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BPX-601
- Development stage
- Discontinued
- Lead developer
- Bellicum Pharmaceuticals
- Modality
- Nucleic Acid-Directed Small Molecules → Small Molecules, Gene Addition/Replacement → Gene Therapies, Classical Binding Small Molecules → Small Molecules, Vaccines & Immunotherapeutics, Gene Silencing → Gene Therapies, Covalent Small Molecules → Small Molecules, Gene Editing → Gene Therapies, CAR-T Cells → Engineered T Cells → Adoptive Cell Transfer → Cell Therapies
- Administration
- Intravenous
01
Overview
BPX-601 is an investigational autologous chimeric antigen receptor T cell (CAR-T) therapy developed by Bellicum Pharmaceuticals for the treatment of solid tumors expressing prostate stem cell antigen (PSCA), including cancers of the prostate, pancreas, and stomach. The therapy uses a proprietary inducible MyD88/CD40 co-stimulatory domain (iMC), which can be pharmacologically activated by rimiducid to enhance T cell proliferation, persistence, and cytokine production. This design aims to improve CAR-T efficacy in solid tumors by overcoming immune inhibitory mechanisms such as PD-1 and TGF-beta. Unlike standard CARs that rely solely on antigen presence for activation and survival, BPX-601 allows external control over T-cell activity through rimiducid dosing[1][4][6]. Clinical trials demonstrated preliminary biologic activity but also revealed significant toxicities at higher doses; development was discontinued after phase 1/2 studies in advanced solid tumors[2][3][4].
- Other names
- GoCAR-T
02
Targets
PSCA (Prostate stem cell antigen)
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