20240010707. De-immunized Factor VIII Molecule and Pharmaceutical Compositions Comprising the Same simplified abstract (Biotest AG)
De-immunized Factor VIII Molecule and Pharmaceutical Compositions Comprising the Same
Organization Name
Inventor(s)
Karina Winterling of Darmstadt (DE)
Steffen Kistner of Frankfurt/Main (DE)
Annie De Groot of Providence RI (US)
William Martin of Providence RI (US)
Christopher Ungerer of Langen (DE)
De-immunized Factor VIII Molecule and Pharmaceutical Compositions Comprising the Same - A simplified explanation of the abstract
This abstract first appeared for US patent application 20240010707 titled 'De-immunized Factor VIII Molecule and Pharmaceutical Compositions Comprising the Same
Simplified Explanation
The present invention is related to therapeutic proteins, specifically recombinant coagulation factors. It introduces a recombinant factor VIII (FVIII) protein that has specific point mutations at defined positions, which reduce its immunogenicity while retaining its coagulant activity. The invention also includes nucleic acids encoding this de-immunized protein, cell lines, methods of recombinant preparation, and pharmaceutical compositions comprising the recombinant FVIII. This technology is advantageous for treating patients with hemophilia A, especially those who have not yet been treated with an FVIII product. It can also be a safe alternative for previously treated patients and those who have developed an immune response to FVIII. Additionally, the invention provides an assay for determining the immunogenicity of a protein.
- Recombinant factor VIII (FVIII) protein with specific point mutations to reduce immunogenicity while maintaining coagulant activity
- Nucleic acids encoding the de-immunized FVIII protein
- Cell lines and methods for recombinant preparation
- Pharmaceutical compositions containing the recombinant FVIII
- Advantages for treating hemophilia A patients, especially those who have not been previously treated with FVIII
- Safe alternative for previously treated patients and those with immune responses to FVIII
- Assay for determining the immunogenicity of a protein
Potential Applications
This technology can be applied in the treatment of patients with hemophilia A, particularly those who have not yet received FVIII treatment. It can also be used as a safe alternative for previously treated patients and those who have developed an immune response to FVIII. Furthermore, this technology can be utilized in immune-tolerance-induction therapy (ITI/ITT) or rescue ITI for patients with hemophilia A.
Problems Solved
The technology addresses the issue of immunogenicity associated with FVIII protein used in the treatment of hemophilia A. By introducing specific point mutations, the immunogenicity of the FVIII protein is reduced while still maintaining its coagulant activity. This solves the problem of immune responses and potential complications in patients receiving FVIII treatment.
Benefits
The benefits of this technology include:
- Reduced immunogenicity of the FVIII protein
- Retention of coagulant activity
- Safe and effective treatment option for patients with hemophilia A
- Potential use in immune-tolerance-induction therapy and rescue ITI
- Improved patient outcomes and quality of life
Original Abstract Submitted
the present invention relates to the field of therapeutic proteins, in particular, to recombinant coagulation factors. it provides a recombinant factor viii (fviii) protein comprising specific point mutations at defined positions, which serve to reduce the immunogenicity of said fviii protein, wherein the factor viii protein substantially retains its coagulant activity. it further provides nucleic acids encoding said de-immunized protein, cell lines and methods of recombinant preparation as well as pharmaceutical compositions comprising the recombinant fviii of the invention, which are advantageous for use in treatment of patients with hemophilia a, particularly those who have not yet been treated with a fviii product. additionally, it can be a safe alternative for previously treated patients and even for patients who have developed an immune-response to fviii, e.g., for immune-tolerance-induction therapy (iti/itt) or rescue iti. the invention also provides an assay for determining immunogenicity of a protein.
