of exon mutations in the Duchenne population. We will leverage our technology to also address other common mutations and anticipate development of sequences targeting a majority of Duchenne mutations in the future.

Green exons are deleted in Duchenne

We are evaluating other serious neuromuscular diseases that could be addressed with this technology.

Exonics has demonstrated that a single systemic administration of SingleCut CRISPR gene editing components delivered in AAV is sufficient to efficiently restore dystrophin expression throughout all skeletal muscles and in the heart of mice with “humanized” Duchenne mutations.

SingleCut CRISPR

About SingleCut CRISPR Gene Editing

CRISPR/Cas9 is a revolutionary method for permanently correcting errors in the DNA sequence that cause disease by cutting the DNA of chromosomes at selected sites to remove or insert segments.

Gene editing is a novel therapeutic area, and Exonics is generating data that supports the development of a safe and efficacious one-time treatment leveraging SingleCut CRISPR gene editing to potentially provide lifelong benefit to Duchenne patients.

Exonics’ SingleCut CRISPR gene editing technology:

  • Uses cell’s natural mechanisms for gene repair to induce normal expression of dystrophin
  • Single cut makes minimum genomic modification, at precise location
  • Targets specific cell types to repair: skeletal muscle, cardiac, diaphragm
  • Durable effect after one administration
  • Treats underlying cause of neuromuscular genetic diseases

Future Areas of Interest

While Exonics’ initial focus is on Duchenne muscular dystrophy, our SingleCut CRISPR gene editing technology has the potential to address many severe neuromuscular diseases. As we advance our Duchenne program, we are also evaluating other neuromuscular diseases with significant unmet need that could benefit from this technology.