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US 12,544,459 B2Granted
Methods and means for the prevention and/or treatment of hemophilic arthropathy in hemophilia
AssigneeuniQure biopharma B.V.
InventorEllen Broug-Holub
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Abstract
The invention relates to a gene therapy vehicle for use in preventing, arresting and/or treating hemophilic arthropathy of a joint in a patient having hemophilia B, where the gene therapy vehicle comprises a nucleic acid that encodes a coagulation factor having Factor IX activity.
Claims17
A method of preventing, arresting and/or treating an effect of hemophilic arthropathy of a joint in a patient having hemophilia B, comprising:
- measuring a pre-treatment baseline HJHS 2.1 score;
- administering to the patient an effective amount of a gene therapy vehicle;
- measuring a post-treatment HJHS 2.1 score at least 1 year after the administering;
- and
- comparing the pre-treatment baseline HJHS 2.1 score to the post-treatment baseline HJHS 2.1 score;
- wherein the gene therapy vehicle comprises a promoter and a nucleic acid that encodes a coagulation Factor IX or a hyperactive variant thereof;
- wherein the effect of hemophilic arthropathy is at least one of pain, loss of range of motion, loss of activities, restrictions in participation in society, joint bleeds, synovitis, chronic arthropathy, or combinations thereof;
- and
- wherein the post-treatment HJHS 2.1 score is no higher than pre-treatment baseline HJHS 2.1 score.
6 more elements
Dependencies are inferred from the claim text.