Drug intelligence / Profile preview

ARTbase-A1

Development stage
Preclinical
Lead developer
Accuredit Therapeutics
Modality
CRISPR-Cas9 → CRISPR Systems → Programmable Nucleases → Gene Editing → Gene Therapies
Administration
Intravenous, Subcutaneous, Parenteral
01

Overview

ARTbase-A1 is a proprietary adenine base editor (ABE) developed by Accuredit Therapeutics for the treatment of metabolic dysfunction-associated steatohepatitis (MASH). It is designed to introduce protective loss-of-function (LoF) mutations into the HSD17B13 gene, which is strongly associated with reduced risks of chronic liver disease. Unlike traditional Cas9 nucleases that create double-strand breaks, ARTbase-A1 utilizes base editing technology to achieve precise genomic modifications. Preclinical data presented at ASGCT 2026 demonstrated that ARTbase-A1 provides efficient and clean targeted editing in humanized mouse models and primary human hepatocytes, outperforming the clinical benchmark ABE8.8. Additionally, the therapeutic edits made by ARTbase-A1 can be reversed using cytosine base editors (CBE), offering a unique safety feature for one-time genetic treatments.

02

Targets

HSD17B13 (17β-Hydroxysteroid Dehydrogenase Type 13)

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