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AAV-LSEC1
- Development stage
- Preclinical
- Lead developer
- Children's Hospital of Philadelphia
- Modality
- Gene Therapies
- Administration
- Intravenous
01
Overview
AAV-LSEC1 is a bioengineered adeno-associated virus (AAV) capsid variant designed for targeted gene therapy. Developed by researchers at The Children's Hospital of Philadelphia and the University of Pennsylvania, AAV-LSEC1 was identified through an in vivo screen of an AAV2-based capsid library containing random 7-mer peptide insertions in the VP3 capsid protein. It exhibits significantly enhanced tropism for liver sinusoidal endothelial cells (LSECs), which are the endogenous site of coagulation factor VIII (FVIII) synthesis. By directing FVIII expression to its native cellular source, AAV-LSEC1 serves as a promising platform for next-generation gene therapies for hemophilia A.
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