This week looking forward to:
Sio Gene Therapies Announces Four Upcoming Oral Presentations At 24th Annual Meeting Of American Society Of Gene And Cell Therapy
Sio Gene Therapies Inc. (NASDAQ:SIOX), a clinical-stage company focused on developing gene therapies to radically transform the lives of patients with neurodegenerative diseases, today announced four upcoming oral presentations at the 24th Annual Meeting of the American Society of Gene & Cell Therapy (ASGCT), to be held virtually between May 11th to May 14th, 2021.
The AXO-AAV-GM1 presentation will include a review of patient-level data on safety and efficacy at 6 months follow up from the low-dose cohort of the Company's ongoing clinical study. Additionally, Dr. Cynthia Tifft, the lead investigator for the study, will present 6-month biomarker data from cerebrospinal fluid (CSF) in the 5 children who received intravenous AAV9 gene therapy.
Oral Presentation Details:
Presentation Title: AXO-AAV-GM1 for the Treatment of GM1 Gangliosidosis: Preliminary Results from a Phase I-II trial
Abstract Number: 162
Session: Clinical Trials and Advanced Preclinical Studies for Neurologic Diseases
Presenting Author: Cynthia Tifft, MD, PhD, Deputy Clinical Director, National Human Genome Research Institute
Presentation Date and Time: Thursday, May 13, 2021 6:15 PM – 6:30 PM EDT
Presentation Title: AXO-Lenti-PD gene therapy for Parkinson's disease: efficacy, safety, and tolerability data from the second cohort in open-label dose evaluation study SUNRISE-PD at 6 months post administration
Abstract Number: 163
Session: Clinical Trials and Advanced Preclinical Studies for Neurologic Diseases
Presenting Author: Gavin Corcoran, MD, Chief R&D Officer
Presentation Date and Time: Thursday, May 13, 2021 6:30 PM – 6:45 PM EDT
Presentation Title: Immune Modulation Preceding AAV9-GLB1 Gene Therapy Preserves the Possibility for Re-Dosing in Children with GM1 Gangliosidosis
Abstract Number: 179
Session: Immunotherapy and Vaccines
Presenting Author: Precilla D'Souza, DNP, MSN, CRNP, National Human Genome Research Institute
Presentation Date and Time: Thursday, May 13, 2021 7:00 PM – 7:15 PM EDT
Presentation Title: A GLP Safety and Biodistribution Study of AXO-Lenti-PD Manufactured via Two Processes Delivered at a Higher Volume and Flow Rate
Abstract Number: 256
Session: Pharmacology/Toxicology Studies or Assay Development
Presenting Author: Thomas Pack, PhD, Sio Gene Therapies
Presentation Date and Time: Friday, May 14, 2021 from 1:45 PM – 2:00 PM EDT
About AXO-AAV-GM1
AXO-AAV-GM1 delivers a functional copy of the GLB1 gene via an adeno-associated viral (AAV) vector, with the goal of restoring ß-galactosidase enzyme activity for the treatment of GM1 gangliosidosis. The gene therapy is delivered intravenously, which has the potential to broadly transduce the central nervous system and treat peripheral manifestations of the disease as well. Preclinical studies in murine and a naturally-occurring feline model of GM1 gangliosidosis have supported AXO-AAV-GM1's ability to improve ß-galactosidase enzyme activity, reduce GM1 ganglioside accumulation, improve neuromuscular function, and extend survival.
AXO-AAV-GM1 has received both Orphan Drug Designation and Rare Pediatric Disease Designation from the Food and Drug Administration and is the only gene therapy in clinical development for both Type I and Type II GM1 gangliosidosis.
In 2018, Sio licensed exclusive worldwide rights from the University of Massachusetts Medical School for the development and commercialization of gene therapy programs for GM1 gangliosidosis and GM2 gangliosidosis, including Tay-Sachs and Sandhoff diseases.
About AXO-Lenti-PD
AXO-Lenti-PD is an investigational gene therapy for the treatment of Parkinson's disease that is designed to deliver three genes (tyrosine hydroxylase, cyclohydrolase 1, and aromatic L-amino acid decarboxylase) via a single lentiviral vector to encode a set of critical enzymes required for dopamine synthesis, with the goal of reducing variability and restoring steady levels of dopamine in the brain. The investigational gene therapy aims to provide patient benefit for years following a single administration. Axovant expects to dose the first patient in EXPLORE-PD, a randomized, sham controlled study in 2021.
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