Drug intelligence / Profile preview

adenosine base editor

Development stage
Preclinical
Lead developer
ElevateBio
Modality
CRISPR-Cas9 → CRISPR Systems → Programmable Nucleases → Gene Editing → Gene Therapies
Administration
Intravenous
01

Overview

Adenosine base editors (ABEs) are a class of CRISPR-based genome editing tools designed to convert A•T base pairs into G•C base pairs without generating double-strand DNA breaks. The architecture typically involves a catalytically inactive or nickase version of a CRISPR protein (such as Cas9 or other RNA-guided nucleases) fused to an engineered deoxyadenosine deaminase enzyme. When directed to a target genomic site by a guide RNA (gRNA), the deaminase converts adenine to inosine, which the cell's machinery subsequently reads as guanine. This technology, being advanced by companies like Life Edit Therapeutics (ElevateBio), is particularly suited for correcting pathogenic point mutations in monogenic diseases and offers a potentially safer profile compared to traditional CRISPR-Cas9 nucleases by avoiding stochastic indels associated with non-homologous end joining.

Other names
adenine base editor
02

Targets

On-target genomic DNA locus complementary to the sgRNA

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