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CD117 ADC
- Development stage
- Preclinical
- Lead developer
- CRISPR Therapeutics
- Modality
- CRISPR-Cas9 → CRISPR Systems → Programmable Nucleases → Gene Editing → Gene Therapies, Cytotoxic ADCs → Antibody-Drug Conjugates (ADCs) → Antibody Conjugates → Antibody-Based Therapeutics
- Administration
- Intravenous
01
Overview
CRISPR Therapeutics is developing an anti-CD117 (c-Kit) antibody-drug conjugate (ADC) as a targeted conditioning agent for patients with hemoglobinopathies, such as sickle cell disease and beta thalassemia. The ADC is designed to selectively target and deplete CD117-positive hematopoietic stem cells (HSCs) in the bone marrow, providing a more targeted and potentially less toxic alternative to traditional busulfan-based myeloablative conditioning. This program is intended to support the administration of ex vivo gene-edited therapies like exagamglogene autotemcel (Casgevy) by expanding the eligible patient population. Additionally, the company is conducting research into in vivo HSC editing, which aims to deliver CRISPR/Cas9 components directly to stem cells within the patient's body, potentially eliminating the need for conditioning altogether and expanding the geographic reach of genetic medicines.
- Other names
- anti-CD117 ADCanti-CD-117 ADCanti-CD 117 ADCc-Kit ADCNext-generation conditioning (CRISPR Therapeutics)CD117 ADC / In vivo HSC editing
02
Targets
KIT (c-KIT proto-oncogene receptor tyrosine kinase)
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