Trabedersen is an antisense molecule consisting of 18 DNA oligonucleotides which is specifically designed to target the human transforming growth factor beta 2 (TGF-β2) messenger RNA. This results in the inhibition TGF-β2 protein synthesis.
TGF-β2 is overexpressed in many highly aggressive tumors. It is a key cancerogenic protein which is decisive for explosive tumor proliferation and aggressive metastasizing of tumors.
A main characteristic of TGF-β2 is its ability to suppress the body’s own immune system. In simple terms, TGF-β2 forms a protective shield around the tumor, not allowing the body’s immune cells to recognize the tumor cells and inhibiting their elimination.
Trabedersen is believed to reverse the immunosuppressive effects of TGF-β2, rendering the tumor visible to the patient’s immune system and resulting in priming and specific activation of the patient’s anti-tumor immune response.
Trabedersen has been evaluated extensively in several preclinical and clinical studies across selected oncological indications. The compound has shown encouraging early signs of therapeutic activity in cancer patients.
Trabedersen has been granted orphan designation for three tumor indications: high-grade glioma (US, EU), pancreatic cancer (US, EU) and malignant melanoma (US).