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Drug intelligence / Profile preview
AAV9-mini-agrin + AAV9-αLNNd
- Development stage
- Preclinical
- Lead developer
- Sealy
- Modality
- Gene Therapies
- Administration
- Intravenous
01
Overview
AAV9-mini-agrin + AAV9-αLNNd is an investigational dual-vector gene therapy developed by SEAL Therapeutics (a spin-off of the University of Basel) in collaboration with Rutgers University for the treatment of LAMA2-related muscular dystrophy (LAMA2 MD, also known as MDC1A). LAMA2 MD is a severe congenital muscular dystrophy caused by mutations in the LAMA2 gene, which encodes the laminin-α2 chain of laminin-211. Because the coding sequence for laminin-α2 is too large to fit into a single adeno-associated virus (AAV) vector, this therapy utilizes the Simultaneous Expression of Artificial Linkers (SEAL) technology. It co-delivers two engineered linker proteins via separate AAV9 vectors: mini-agrin (mag), which binds to the compensatory laminin-411 and anchors it to α-dystroglycan on the cell membrane, and αLNNd, which binds to laminin-411 and enables its polymerization. Together, these linkers functionally replace the missing laminin-211, restoring basement membrane assembly, improving muscle histology and force, and preventing peripheral neuropathy.
- Other names
- AAV9-CBh-mag + AAV9-CBh-αLNNdAAV9-mag + AAV9-αLNNdAAV9-mini-agrin + AAV9-αLNNdΔG2'SEAL technologySimultaneous Expression of Artificial Linkers
02
Targets
DAG1 (Dystroglycan 1)LAMA4 (Laminin subunit alpha-4)
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