CLN3 Gene Therapy Showing Safety, Possible Efficacy in Trial, Amicus Reports

CLN3 Gene Therapy Showing Safety, Possible Efficacy in Trial, Amicus Reports

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CLN3 Gene Therapy Showing Safety, Possible Efficacy in Trial, Amicus Reports

AT-GTX-502, Amicus Therapeutics’ investigational gene therapy for juvenile Batten disease, has been well-tolerated and is showing a potential to stabilize early signs of the disease in children and infants. These are the initial findings of a first Phase 1/2 trial (NCT03770572) investigating the safety and efficacy of a single dose of AT-GTX-502 in infants and children, ages 3–10, with juvenile Batten disease, also known as CLN3 disease. Based on these promising findings, Amicus is planning to…

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