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YOLT-204

Development stage: Phase 1
Lead developer: YolTech Therapeutics
Modality: Gene Silencing → Gene Therapies, Gene Editing → Gene Therapies, Gene Addition/Replacement → Gene Therapies, Lipid-based Nanoparticles → Nanoparticles → Drug Delivery Systems
Administration: Intravenous

Overview

YOLT-204 is an in vivo gene editing therapy developed by YolTech Therapeutics for the treatment of transfusion-dependent beta-thalassemia (TDT). It uses lipid nanoparticles (LNPs) to deliver gene-editing tools to hematopoietic stem cells, targeting the regulatory region of hemoglobin to induce fetal hemoglobin production. This approach aims to restore hemoglobin balance and reduce the need for blood transfusions in TDT patients. YOLT-204 also shows potential for treating sickle cell disease by increasing fetal hemoglobin levels.

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