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base editing therapy targeting ELANE
- Development stage
- Preclinical
- Lead developer
- Centre for Stem Cell Research
- Modality
- Adenine Base Editors → Base Editing → Gene Editing → Gene Therapies, Cytosine Base Editors → Base Editing → Gene Editing → Gene Therapies
- Administration
- Intravenous
01
Overview
PRECISE is an adenine base editing therapy developed to treat severe congenital neutropenia (CN) and cyclic neutropenia (CyN) by targeting the ELANE (elastase, neutrophil expressed) gene. The therapy utilizes an adenine base editor (specifically ABE8.20-m) to introduce precise A-to-G conversions within the TATA box of the ELANE promoter, which suppresses the expression of neutrophil elastase and restores normal granulocytic differentiation in hematopoietic stem and progenitor cells (HSPCs). Developed by researchers at the Centre for Stem Cell Research (inStem) and Christian Medical College (CMC) Vellore, this approach aims to provide a safer alternative to double-strand break-mediated CRISPR/Cas9 strategies by avoiding p53 pathway activation and unintended genetic alterations. Additional research presented at ASH 2025 explores complementary base editing strategies for ELANE knockout, including the generation of premature termination codons and splice site disruption using various cytosine (CBE6b) and adenine (ABE8e) base editor variants.
- Other names
- PRECISEELANE base editing therapyadenine base editing therapy targeting ELANE
02
Targets
Neutrophil elastase promoter
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