This study is an open-label, single ascending dose clinical trial in participants who have ABCA4-related retinopathies. This is the first-in-human clinical trial in which ACDN-01 will be evaluated for safety, tolerability, and preliminary efficacy following a single subretinal injection of ACDN-01.
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Safety and tolerability of ACDN-01 as measured by the number and severity of adverse events and serious adverse events.
Timeframe: 12 months
Associate Director, Clinical Operations