Vertex Pharmaceuticals to Acquire Exonics Therapeutics for $1 Billion to Advance CRISPR-Based Gene Editing Therapies for Duchenne Muscular Dystrophy and Myotonic Dystrophy Type 1

VCBeat (WeChat ID: vcbeat) has learned that Vertex Pharmaceuticals recently entered into a definitive acquisition agreement with Exonics Therapeutics (“Exonics”). The transaction is expected to close in the third quarter of 2019.

Under the agreement, Vertex will acquire Exonics for $1 billion. The controlling shareholders of Exonics will receive an upfront payment of $245 million and will be eligible for potential milestone payments upon achievement of specified milestones.

The purpose of this acquisition is to strengthen Vertex Pharmaceuticals’ gene-editing capabilities to develop gene-editing therapies for Duchenne muscular dystrophy (DMD) and myotonic dystrophy type 1 (DM1).

Exonics Therapeutics, founded in 2017, is a biotechnology company based in Boston, Massachusetts, dedicated to developing gene-editing therapies for patients with Duchenne muscular dystrophy (DMD) and other severe hereditary neuromuscular disorders. Dr. Eric Olson is the scientific founder of Exonics Therapeutics and the inventor of SingleCut CRISPR technology.

Eric Olson has extensive experience in the field of gene editing. He is the Chair of the Department of Molecular Biology at the University of Texas Southwestern Medical Center (UTSW) and is recognized as one of the global experts in muscle cell research and gene-editing therapies. Additionally, he has founded several publicly traded biotechnology companies dedicated to developing treatments for muscle diseases.

Exonics is developing advanced SingleCut CRISPR gene-editing therapies to correct genetic mutations. The company has successfully used SingleCut CRISPR to repair the dystrophin gene in multiple small and large animal preclinical models of Duchenne muscular dystrophy (DMD), achieving promising results.

Vertex, founded in 1989 and headquartered in Boston, Massachusetts, USA, is a pharmaceutical company dedicated to developing medicines for patients with serious diseases. The company’s key focus areas include cystic fibrosis, cancer, infectious diseases, inflammatory bowel disease, and neurological disorders.

To further expand its gene-editing capabilities, Vertex also announced a collaboration agreement with CRISPR Therapeutics to discover and develop gene-editing therapies for DMD and DM1. CRISPR Therapeutics is developing drugs using its proprietary CRISPR/Cas9 gene-editing platform.

Relevant experiments have demonstrated that using adeno-associated virus (AAV) to deliver CRISPR/Cas9-based payloads can identify and correct exon mutations, thereby restoring dystrophin production and achieving therapeutic effects for DMD and DM1.

Jeffrey Leiden, Ph.D., Chairman, President and Chief Executive Officer of Vertex, stated, “By acquiring Exonics Therapeutics and expanding our collaboration with CRISPR Therapeutics, we have combined the intellectual property, technologies, and specialized expertise of three companies to establish a leading gene-editing platform for patients with Duchenne muscular dystrophy (DMD) and myotonic dystrophy type 1 (DM1).”

Eric Olson, Founder and Chief Scientific Advisor of Exonics Therapeutics, stated, “Both DMD and DM1 are severe muscle diseases for which there are currently no standard treatments. Vertex has a proven capability to develop therapies for serious diseases. Through our collaboration with Vertex, we believe we can jointly develop safe and effective therapies for patients with severe neuromuscular diseases.”

(Compiled by Jiao Yanli)