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Taysha Gene Therapies Announces Publication of Positive Proof-of-Concept Preclinical Data for an AAV-mediated UBE3A Gene Replacement Approach Demonstrating Therapeutic Potential for The Treatment of Angelman Syndrome in the Journal JCI Insight
AAV-mediated UBE3A gene replacement recapitulates endogenous isoform ratios by replacing both the short and long isoforms of UBE3A in key regions of the
About this update from Taysha Gene Therapies, Inc.
[{"type":"text","content":"\nAAV-mediated UBE3A gene replacement recapitulates endogenous isoform ratios by replacing both the short and long isoforms of UBE3A in key regions of the brain, leading to improvements in motor learning, behavior outcomes, and seizure phenotypes in Angelman mouse models\n\nNovel construct, originally developed in the laboratories of Dr. Ben Philpot and Dr. Steven Gray, packages both short and long isoforms of UBE3A into a single viral vector, which is expected to confer significant advantages over approaches that express only one of the isoforms\n\nProof-of-concept preclinical data support further study of UBE3A gene replacement therapy as a potentially safe and effective treatment for Angelman syndrome\n\nAdding to an existing vectorized shRNA knockdown approach to unsilence the paternal UBE3A allele, UBE3A gene replacement bolsters Taysha’s pipeline in Angelman syndrome and strengthens its position as a world-leader in developing gene therapies for monogenic CNS disease\n\nAngelman syndrome investor day on Tuesday, October 26, 2021, 10:00 am-1:00 pm ET\n\n DALLAS--(BUSINESS WIRE)--\nTaysha Gene Therapies, Inc. (Nasdaq: TSHA), a patient-centric, pivotal-stage gene therapy company focused on developing and commercializing AAV-based gene therapies for the treatment of monogenic diseases of the central nervous system (CNS) in both rare and large patient populations, today announced the publication of new preclinical data for an AAV-mediated UBE3A gene replacement approach for the treatment of Angelman syndrome in the Journal of Clinical Investigation Insight (JCI Insight).\n\nAngelman syndrome (AS) is a monogenic neurodevelopmental disorder caused by deletions or mutations in the maternal ubiquitin protein ligase E3A (UBE3A) gene. UBE3A encodes both short and long protein isoforms. Whereas the short isoform is most abundant, clinical data indicate that long UBE3A isoforms also contribute to healthy brain development and function. Common signs and symptoms of Angelman syndrome typically appear early in childhood and include intellectual and developmental disabilities, walking and balance disorders, gastrointestinal issues, seizures, and little to no speech. There are currently no approved treatments for Angelman syndrome and current interventions are focused on managing medical and developmental issues. Angelman syndrome impacts a...