First patients receive experimental gene therapy injection for rare inherited blindness
NCT ID NCT06461286
Summary
This is the first study in people to test the safety of an experimental drug called PYC-001 for a rare, inherited eye disease that causes progressive vision loss. Up to 18 adults with a confirmed OPA1 gene mutation will receive a single injection of the drug into one eye. The main goal is to see if the treatment is safe and well-tolerated at different dose levels.
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Contacts and locations
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Locations
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Center for Eye Research Australia (CERA)
East Melbourne, Victoria, 3002, Australia
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Save Sight Institute - Sydney Eye Hospital
Sydney, New South Wales, 2000, Australia
Conditions
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