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AB-1003
- Development stage
- Unknown
- Lead developer
- AskBio
- Modality
- Gene Addition/Replacement → Gene Therapies, Gene Editing → Gene Therapies, Gene Silencing → Gene Therapies
- Administration
- Intravenous
01
Overview
AB-1003 (also known as LION-101) is an investigational recombinant adeno-associated virus (AAV) vector-based gene therapy designed for the treatment of limb-girdle muscular dystrophy type 2I/R9 (LGMD2I/R9). LGMD2I/R9 is a rare, progressive genetic neuromuscular disorder caused by mutations in the fukutin-related protein (FKRP) gene, which leads to a deficiency in functional FKRP enzyme activity and subsequent progressive muscle wasting. AB-1003 utilizes an engineered AAV9 capsid to deliver a functional copy of the human FKRP gene preferentially to muscle tissue, aiming to restore FKRP enzyme activity, promote proper glycosylation of alpha-dystroglycan, and stabilize muscle fiber integrity. Developed by AskBio (a subsidiary of Bayer), the therapy is administered as a single intravenous infusion and is currently being evaluated in Phase I/II clinical trials.
- Other names
- Itabacelline-29Itabacelline29Itabacelline 29
02
Targets
FKRP (Fukutin-related protein)
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