AGTC Announces Stargardt Disease as its Second Preclinical Ophthalmology Program (Eyewire News)

AGTC Announces Stargardt Disease as its Second Preclinical Ophthalmology Program

UF startup and UF Innovate | Sid Martin Biotech graduate Applied Genetic Technologies Corporation (AGTC), a biotechnology company conducting human clinical trials of adeno-associated virus (AAV)-based gene therapies for the treatment of rare diseases, announced that it has identified Stargardt disease as the second ophthalmology program in its previously announced preclinical pipeline expansion, which also includes a program targeting the dry form of age-related macular degeneration (AMD). The company is also reporting proof-of-concept expression data for its Stargardt disease gene therapy candidate in non-human primates (NHPs).

“The expansion of our preclinical pipeline to include Stargardt disease and dry AMD demonstrates the broad therapeutic and commercial utility of our proprietary AAV gene delivery platform,” said Sue Washer, president and CEO of AGTC. “Our Stargardt disease program underscores our ability to leverage our expertise in AAV vector and genome engineering to expand AAV gene therapy into new indications that require delivery of larger DNA payloads. Dry AMD represents a compellingly large market opportunity given that the disease affects over 24 million people globally. Each of these preclinical programs builds on our industry-leading capabilities in gene therapy for retinal diseases and will leverage our expanding expertise in designing and implementing preclinical and clinical studies that are optimized for success.”

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