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umiposgene parvec
- Development stage
- Unknown
- Lead developer
- AskBio
- Modality
- Gene Therapies
- Administration
- Intra-arterial
01
Overview
Umiposgene parvec (AB-1002) is an investigational adeno-associated virus (AAV)-based gene therapy designed to treat congestive heart failure, specifically non-ischemic heart failure with reduced ejection fraction (HFrEF). Developed by AskBio (a subsidiary of Bayer), the therapy delivers a constitutively active, truncated version of the protein phosphatase inhibitor-1 (I-1c) directly to the heart via a one-time cardiac catheter infusion. By inhibiting protein phosphatase 1 (PP1), umiposgene parvec aims to restore the phosphorylation of phospholamban and improve calcium handling in cardiomyocytes, thereby enhancing cardiac contractile function. It is currently being evaluated in the Phase 2 GenePHIT clinical trial, which is one of the largest randomized gene therapy trials in heart failure to date.
- Other names
- AB-1002AB1002AB 1002I-1c gene therapyI1c gene therapyI 1c gene therapy
02
Targets
PP1 (Protein phosphatase 1)DUSP1
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