Compositions and methods for modulating the function of factor VIII
FVIII variants with mutations at positions 336 and/or 562 provide enhanced resistance to APC cleavage, addressing the limitations of current treatments for hemophilia A by significantly improving hemostatic efficacy.
JP7864071B2Active Publication Date: 2026-05-22THE CHILDRENS HOSPITAL OF PHILADELPHIA
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Patent Information
- Authority / Receiving Office
- JP · JP
- Patent Type
- Patents
- Current Assignee / Owner
- THE CHILDRENS HOSPITAL OF PHILADELPHIA
- Filing Date
- 2020-12-07
- Publication Date
- 2026-05-22
AI Technical Summary
Technical Problem
Current treatments for hemophilia A, such as FVIII replacement therapy and gene therapy using AAV vectors, are expensive and limited by safety concerns, necessitating the development of FVIII molecules with improved biological properties to enhance hemostasis.
Method used
Development of FVIII variants with mutations at positions 336 and/or 562, specifically replacing Arg with Gln, to increase resistance to APC cleavage and enhance hemostatic efficacy.
Benefits of technology
The FVIII variants demonstrate superior hemostatic efficacy compared to wild-type FVIII, exhibiting up to five times better in vivo function in modulating the coagulation cascade and thrombus formation.
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Abstract
Factor VIII variants and methods of use thereof are disclosed. In accordance with the present invention, compositions and methods are provided for modulating hemostasis in patients in need thereof. More specifically, factor VIII (FVIII) variants that modulate (e.g., increase) hemostasis are provided. In certain embodiments, the FVIII variants contain mutations at least at positions 336 and / or 562.
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