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Homology Medicines Presents Data Demonstrating In Vivo Transduction of Non-Human Primate and Human Retinal Cells at ARVO Annual Meeting
- Data Showed AAVHSCs Edited Human Retinal Cell Types Across Two Targets - - Demonstrated 11 AAVHSC Capsids Crossed Blood-Retinal and Blood-Brain Barriers in
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[{"type":"text","content":"- Data Showed AAVHSCs Edited Human Retinal Cell Types Across Two Targets - - Demonstrated 11 AAVHSC Capsids Crossed Blood-Retinal and Blood-Brain Barriers in In Vivo Studies of Non-Human Primates With Single I.V. Dose - BEDFORD, Mass., May 03, 2021 (GLOBE NEWSWIRE) -- Homology Medicines, Inc. (Nasdaq: FIXX), a clinical-stage genetic medicines company, announced today new preclinical data that demonstrated in vivo nuclease-free gene editing of retinal cells at the virtual Association for Research in Vision and Ophthalmology (ARVO) Annual Meeting. “For the first time, we have shown proof of principle in two gene targets that our AAVHSCs were able to transduce and edit human retinal cell types,” stated Albert Seymour, Ph.D., Chief Scientific Officer of Homology Medicines. “We have now demonstrated through in vivo studies in NHPs that 11 of our capsids crossed the blood-retinal and blood-brain barriers, and the regional tropism of our capsids enables us to select those that are best suited to address retinal diseases. We believe our AAVHSCs have the potential to deliver one-time treatments for retinal diseases, and these data support further development of our ophthalmology program.” In a poster titled, “AAVHSCs, a Nuclease-independent Approach for Transduction in Non-human Primate Brain and Retina & Editing of Retinal Cells in Human Organotypic Explants,” Homology and Novartis presented findings from their work, which evaluated Homology’s human hematopoietic stem cell-derived adeno-associated virus vectors (AAVHSCs) in in vivo studies with non-human primates (NHPs) and ex vivo studies with human retinal cells following a single intravenous (I.V.) or subretinal dose, respectively. Highlights from the presentation include: All 11 capsids evaluated in NHPs: Crossed the blood-retinal and blood-brain barriersTransduced key cells in therapeutically relevant relay points along the retinogeniculate and retinotectal pathwaysShowed a diverse pattern in cellular tropism between the visual relay points, expanding capsid selection capabilities for a given ophthalmic disease AAVHSC15 achieved cross-species transduction of human and NHP photoreceptor cellsSeamless editing in two independent loci and detection of hybrid transcript in human retinal cells was confirmed using molecular methods The e-poster presentation will be available to view each ...