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mcp4

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mcp4

Re: None

Wednesday, 08/15/2018 2:32:59 PM

Wednesday, August 15, 2018 2:32:59 PM

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FDA has granted orphan drug designation to MeiraGTx's AAV-CNGA3 gene therapy candidate for the treatment of achromatopsia caused by mutations in the CNGA3 gene.

Achromatopsia is a genetic retinal disease that impairs a person’s sight by preventing cone photoreceptors in the eye from functioning. AAV-CNGA3 is intended to restore cone function and is delivered to the cone receptors at the back of the eye via subretinal injection.
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