Cardiac biopsy : healthy tissue, no inflammation, and widespread transgene expression in ~50% of cardiomyocytes transgene expression was highly selective for cardiomyocytes: ~4.4 genomes/cardiomyocyte (qPCR) and ~16.2 RNA transcripts/cardiomyocyte (RT-qPCR)ĤD-310 Cardiac clinical endpoints results summary (data cut-off date of December 5, 2022): Pts ( U.S.Cardiac quality of life (physical limitations, symptoms) assessed with KCCQ : clinically meaningful improvement in 2 of 2 patients, and the third remained stable at 100% (no possibility for improvement from baseline).Exercise capacity (peak VO 2 ) assessed by CPET : meaningful improvement in 2 of 3 patients (worsened on ERT natural history study).Cardiac contractility (GLS) assessed on ECHO : meaningful improvement in 3 of 3 patients (GLS worsened on ERT natural history study).Cardiac biopsy available from one patient in the INGLAXA-2 trial.Quality of life status – Change from baseline at 12 months in Kansas City Cardiomyopathy Questionnaire (KCCQ).Exercise capacity – Change from baseline at 12 months in cardiopulmonary exercise testing (peak VO2 by CPET). Left ventricular contractility – Change from baseline at 12 months in global longitudinal strain on echocardiography (GLS on ECHO).Assessed FDA-recommended pivotal trial cardiac endpoints in three patients who completed 12 months of follow-up (cut-off date of December 5, 2022) including:.Enrolled six patients, each treated with 1E13 vg/kg of 4D-310 and a prophylactic corticosteroid immunosuppressive regimen.Treated patients in three geographies: INGLAXA-1 ( U.S.) and INGLAXA-2 ( Taiwan and Australia).
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