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PBGENE-HbE
- Development stage
- Preclinical
- Lead developer
- Precision BioSciences
- Modality
- CRISPR-Cas9 → CRISPR Systems → Programmable Nucleases → Gene Editing → Gene Therapies
- Administration
- Intravenous
01
Overview
PBGENE-HbE is an in vivo gene editing product developed through a collaboration between Precision BioSciences and Novartis Pharma AG. It utilizes Precision's proprietary ARCUS nuclease technology to target hemoglobinopathies, specifically focusing on sickle cell disease and beta thalassemia. Precision BioSciences is responsible for the development of the ARCUS nuclease and in vitro characterization, while Novartis Pharma AG handles all subsequent development, manufacturing, and commercialization of the product.
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