...A. SRP-9001-103: Having worked with FDA, anticipates dosing a trial with commercial process material this year. 1. 2. SRP-9001-103 is Co.'s gene therapy and goal [of which] is to treat Duchenne muscular dystrophy (DMD) by safely delivering skeletal, diaphragm and cardiac muscle, a gene that robustly codes for truncated but functional form of structural protein dystrophin that Co. calls micro-dystrophin. After many years of design and pre-clinical testing by doctors Jerry Mendell and Louise Rodino-Klapac, Co. commenced and completed small four-patient proof-of-concept study, Study 101, that was positive and encouraging. a. b. Based on proof-of-concept study, commenced randomized, blinded, placebo-controlled trial, called Study 102. c. Study 102 intended to show that in well-controlled trial, that SRP-9001 is safe and effective in children with DMD. d. Last one-year visit will occur in Dec. e. Plans to release results from Study 102 in 1Q21. f. Now material for Study 101 and 102 was manufactured...
