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AAV9-ALOX5
- Development stage
- Preclinical
- Lead developer
- University of Minnesota
- Modality
- Gene Therapies
- Administration
- Intravenous
01
Overview
AAV9-ALOX5 is an experimental cardiac-specific gene therapy designed to treat heart failure with preserved ejection fraction (HFpEF) associated with diabetes mellitus. It utilizes an adeno-associated virus serotype 9 (AAV9) vector to deliver the ALOX5 gene, which encodes the enzyme arachidonate 5-lipoxygenase (5-LOX), under the control of the cardiac-specific alpha-myosin heavy chain (alpha-MHC) promoter. By upregulating 5-LOX expression specifically in cardiomyocytes, the therapy promotes the synthesis of specialized pro-resolving mediators (SPMs), such as Resolvin D1 (RvD1). This localized increase in SPMs helps resolve cardiac inflammation and reduces NLRP3 inflammasome activity, thereby improving diastolic function without causing systemic immunosuppression.
- Other names
- AAV9-alphaMHC-ALOX5AAV-9-alphaMHC-ALOX5AAV 9-alphaMHC-ALOX5
02
Targets
ALOX5 (Arachidonate 5-lipoxygenase)Host galactose-containing glycans
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