Systemic delivery of SingleCut CRISPR gene editing components restored dystrophin expression to up to 92 percent of normal expression in heart muscle in canines. First study of Exonics’ SingleCut technology in large mammals supports advancement toward the clinic. Study published online in the journal Science.
August 30, 2018 || Exonics Announces Publication of Research Demonstrating Gene Repair Technology Restored Dystrophin Expression in Canine Model of Duchenne Muscular Dystrophy
January 31, 2018 || Exonics Therapeutics Announces Publication of Research Demonstrating Gene Repair Technology Corrected Cardiac Abnormalities Associated with Duchenne Muscular Dystrophy
SingleCut CRISPR efficiently restored dystrophin expression in human engineered heart muscle and demonstrated broad applicability across a range of Duchenne mutations. Study published online in the journal Science Advances.
January 30, 2018 || Exonics Therapeutics Appoints Roger M. Perlmutter, M.D., Ph.D., to Board of Directors
Dr. Perlmutter brings extensive biopharmaceutical research and development experience to Exonics’ board. Since 2013, Dr. Perlmutter has served as executive vice president of Merck and president of Merck Research Laboratories.
November 29, 2017 || Exonics Therapeutics Announces Publication of Research Demonstrating Gene Repair Technology Corrected Dystrophin Expression in Mouse Model of Duchenne Muscular Dystrophy
Exonics Therapeutics today announced the publication of a preclinical study demonstrating the Company’s SingleCut CRISPR technology efficiently corrected in vivo dystrophin expression in a mouse model of Duchenne.
November 8, 2017 || Exonics Therapeutics Raises $40 Million in Series A Financing
Funds to support continued development of SingleCut CRISPR technology for Duchenne muscular dystrophy
June 11, 2017 || NBC’s Sunday Night with Megyn Kelly
Watch Exonics’ scientific founder Dr. Eric Olson discuss the promise of gene editing for Duchenne muscular dystrophy
May 6, 2017 || The Great Hope / A Doctor’s Dilemma Documentary
Watch Dr. Olson and patient Ben Dupree discuss the potential of Exonics’ gene-editing therapy for Duchenne in this two-part German documentary on gene editing
February 28, 2017 || A CRISPR/Cas9 Startup Launches on the R&D Frontier, Working on a One-time Fix for Duchenne MD
February 27, 2017 || Exonics to Use CRISPR in an Effort to Treat Majority of DMD Boys
February 27, 2017 || Startup Aims to Treat Muscular Dystrophy with CRISPR
MIT Technology Review