Safety and efficacy of ATSN-101 in patients with Leber congenital amaurosis caused by biallelic mutations in GUCY2D: a phase 1/2, multicentre, open-label, unilateral dose escalation study.

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Title: Safety and efficacy of ATSN-101 in patients with Leber congenital amaurosis caused by biallelic mutations in GUCY2D: a phase 1/2, multicentre, open-label, unilateral dose escalation study.
Authors: Yang P; Casey Eye Institute, Oregon Health & Science University, Portland, OR, USA. Electronic address: yangp@ohsu.edu., Pardon LP; Atsena Therapeutics, Durham, NC, USA., Ho AC; Wills Eye Hospital, Philadelphia, PA, USA., Lauer AK; Casey Eye Institute, Oregon Health & Science University, Portland, OR, USA., Yoon D; Atsena Therapeutics, Durham, NC, USA., Boye SE; Division of Cellular and Molecular Therapy, Department of Pediatrics, University of Florida, Gainesville, FL, USA., Boye SL; Powell Gene Therapy Center, Department of Pediatrics, University of Florida, Gainesville, FL, USA., Roman AJ; Center for Hereditary Retinal Degenerations, Scheie Eye Institute, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA, USA., Wu V; Center for Hereditary Retinal Degenerations, Scheie Eye Institute, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA, USA., Garafalo AV; Center for Hereditary Retinal Degenerations, Scheie Eye Institute, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA, USA., Sumaroka A; Center for Hereditary Retinal Degenerations, Scheie Eye Institute, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA, USA., Swider M; Center for Hereditary Retinal Degenerations, Scheie Eye Institute, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA, USA., Viarbitskaya I; Center for Hereditary Retinal Degenerations, Scheie Eye Institute, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA, USA., Aleman TS; Center for Hereditary Retinal Degenerations, Scheie Eye Institute, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA, USA., Pennesi ME; Casey Eye Institute, Oregon Health & Science University, Portland, OR, USA; Retina Foundation of the Southwest, Dallas, TX, USA., Kay CN; VitreoRetinal Associates, Gainesville, FL, USA., Fujita KP; Atsena Therapeutics, Durham, NC, USA., Cideciyan AV; Center for Hereditary Retinal Degenerations, Scheie Eye Institute, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA, USA. Electronic address: cideciya@pennmedicine.upenn.edu.
Source: Lancet (London, England) [Lancet] 2024 Sep 07; Vol. 404 (10456), pp. 962-970.
Publication Type: Journal Article; Clinical Trial, Phase II; Multicenter Study; Clinical Trial, Phase I; Research Support, Non-U.S. Gov't
Journal Info: Publisher: Elsevier Country of Publication: England NLM ID: 2985213R Publication Model: Print Cited Medium: Internet ISSN: 1474-547X (Electronic) Linking ISSN: 01406736 NLM ISO Abbreviation: Lancet Subsets: MEDLINE
Database: MEDLINE Ultimate
Description
ISSN:1474-547X
DOI:10.1016/S0140-6736(24)01447-8