AskBio’s AB-1003 Secures the US FDA’s Rare Pediatric Disease and Orphan Drug Designations to Treat Limb-Girdle Muscular Dystrophy Type 2I/R9
Shots:
- The US FDA has granted ODD & RPDD to AB-1003 (also known as LION-101) for the treatment of limb-girdle muscular dystrophy type 2I/R9 (LGMD2I/R9)
- AB-1003 (IV) is being investigated under P-I/II (LION-CS101) study vs matching PBO for its safety among adults (18-65yrs.) with limb-girdle muscular dystrophy type 2I/R9. First patient has been dosed, with recruitment underway
- AB-1003 uses an AAV vector to transfer a healthy FKRP gene, a common approach in gene therapy due to AAVs' safety, non-replicating nature, and ease of engineering
Ref: AskBio | Image: AskBio
Related News:- AskBio’s (Bayer's Subsidiary) AB-1005 Gains the US FDA’s Fast Track and MHRA’s Innovation Passport Designations for Parkinson’s Disease
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Disha is a content writer at PharmaShots. She is passionate and curious about recent updates and developments in MedTech and Pharma industry. She covers news related to clinical trial results and updates. She can be contacted at connect@pharmashots.com.
