US20250281533
2025-09-11
Human necessities
A61K35/17
The invention focuses on a polypeptide that binds specifically to mucin 1, a glycoprotein overexpressed in various cancers. It includes an isolated polynucleotide encoding this polypeptide, vectors carrying the polynucleotide, and cells containing the vector. Additionally, it involves a chimeric antigen receptor (CAR) incorporating the polypeptide, and immune cells expressing the CAR. These components are utilized in compositions and methods for cancer treatment.
Mucin 1 is a glycoprotein found in epithelial cells across multiple organs. It consists of two subunits, MUC1-N and MUC1-C, with MUC1-N containing repeated sequences that interact with MUC1-C. Under normal conditions, mucin 1 is expressed at low levels and localized to specific cell areas. In contrast, it is overexpressed and abnormally distributed across cancer cell surfaces, making it a suitable target for cancer detection and therapy.
Existing treatments using anti-MUC1 antibodies face limitations such as specificity to certain cancer antigens, short duration of effects, and resistance development. The invention addresses these challenges by providing a polypeptide with strong binding affinity to mucin 1, potentially enhancing the immune system's ability to combat various cancers effectively.
The invention encompasses several embodiments: an isolated polynucleotide encoding the mucin 1-binding polypeptide; vectors containing this polynucleotide; cells with the vector; a CAR including the polypeptide; and immune cells expressing the CAR. These components form compositions for cancer treatment, offering potential for improved therapeutic outcomes.
Figures included demonstrate the effectiveness of CAR-T cells developed from this invention in secreting cytokines and exhibiting cytotoxicity against MUC1-positive tumor cells. The results show significant tumor growth inhibition in animal models injected with these CAR-T cells, highlighting their potential as a robust cancer treatment strategy.