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Beam Therapeutics in vivo HSC base editing program
- Development stage
- Preclinical
- Lead developer
- Beam Therapeutics
- Modality
- Adenine Base Editors → Base Editing → Gene Editing → Gene Therapies, CRISPR-Cas9 → CRISPR Systems → Programmable Nucleases → Gene Editing → Gene Therapies, Cytosine Base Editors → Base Editing → Gene Editing → Gene Therapies, RNA Therapeutics → Nucleic Acid Therapeutics, Lipid-based Nanoparticles → Nanoparticles → Drug Delivery Systems
- Administration
- Intravenous
01
Overview
Beam Therapeutics is developing an in vivo base editing program for the treatment of sickle cell disease and beta-thalassemia. As part of the company's 'Wave 3' strategy, this approach utilizes lipid nanoparticles (LNPs) to deliver base editors directly to hematopoietic stem cells (HSCs) via intravenous infusion. By targeting genetic regulators of fetal hemoglobin (HbF) expression, such as the BCL11A erythroid enhancer or the HBG1/2 promoters, the therapy aims to induce the production of HbF to compensate for defective adult hemoglobin. This in vivo method is designed to provide a one-time curative treatment while eliminating the need for the intensive conditioning regimens and complex ex vivo cell manufacturing required for traditional autologous stem cell transplants.
- Other names
- Beam Wave 3 in vivo programIn vivo base editing for sickle cell diseaseIn vivo HSC editing program
02
Targets
Hemoglobin subunit gamma-1 promoterHbF (Fetal hemoglobin)HBG2 (Hemoglobin subunit gamma-2)
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