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ASM-AAV (MOD3)
- Development stage
- Preclinical
- Lead developer
- Children's National Hospital
- Modality
- Gene Therapies
- Administration
- Intravenous
01
Overview
ASM-AAV (MOD3) is an optimized, liver-targeted adeno-associated virus (AAV) gene therapy candidate designed for the treatment of dysferlinopathies, including Limb-Girdle Muscular Dystrophy 2B (LGMD2B) and Miyoshi Myopathy (MM). Developed by researchers at Children's National Medical Center, the therapy delivers an optimized version of the human acid sphingomyelinase (ASM) gene (SMPD1). In dysferlinopathy, mutations in the DYSF gene impair the injury-triggered secretion of ASM, which is crucial for muscle cell membrane repair. ASM-AAV (MOD3) utilizes a codon-optimized cDNA sequence with the removal of the C-terminal cysteine (MOD3 modification), which results in a constitutively active form of ASM upon secretion from the liver. This modification significantly enhances the enzymatic activity of the secreted ASM, thereby lowering the required viral dose to facilitate myofiber membrane repair.
- Other names
- AAV-ASMAAV-SMPD1AAV-SMPD-1AAV-SMPD 1ASM-AAV
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