Publications & Presentations
Friedreich Ataxia
Identification of Safe and Effective Intravenous Dose of AAVrh.10hFXN to Treat the Cardiac Manifestations of Friedreich's Ataxia
PublicationStress-Induced Mouse Model of the Cardiac Manifestations of Friedreich's Ataxia Corrected by AAV-mediated Gene Therapy
PublicationCorrection of half the cardiomyocytes fully rescue Friedreich ataxia mitochondrial cardiomyopathy through cell-autonomous mechanisms
PublicationA 22-Year Follow-up Study of Long-term Cardiac Outcome and Predictors of Survival in Friedreich Ataxia
Publication
Arrhythmogenic Cardiomyopathy
Safety and Preliminary Efficacy of AAVrh.10PKP2 (LX2020) Gene Therapy in PKP2-Arrhythmogenic Cardiomyopathy
Oral PresentationPlakophilin 2 gene therapy prevents and rescues arrhythmogenic right ventricular cardiomyopathy in a mouse model harboring patient genetics
PublicationPlakophilin-2 Gene Therapy Prevents Arrhythmogenic Right Ventricular Cardiomyopathy Development In A Novel Mouse Model Harboring Patient Genetics
PublicationDiagnostic and therapeutic strategies for arrhythmogenic right ventricular dysplasia/cardiomyopathy patient
PublicationTruncating plakophilin-2 mutations in arrhythmogenic cardiomyopathy are associated with protein haploinsufficiency in both myocardium and epidermis
PublicationMolecular Insights into Arrhythmogenic Right Ventricular Cardiomyopathy Caused by Plakophilin-2 Missense Mutations
Publication
Vector Serotypes and AAVrh10
Favorable Complement Profile of AAVrh10: Clinical Monitoring Experience from Three Gene Therapy Studies Across Two Programs
Poster PresentationVector Distribution and Transgene Expression in the Heart Following Gene Transfer of AAVrh.10 vs. AAV9 Capsids
Poster PresentationQuantitative Whole-Body Imaging of I-124-Labeled Adeno-Associated Viral Vector Biodistribution in Nonhuman Primates (December 2020)
Publication
Chemistry Manufacturing and Controls
Demonstrated Comparability Between Adherent HEK293 and Sf9 Baculovirus Suspension AAVrh.10 Clinical Manufacturing Processes
Oral PresentationScale-up and Manufacturing Comparability of an Sf RVN Baculovirus Production Process from 10L to 200L for AAV
Poster PresentationImproving VP1 Ratios Impact on CQAs in rh10 AAV Manufactured through Sf9 Platform
Poster PresentationDevelopment of a Novel High-Yielding Scalable Sf9-Baculovirus Platform to Produce Quality AAV at 200L Scale
Poster Presentation
Preclinical Programs
APOE4 Associated Alzheimer’s
AAVrh.10 delivery of novel APOE2-Christchurch variant suppresses amyloid and Tau pathology in Alzheimer’s disease mice
PublicationAPOE in the bullseye of neurodegenerative diseases: impact of the APOE genotype in Alzheimer's disease pathology and brain diseases
PublicationResistance to autosomal dominant Alzheimer’s disease in an APOE3 Christchurch homozygote: a case report
PublicationAAVrh.10-Mediated APOE2 Central Nervous System Gene Therapy for APOE4-Associated Alzheimer's Disease
PublicationIntracerebral adeno-associated virus gene delivery of apolipoprotein E2 markedly reduces brain amyloid pathology in Alzheimer's disease mouse models
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