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EmendoBio ELANE CRISPR therapy
- Development stage
- Preclinical
- Lead developer
- EmendoBio
- Modality
- CRISPR-Cas9 → CRISPR Systems → Programmable Nucleases → Gene Editing → Gene Therapies
- Administration
- Intravenous
01
Overview
EmendoBio's ELANE-targeted CRISPR therapy is an investigational genome editing treatment developed for the treatment of ELANE-related severe congenital neutropenia (SCN). Utilizing the company's proprietary OMNI nuclease technology platform, the therapy is designed to perform allele-specific editing, selectively knocking out the mutant *ELANE* allele while sparing the functional wild-type allele. This approach aims to restore normal neutrophil differentiation and maturation in patients with SCN, a life-threatening bone marrow failure disorder. Preclinical studies using patient-derived hematopoietic stem cells have demonstrated the restoration of neutrophil production with no detectable off-target effects. Beyond SCN, EmendoBio is exploring the application of its CRISPR platform in other hematology, ophthalmology, and immuno-oncology indications.
- Other names
- ELANE-targeted CRISPR therapyOMNI nuclease ELANE therapy
02
Targets
ELANE (Human Neutrophil Elastase)
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