Drug intelligence / Profile preview

ABE8e mRNA

Development stage
Preclinical
Lead developer
Broad Institute
Modality
mRNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Gene Therapies
Administration
Topical, Intravenous
01

Overview

ABE8e mRNA is a research-grade gene editing tool that encodes the ABE8e adenine base editor, an evolved version of ABE7.10. ABE8e was developed using directed evolution (PANCE and PACE) and contains eight additional mutations in the TadA deaminase domain, resulting in approximately 590-fold higher editing activity compared to its predecessor. The editor functions by converting A-T base pairs to G-C base pairs without inducing double-stranded DNA breaks or requiring donor DNA templates. When delivered as mRNA via lipid nanoparticles or electroporation, it has demonstrated high efficiency in correcting pathogenic point mutations, such as the COL7A1 nonsense mutation in recessive dystrophic epidermolysis bullosa (RDEB) and reactivating fetal hemoglobin for blood disorders. It is primarily used as a laboratory reagent and is not currently an approved therapeutic.

Other names
ABE8eABE-8eABE 8eadenine base editor 8e mRNA
02

Targets

DNA

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