Drug intelligence / Profile preview

SGT-53 + topotecan + cyclophosphamide

Development stage
Phase 1
Lead developer
SynerGene Therapeutics
Modality
Gene Therapies, Small Molecules, Nanoparticles → Drug Delivery Systems
Administration
Intravenous
01

Overview

SGT-53 + topotecan + cyclophosphamide is an experimental combination therapy consisting of **SGT-53**, a tumor-targeted liposomal nanocomplex delivering wild-type TP53 gene, with the chemotherapeutics **topotecan** and **cyclophosphamide**. SGT-53 uses a liposome encapsulating p53 cDNA and targets tumors via an anti-transferrin receptor single-chain antibody fragment. This nanomedicine aims to restore p53 function in tumor cells, enhancing apoptosis and sensitizing tumors to chemotherapy and immunotherapy. Topotecan is a topoisomerase I inhibitor causing DNA damage, while cyclophosphamide is an alkylating agent that crosslinks DNA, leading to apoptosis. The combination is under investigation for various advanced solid tumors, including potential applications in brain tumors and relapsed or refractory pediatric solid tumors[1][4][5][6].

02

Targets

DNATOP1 (DNA Topoisomerase I)TFRC (Transferrin Receptor)TP53 (Cellular Tumor Antigen p53 R175H)

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