SBT101

SBT101 is an investigational gene therapy developed by SwanBio Therapeutics designed to treat adrenomyeloneuropathy (AMN), a rare, adult-onset degenerative spinal cord disease that occurs in patients with adrenoleukodystrophy (ALD). SBT101 uses a non-replicating recombinant adeno-associated virus serotype 9 (AAV9) vector to deliver a functional copy of the human ABCD1 gene directly into the central nervous system via intrathecal administration. The therapy aims to compensate for disease-causing mutations in the ATP-binding cassette sub-family D member 1 (ABCD1) gene, increase ABCD1 protein expression, and reduce toxic accumulation of very long chain fatty acids (VLCFA) in affected tissues. Preclinical studies have shown dose-dependent improvements in AMN disease markers and good tolerability. There are currently no approved therapies for AMN; SBT101 is being evaluated as a potential first-in-class, disease-modifying treatment.

Other names

AAV9-hABCD1AAV-9-hABCD1AAV 9-hABCD1AAV9-ABCD1AAV-9-ABCD1AAV 9-ABCD1