adeno-associated virus (AAV)-based gene therapy / Amicus, BioMarin - LARVOL DELTA

Gene Therapy Mediated Cross Correction for CDKL5 Deficiency Disorder (ASGCT 2022) - "While current therapeutic options for CDD are limited, adeno-associated virus (AAV)-based gene replacement therapy represents a promising option for restoring CDKL5 and mitigating pathology...In summary, these data indicate that hu68-BIP-TATk28-CDKL5 cross correct cells in relevant brain regions and mitigates EEG abnormalities including the increase in gamma frequency power associated with seizure susceptibility. Importantly, these preclinical findings suggest the remarkable possibility that the window for genetic therapeutic intervention in CDD is wider than previously recognized and may extend into adulthood."

CNS Disorders • Cognitive Disorders • Developmental Disorders • Epilepsy • Gene Therapies • Genetic Disorders • Immunology • Mental Retardation