SBT-101 / Singh Biotech - LARVOL DELTA

An AAV9 Encoding Human ABCD1 (SBT101) Shows Functional Improvement Following Spinal Cord Delivery in a Rodent Model of Adrenomyeloneuropathy (ASGCT 2022) - "Intrathecal delivery of SBT101 to DKO mice demonstrated a dose dependent improvement of both 4-paw grip strength and VLCFA. These data support further preclinical development of AAV9-hABCD1 as a potential treatment of AMN."

Preclinical • CNS Disorders • Gene Therapies • Genetic Disorders • Pain

Optimization of Intrathecal Delivery of an Infused AAV9 Vector for Delivery of a Gene Therapy Candidate for Adrenomyeloneuropathy (AMN) in Non-Human Primates (NHP) (ASGCT 2022) - "We are developing SBT101, an AAV9-based gene therapy encoding a functional hABCD1, for use as a treatment for AMN... These results provide evidence that a 6-hour intrathecal lumbar infusion of an AAV9 vector can produce widespread biodistribution to the SC+DRG comparable to a 24-hour and potentially more extensive than bolus infusion, at doses predicted to be potentially clinically relevant in patients."

Gene Therapies • Pain

Preclinical Pharmacology and Toxicology of Intrathecally Infused AAV9-hABCD1 (SBT101), a Gene Therapy Candidate for AMN, in Non-Human Primates (ASGCT 2022) - "Intrathecal delivery of SBT101, up to 7.6E13 vg/animal, was well tolerated in cynomolgus monkeys at doses predicted to be potentially clinically relevant in AMN patients. The safety profile and biodistribution of SBT101 further enables our path to the clinic for the investigation of AMN treatment."

Preclinical • CNS Disorders • Gene Therapies • Ophthalmology • Pain

Selection of Clinical Doses for SBT101, an AAV9-hABCD1 Vector for the Treatment of Adrenomyeloneuropathy (ASGCT 2022) - "This range falls within doses that have been shown to be associated with activity and efficacy in mice. These levels also translate into doses that efficiently transduce the spinal cord and DRG neurons in NHPs at >25% and have shown safety in both species with a safety margin for the high dose of approximately 2.75‑fold."

Clinical • Gene Therapies • Genetic Disorders • Pain

Optimization of intrathecal delivery of an infused AAV9 vector for delivery of a gene therapy candidate for Adrenomyeloneuropathy (AMN) in Non-Human Primates (NHP) (AAN 2022) - "We are developing SBT101, an AAV9-based gene therapy encoding a functional hABCD1, for use as a treatment for AMN... These results provide evidence that a 6-hour intrathecal lumbar infusion of an AAV9 vector can deliver widespread biodistribution to the SC/DRG comparable to or potentially above a 24-hour or bolus infusion respectively, at doses predicted to be clinically relevant in patients"

Gene Therapies • Genetic Disorders • Pain

Preclinical Pharmacology and Toxicology of intrathecally infused AAV9-hABCD1, a gene therapy candidate for AMN, in Non-Human Primates (AAN 2022) - "These results provide evidence that delivery of SBT101 at doses predicted to be clinically relevant in AMN patients has a promising and relatively unremarkable safety profile in non-human primates, and further enables our path to the clinic for the potential treatment of AMN."

Preclinical • Gene Therapies • Genetic Disorders • Pain

An AAV Encoding Human ABCD1 Shows Dose-Responsive Expression and Function Following Spinal Cord Delivery in a Rodent Model of Adrenomyeloneuropathy (AAN 2022) - "Successful targeting of the spinal cord with SBT101 therapy was demonstrated together with dose-dependent improvement of disease markers in a mouse model of AMN. These data support further preclinical development of AAV9-ABCD1 as a potential treatment of AMN."

Preclinical • CNS Disorders • Genetic Disorders • Pain

[VIRTUAL] ThreeMonth Preclinical Safety Data of AAV9hABCD1 following intrathecal delivery in Nonhuman Primates (ESGCT 2021) - "Microscopic histopathological evaluations revealed slight changes in the spinal cord, DRGs and peripheral tissue, consistent with or less than previously described for AAV9 alone, or in all animals irrespective of treatment following intrathecal delivery. These results suggest SBT101 is tolerable and without toxicity at a range of doses in nonhuman primates, supporting potential translation to humans and further investigation of SBT101 as a potential treatment for AMN."

Preclinical • CNS Disorders • Gene Therapies • Genetic Disorders • Pain

[VIRTUAL] Intrathecal delivery of an AAV encoding human ABCD1 shows doseresponsive expression and activity in a mouse model of adrenomyeloneuropathy (ESGCT 2021) - "Therefore, successful targeting of the spinal cord with SBT101 was demonstrated together with dosedependent improvement of disease markers in a mouse model of AMN. These data support further preclinical investigation of AAV9hABCD1 as a potential therapeutic for the treatment of AMN."

Preclinical • Genetic Disorders • Pain