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Nb.rAAV9
- Development stage
- Preclinical
- Lead developer
- UMass Chan Medical School
- Modality
- Nanobodies (VHH) → Antibody Fragments → Engineered Antibody Formats → Antibody-Based Therapeutics, Gene Therapies
- Administration
- Intravenous
01
Overview
Nb.rAAV9 is an engineered recombinant adeno-associated virus serotype 9 (rAAV9) vector designed for targeted gene delivery to synovial fibroblasts in the treatment of rheumatoid arthritis. Developed by researchers at the University of Massachusetts Chan Medical School, the vector features anti-fibroblast activation protein alpha (FAPα) nanobody sequences grafted onto the N-terminus of the VP2 capsid protein. This modification aims to improve tissue specificity and reduce off-target transduction, particularly in the liver and muscle, by targeting FAPα, which is highly expressed on activated synovial fibroblasts in inflamed joints. In preclinical studies, these vectors have been used to deliver therapeutic payloads such as soluble IL-1 receptor antagonist (sIL-1Ra) to suppress joint inflammation and bone erosion.
- Other names
- nanobody-guided AAV9anti-FAPα nanobody-grafted AAV9
02
Targets
IL-1R (Interleukin-1 receptor type 1 (IL-1R1))FAP (Fibroblast activation protein alpha)
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