First patient dosed with experimental BT-001 therapy for rectal cancer

11 November 2026
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First patient dosed with experimental BT-001 therapy for rectal cancer

First patient dosed with experimental BT-001 therapy for rectal cancer


Transgene, together with the University of Copenhagen, has started a Phase 1 clinical trial of BT-001, with the first patient now receiving treatment.


The study focuses on patients with pMMR rectal cancer, which represents around 95% of rectal cancer cases and has historically shown limited response to immunotherapy alone.


How does BT-001 work?

BT-001 is an engineered oncolytic virus injected directly into the tumour. It is designed to attack tumour cells and stimulate the immune system to respond more strongly against the cancer. Patients then receive the immunotherapy drug pembrolizumab intravenously.


Researchers are investigating whether this combination can:

• strengthen the immune response against the tumour;

• reduce the risk of recurrence;

• potentially reduce the need for surgery in some patients.

This is currently a Phase 1 clinical trial, so the main goals are to assess safety, tolerability and early signs of effectiveness.


Why does this matter? If the approach proves safe and effective, it could provide a new treatment option for patients with rectal cancer that usually responds poorly to immunotherapy.

Source: PharmaTimes / Transgene

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