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PBGENE-NVS

Development stage: Preclinical
Lead developer: Novartis
Modality: Gene Therapies
Administration: Intravenous

Overview

PBGENE-NVS is an investigational gene editing therapy being developed by Precision BioSciences in collaboration with Novartis for the treatment of hemoglobinopathies, including sickle cell disease and beta thalassemia. The therapy utilizes Precision's proprietary ARCUS genome editing platform to enable targeted gene insertion. The program aims to provide a potentially curative approach for patients with these inherited blood disorders by correcting or compensating for the underlying genetic defects[1][3].

Targets

HBB locus (Hemoglobin subunit beta (HBB) gene locus)

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