PRECISION BIOSCIENCES INC
For the period ended Nov 4, 2024. Filed Nov 4, 2024.
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Nov 4, 2024 · Filed Nov 4, 2024
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Contents
EX-99.1 · Partnered Programs
Partnered Programs
iECURE-OTC (Gene Insertion Program): Led by iECURE, ECUR-506 is an ARCUS-mediatedin vivo gene editing program currently in a first-in-human phase 1/2 trial (OTC-HOPE) evaluating ECUR-506 as a potential treatment for neonatal onset ornithine transcarbamylase (OTC) deficiency. iECURE expects initial data from this trial to be available in 2025.
PBGENE-NVS (Gene Insertion Program): Precision continues to advance its gene editing program with Novartis to develop a custom ARCUS nuclease for patients with hemoglobinopathies, such as sickle cell disease and beta thalassemia. The collaborative intent is toinsert, in vivo, a therapeutic transgene as a potential one-time transformative treatment administered directly to the patient to overcome disparities in patient access to treatment with other therapeutic technologies, including those that are targeting anex vivo gene editing approach.
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