Drug intelligence / Profile preview

ABE8.8

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

Overview

ABE8.8 is a high-efficiency adenine base editor (ABE) variant designed to enable precise adenine-to-guanine (A-to-G) transitions in genomic DNA without inducing double-strand breaks. Developed by researchers at the Broad Institute, ABE8.8 consists of an evolved TadA-8e deoxyadenosine deaminase fused to a CRISPR-Cas9 nickase (nCas9). This variant features multiple mutations in the deaminase domain that significantly enhance its editing potency and broaden its targeting range compared to earlier generations like ABE7.10. In the context of therapeutic development, ABE8.8 is frequently utilized as a clinical benchmark for evaluating the performance of next-generation base editing platforms. It has been specifically investigated for its ability to introduce protective loss-of-function mutations in the HSD17B13 gene for the treatment of metabolic dysfunction-associated steatohepatitis (MASH).

Other names
Adenine Base Editor 8.8
02

Targets

ssDNA (Single-stranded DNA)

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