[{"orgOrder":0,"company":"Sio Gene Therapies","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Axovant Announces Upcoming Presentations at the Annual Meeting of the American Society of Gene and Cell Therapy","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Undisclosed","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"May 2020","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Undisclosed"},{"orgOrder":0,"company":"Sio Gene Therapies","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Axovant Completes Enrollment of Low-Dose Cohort in Phase 1\/2 Study of AXO-AAV-GM1 and Expands Study to Include Type I Patients with GM1 Gangliosidosis","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I\/ Phase II","country":"U.S.A","productType":"Cell and Gene 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Cease the License Agreement for Gene Therapy Programme in Parkinson\u2019s Disease","therapeuticArea":"Neurology","highestDevelopmentStatus":"Phase I\/ Phase II","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"January 2022","url1":"","url2":"","graph1":"Neurology","graph2":"Phase I\/ Phase II"}]
Find Clinical Drug Pipeline Developments & Deals by Axovant Gene Therapies
Sio intends to return the global rights for AXO-Lenti-PD and that they intend to cease work on this gene therapy programme in Parkinson’s Disease. Sio is currently conducting a Phase 2 SUNRISE-PD trial with AXO-Lenti-PD.
Preclinical studies in murine and a naturally-occurring feline model of GM1 gangliosidosis have supported AXO-AAV-GM1’s ability to increase β-galactosidase enzyme activity, reduce GM1 ganglioside accumulation, improve neuromuscular function, and extend survival.
AXO-AAV-GM1, the company’s adeno-associated viral vector (AAV)9-based gene therapy for the treatment of Type I (early infantile onset) and Type II (late infantile and juvenile onset) GM1 gangliosidosis.
Initial data from the ongoing Phase 1/2 study in five patients in the low-dose cohort showed that AXO-AAV-GM1 was generally well tolerated with a favorable safety profile and provide early indications of clinical disease stability.
AXO-AAV-GM2 is the first investigational gene therapy to achieve IND clearance for Tay-Sachs and Sandhoff diseases. The Company received a letter from the FDA indicating that it has satisfactorily addressed all issues related to the clinical hold.
AXO-Lenti-PD gene therapy demonstrate consistent treatment outcomes including Favorable safety and tolerability profile, with no serious adverse events attributable to gene therapy; Improvement in Hauser diary “Good ON time” and “OFF time” and other outcomes.
AXO-AAV-GM1 is the first gene therapy to enter clinical trials for GM1 gangliosidosis and the company believes that it has the potential to provide meaningful clinical benefit to both Type I and Type II patients.
AXO-Lenti-PD was well-tolerated with no treatment-related serious adverse events at 6 months. EXPLORE-PD, a randomized, sham-controlled study of AXO-Lenti-PDis expected to begin dosing in 2021.
Axovant will have access to manufacturing resources for Axovant’s AAV-based gene therapy programs, AXO-AAV-GM1 for GM1 gangliosidosis and AXO-AAV-GM2 for GM2 gangliosidosis with sufficient capacity to support ongoing development and eventual commercialization.
Oxford Biomedica will manufacture GMP batches for Axovant to support the ongoing and future clinical development of AXO-Lenti-PD, a clinical-stage gene therapy product to treat moderate to severe Parkinson’s Disease based on Oxford Biomedica’s LentiVector® platform.
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