Pipeline & Progress

Leading with Duchenne muscular dystrophy, our pipeline is rich with personalized treatments targeted to pinpoint and remedy a myriad of genetic mutations in a growing cohort of patients.

  • Successful Pre-IND meeting with the FDA for first patient in Fall 2020. 

  • Positive comments received regarding study plan

  • PreIND guidance provides template for all future customized therapeutics

  • NAbs prevent the successful delivery of AAV drugs including CRISPR and micro-dystrophin. Currently estimated at 30-50% of the population and a major hurdle.

  • NAb research study established to measure patient NAb levels. Over 30 samples analyzed thus far with a target of 200. 

  •  Formed NAb technical steering committee to help guide development and implementation of technologies to suppress NAbs in 1-2 years.

  • Human-grade therapeutic in production - expected completion: July 2021

  • Therapeutic constructs under development for several additional patients

  • Targeted dosing of patient 1 is second half of 2021

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