Drug intelligence / Profile preview

CD45-saporin

Development stage
Preclinical
Lead developer
Massachusetts General Hospital
Modality
Cytotoxic ADCs → Antibody-Drug Conjugates (ADCs) → Antibody Conjugates → Antibody-Based Therapeutics
Administration
Intravenous
01

Overview

CD45-saporin is an experimental immunotoxin designed as a non-genotoxic conditioning agent for hematopoietic stem cell transplantation (HSCT). It consists of a monoclonal antibody targeting the CD45 receptor (PTPRC), which is expressed on nearly all hematopoietic cells, conjugated to saporin, a potent Type I ribosome-inactivating protein (RIP) derived from the plant *Saponaria officinalis*. Upon binding to CD45, the conjugate is internalized via endocytosis; saporin then escapes into the cytosol where it irreversibly halts protein synthesis by depurinating the sarcin/ricin loop of 28S ribosomal RNA. This targeted approach aims to deplete host hematopoietic stem cells and immune cells to create space for donor engraftment while sparing non-hematopoietic tissues, thereby avoiding the systemic toxicities and DNA damage associated with traditional radiation or chemotherapy-based conditioning regimens. Preclinical research has demonstrated its potential in treating models of sickle cell anemia and severe combined immunodeficiency (SCID).

Other names
anti-CD45-saporinanti-CD-45-saporinanti-CD 45-saporinCD45-targeted immunotoxinCD-45-targeted immunotoxinCD 45-targeted immunotoxin
02

Targets

28S rRNA (28S ribosomal RNA)PTPRC (Receptor-type tyrosine-protein phosphatase C)

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