Drug intelligence / Profile preview

CD123 Shielded-Cell & Immunotherapy Pair

Development stage
Preclinical
Lead developer
Cimeio Therapeutics
Modality
Stem Cell Therapies → Cell Therapies, Gene Therapies
Administration
Intravenous
01

Overview

CD123 Shielded-Cell & Immunotherapy Pair (CD123 SCIP) is a preclinical-stage therapeutic system developed by Cimeio Therapeutics. It consists of epitope-edited hematopoietic stem and progenitor cells (HSPCs) paired with a CD123-targeted immunotherapy. CD123 (interleukin-3 receptor alpha-chain) is highly expressed in myeloid malignancies, such as acute myeloid leukemia (AML), but is also present on healthy HSPCs, posing a risk of severe myelotoxicity with standard CD123-targeted therapies. Cimeio's approach utilizes gene editing (such as CRISPR/Cas9) to introduce single amino acid substitutions into the CD123 epitope of donor HSPCs. These engineered cells maintain normal CD123 expression and physiological function but are shielded from binding and depletion by the paired CD123-targeted immunotherapy (such as monoclonal antibodies, antibody-drug conjugates, T-cell engagers, or CAR-T cells). This allows for the selective eradication of CD123-positive malignant cells while preserving the transplanted, shielded hematopoietic system.

Other names
CD123 SCIPCD-123 SCIPCD 123 SCIPSCIP
02

Targets

IL3RA (Interleukin 3 Receptor)CD3 (T-cell surface glycoprotein CD3)FcγR (Low affinity immunoglobulin gamma Fc region receptor II-c)IL3 (IL-3)

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