SBT-101
CAS No. | SBT-101 | Cat. No. | BCP49363 |
Name | SBT-101 | ||
Synonyms | SBT101;SBT 101; | ||
Formula | M. Wt |
Description | SBT101 is the first AAV-based gene therapy in development designed to compensate for the disease-causing ABCD1 mutation, to increase ABCD1 expression, and reduce very long chain fatty acid (VLCFA) levels specifically for people living with adrenomyeloneuropathy (AMN). | ||
Pathways | Jak/Stat Pathway Others Pathway | ||
Targets | Others Stat |
Structure
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