Drug intelligence / Profile preview

UB-VV111

Development stage
Phase 1
Lead developer
Umoja Biopharma
Modality
Small Molecules, CAR-T Cells → Engineered T Cells → Adoptive Cell Transfer → Cell Therapies, Lentiviral Vectors → Retroviral Vectors → Viral Vectors → Gene Addition/Replacement → Gene Therapies
Administration
Intravenous
01

Overview

UB-VV111 is an investigational gene therapy designed to generate CD19-directed chimeric antigen receptor (CAR) T cells in vivo for the treatment of hematologic malignancies. It utilizes a third-generation, self-inactivating, replication-incompetent lentiviral vector (LVV) with a surface-engineered viral envelope pseudotyped with cocal virus fusion glycoprotein and a membrane-bound multidomain fusion (MDF) protein. The vector encodes two key components: an anti-CD19 CAR (comprising an FMC63 single-chain variable fragment and 4-1BB/CD3ζ intracellular signaling domains) and a rapamycin-activated cytokine receptor (RACR), which is designed to enhance the enrichment and expansion of engineered CAR T cells in vivo via STAT5 signaling. This approach aims to overcome limitations of traditional ex vivo CAR-T therapies by enabling direct in situ generation of therapeutic CAR T cells within the patient’s body, potentially eliminating the need for lymphodepletion or complex cell manufacturing processes. UB-VV111 is being developed primarily for B-cell malignancies such as large B-cell lymphoma and chronic lymphocytic leukemia[2][3][6][7].

02

Targets

RacR (DNA-binding transcriptional repressor RacR)CD19 (B lymphocyte antigen CD19)

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