American Society for Gene and Cell Therapy 26th Annual Meeting | May 16-20, 2023

• In vivo selection in non-human primates identifies AAV capsids having enhanced transgene expression and biodistribution in spinal cord after intrathecal administration
• Healthcare utilization, burden of illness, and mortality in adults with AMN: the case for genetic therapies

International Society for Pharmacoeconomics and Outcomes Research 2023 | May 7-10, 2023

• Economic impact of AMN on direct medical costs in commercially insured US adults
• Hospitalizations, clinical characteristics, and mortality in adults with AMN

American Academy of Neurology 2023 Annual Meeting | April 22-27, 2023

• Substantial burden of illness and mortality in men with AMN

European Academy of Neurology 8th Annual Congress | June 25 – 28, 2022

• CYGNET: A prospective multicentre observational study of disease progression in patients with AMN

American Society of Gene & Cell Therapy Annual Meeting | May 16 – 19, 2022

• Selection of Clinical Doses for SBT101, an AAV9-hABCD1 Vector for the Treatment of Adrenomyeloneuropathy
• An AAV9-Encoding Human ABCD1 Shows Functional Improvement Following Spinal Cord Delivery in a Rodent Model of AMN
• Preclinical Pharmacology and Toxicology of Intrathecally Infused AAV9-hABCD1 (SBT101), a Gene Therapy Candidate for AMN, in Non-Human Primates
• Optimization of Intrathecal Delivery of an Infused AAV9 Vector for Delivery of a Gene Therapy Candidate for AMN in Non-Human Primates

American Academy of Neurology Annual Meeting | Apr 2 – 7, 2022

• Preclinical Pharmacology and Toxicology of Intrathecally Infused AAV9-hABCD1, a Gene Therapy Candidate for AMN, in Non-Human Primates
• Optimization of Intrathecal Delivery of an Infused AAV9 Vector for Delivery of a Gene Therapy Candidate for AMN in Non-Human Primates
• An AAV Encoding Human ABCD1 Shows Dose-Responsive Expression and Function Following Spinal Cord Delivery in a Rodent Model of AMN

ESGCT Congress | Oct 19, 2021 – Oct 22, 2021

• Intrathecal delivery of an AAV encoding human ABCD1 shows dose-responsive expression and activity in a mouse model of Adrenomyeloneuropathy
• Three-Month Preclinical Safety Data of AAV9-hABCD1 following intrathecal delivery in Non-human Primates