The abstract (program #2066), titled "Long-term Cone ERG Functional Rescue in CNGB3 Mutant Achromatopsia Dogs by AAV-hCNGB3 Vectors Containing the PR1.7 Promoter and Packaged in AAV5, AAV9 or Mutant AAV2 Capsids," describes a study to evaluate the efficacy of an experimental AAV vector expressing the human cyclic nucleotide gated channel beta 3 (hCNGB3) gene to restore function to cone cells (specialized cells primarily responsible for color vision) in the retinas of dogs affected by ACHM. The study assessed the efficacy of AAV vectors expressing hCNGB3 or codon-optimized hCNGB3 (hCNGB3co) cDNAs, driven by 1.7 kb or 2.1 kb versions of the human red cone opsin promoter (PR1.7 or PR2.1) and packed in AAV5, AAV9 or mutant AAV2 mutant capsids. Results demonstrated that functional rescue of cone cells was observed in nearly 100 percent of eyes treated with AAV5-PR2.1-hCNGB3co (n=3/3), AAV5-PR2.1-hCNGB3 (n=5/5), AAV9-PR1.7-hCNGB3co (n=9/10), AAV2tYF-PR1.7-hCNGB3co (n=10/11), and AAV5-PR1.7-hCNGB3co vector (n=3/3).
"These study results are promising and demonstrate that a novel, AAV-based gene therapy has significant clinical potential in treating achromatopsia," said András Komáromy, DrMedVet, Ph.D., Associate Professor,
ACHM is an inherited retinal disease characterized by complete loss of cone photoreceptor function, and people with ACHM have severe vision loss that renders patients legally blind. ACHM can be caused by mutations in several genes, with mutations in CNGB3 accounting for 50 percent of all cases of ACHM in Western countries. While there is currently no cure for ACHM, AGTC is developing a gene therapy product based on an AAV vector to enable expression of normal CNGB3 protein within cone photoreceptors and restore cone photoreceptor functions in patients. AGTC and its academic collaborators have previously demonstrated that subretinal delivery of gene sequences using an AAV vector restores cone responses in a dog model of ACHM.
"We are grateful to our academic research partners for their study support and guidance and look forward to continuing to advance our pipeline of AAV gene therapy products," said
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AGTC is a clinical-stage biotechnology company that uses its proprietary gene therapy platform to develop products designed to transform the lives of patients with severe diseases in ophthalmology. AGTC's lead product candidates focus on X-linked retinoschisis, achromatopsia and X-linked retinitis pigmentosa, which are inherited orphan diseases of the eye, caused by mutations in single genes that significantly affect visual function and currently lack effective medical treatments. AGTC is also pursuing pre-clinical development of treatments for wet AMD using the company's experience in ophthalmology to expand into disease indications with larger markets.
Casey Williamson Michigan State University College of Veterinary Medicine T: (517) 353-9849 firstname.lastname@example.org Katherine Unger BaillieUniversity of PennsylvaniaT: (215) 898-9194 email@example.com David Carey Lazar Partners Ltd.for AGTC T: (212) 867-1768 firstname.lastname@example.org Larry BullockChief Financial Officer Applied Genetic Technologies CorporationT: (386) 462-2204 email@example.com
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