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AAV9-FKRP
- Development stage
- Unknown
- Lead developer
- AskBio
- Modality
- Gene Therapies
- Administration
- Intravenous
01
Overview
AAV9-FKRP is a target-specific generic descriptor for gene therapies utilizing a recombinant adeno-associated virus serotype 9 (AAV9) vector to deliver a functional copy of the fukutin-related protein (FKRP) gene. This therapeutic approach is primarily developed to treat FKRP-related dystroglycanopathies, most notably limb-girdle muscular dystrophy type 2I/R9 (LGMDR9) and congenital muscular dystrophy type 1C (MDC1C). Mutations in the FKRP gene lead to defective glycosylation of alpha-dystroglycan, a critical glycoprotein for muscle fiber stability. By delivering a functional FKRP gene, AAV9-FKRP therapies aim to restore alpha-dystroglycan glycosylation and improve muscle structure and function. Multiple distinct programs have been developed under this architecture, including Atamyo Therapeutics' ATA-100 (formerly GNT0006) and AskBio's AB-1003 (formerly LION-101), both of which are in clinical development.
- Other names
- AAV9-mediated FKRP gene therapyAAV-9-mediated FKRP gene therapyAAV 9-mediated FKRP gene therapyrAAV9-FKRPrAAV-9-FKRPrAAV 9-FKRP
02
Targets
FKRP (Fukutin-related protein)
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