Muscular Dystrophy Product Pipeline

About Duchenne Muscular Dystrophy (DMD)

DMD is a progressive and fatal muscular wasting disease. It is one of the most prevalent rare genetic disease, with an estimated prevalence between 10,000 to 15,000 cases in the United States.

We are currently in the lead optimization phase for our wholly-owned research candidates for the treatment of certain muscular dystrophies, including DMD. Our product candidates are engineered from proprietary vectors designed for efficient, low dose intravenous delivery to cardiac and skeletal muscle tissues with minimal toxicities, and potential resistance to pre-existing antibodies in the human population.

Several companies are developing gene therapies for DMD using conventional AAVs. While early clinical data in some programs have been encouraging, AAV gene therapies using conventional AAV vectors have been subject to inflammation-related toxicity issues and manufacturing challenges due to the relatively high doses required. In addition to requiring high doses, patients have been excluded from clinical trials due to pre-existing antibodies to the conventional vector used.

We expect to complete lead optimization in the first half of 2020.