Uncategorized
AGTC receives orphan drug designation for gene therapy to treat achromatopsia
The FDA has granted orphan drug designation to AGTC’s gene therapy product candidate for the treatment of achromatopsia caused by mutations in the CNGA3 gene, AGTC announced in a press release. “Receiving U.S. orphan drug designation is another significant milestone as we continue to advance our pipeline of novel gene therapies to treat rare inherited eye disorders,” Sue Washer, president and CEO of AGTC, said in the release.