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ABE8e-SpRY
- Development stage
- Preclinical
- Lead developer
- Beam Therapeutics
- Modality
- CRISPR-Cas9 → CRISPR Systems → Programmable Nucleases → Gene Editing → Gene Therapies, Gene Silencing → Gene Therapies, RNA Therapeutics → Nucleic Acid Therapeutics, Gene Addition/Replacement → Gene Therapies, Lipid-based Nanoparticles → Nanoparticles → Drug Delivery Systems
- Administration
- Parenteral
01
Overview
ABE8e-SpRY is an adenine base editor (ABE) composed of a high-activity deoxyadenosine deaminase (ABE8e) fused to a near-PAMless SpCas9 variant (SpRY). This gene-editing tool is designed to perform precise adenine-to-guanine (A-to-G) nucleotide conversions without requiring double-strand DNA breaks. In the context of sickle cell disease (SCD), ABE8e-SpRY is utilized to target the pathogenic mutation in the *HBB* gene, converting it into the naturally occurring, benign *HBB* Makassar variant. The SpRY component significantly expands the range of addressable genomic sites by bypassing traditional Protospacer Adjacent Motif (PAM) restrictions. Research indicates that ABE8e-SpRY can achieve high editing efficiencies in hematopoietic stem and progenitor cells (HSPCs), particularly in fetal liver-derived cells, supporting its potential for in utero or early postnatal therapeutic interventions.
- Other names
- adenine base editor 8e-SpRY
02
Targets
Hb (Hemoglobin)
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