Target intelligence / Profile preview

BT-001

Molecular classification
Oncolytic virus, Gene therapy vector
01

Overview

BT-001 is a genetically engineered oncolytic virus based on a modified vaccinia virus (Copenhagen strain, double deleted). It is designed for cancer immunotherapy and armed with two key payloads: a human recombinant anti-CTLA-4 monoclonal antibody (mAb, clone 4-E03 IgG1) and the cytokine GM-CSF (Granulocyte-Macrophage Colony-Stimulating Factor). It acts by selectively infecting and replicating within tumor cells, leading to direct lysis and the local expression of anti-CTLA-4 and GM-CSF. The anti-CTLA-4 mAb blocks CTLA-4 signaling, depleting Tregs and promoting effector T cell activity against tumors. GM-CSF recruits dendritic cells and other immune effectors to enhance antigen presentation.

Other names
Oncolytic vaccinia virus expressing anti-CTLA-4 and GM-CSFModified vaccinia virus (Copenhagen strain, double deleted)MVV-CTLA4-GMCSF
02

Mechanism of action

BT-001 is an oncolytic virus that selectively infects and replicates within tumor cells, leading to tumor lysis. It also expresses a human anti-CTLA-4 antibody to block CTLA-4 signaling, depleting Tregs and promoting T cell activity. Furthermore, it produces GM-CSF to enhance immune cell recruitment and activation within the tumor microenvironment.

03

Biological functions

Cancer immunotherapyTumor lysisImmune cell recruitmentT cell activationCheckpoint inhibition
04

Disease associations

Cancer
05

Safety considerations

Viral sheddingDevelopment of neutralizing antibodies against vectorPotential for off-target immune activation
06

Interacting drugs

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