METHODS AND MEANS FOR THE PREVENTION AND/OR TREATMENT OF HEMOPHILIC ARTHROPATHY IN HEMOPHILIA
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.
1 . A gene therapy vehicle for use in preventing, arresting and/or treating hemophilic arthropathy of a joint in a patient having hemophilia B, wherein the gene therapy vehicle comprises a nucleic acid that encodes a coagulation factor having Factor IX activity.
2 . The gene therapy vehicle for use according to claim 1 , wherein the patient having hemophilia B has level of Factor IX activity of less than 1 IU/dL.
3 . The gene therapy vehicle for use according to claim 1 , wherein the vehicle is a viral vector.
4 . The gene therapy vehicle for use according to claim 3 , wherein the AAV based particle is an AAV5 particle.
5 . The gene therapy vehicle for use according to claim 1 , wherein the nucleic acid comprising a wildtype Factor IX, or a hyperactive variant of Factor IX.
6 . The gene therapy vehicle for use according to claim 1 , wherein the nucleic acid further comprises a promoter.
7 . The gene therapy vehicle for use according to claim 1 , wherein the gene therapy vehicle is administered at a dose of from 8×10 10 vg/kg to 2×10 13 vg/kg.
8 . The gene therapy vehicle for use according to claim 1 , wherein the gene therapy vehicle is administered in a single dose.
9 . The gene therapy vehicle for use according to claim 1 , wherein the joint is selected from the group consisting of one or both elbows, one or both knees, one or both ankles, and any combination thereof.
10 . The gene therapy vehicle for use according to claim 1 , wherein the hemophilic arthropathy comprises joint bleeding and/or synovitis.
11 . The gene therapy vehicle for use according to claim 1 , wherein the patient is a human.
12 . A pharmaceutical composition for use in preventing, arresting and/or treating hemophilic arthropathy of a joint in a patient having hemophilia B, wherein the pharmaceutical composition comprises a gene therapy vehicle for use according to claim 1 .
13 . The pharmaceutical composition for use according to claim 12 , wherein the pharmaceutical composition is in a form suitable for intravenous infusion.
14 . The pharmaceutical composition for use according to claim 13 , wherein the pharmaceutical composition is a liquid, or a lyophilized solid.
15 . A method of preventing, arresting and/or treating hemophilic arthropathy of a joint in a patient having hemophilia B, comprising administering to the patient an effective amount of a gene therapy vehicle for use according to claim 1 or a pharmaceutical composition for use according to claim 12 .
16 . The method of claim 15 , wherein the gene therapy vehicle for use, or the pharmaceutical composition for use, is administered via intravenous infusion.
17 . The method of claim 15 , wherein the patient has a pre-treatment baseline HJHS 2.1 score and a post-treatment HJHS 2.1 score, the post-treatment HJHS 2.1 score being no higher than the pre-treatment baseline HJHS 2.1 score.
18 . The method of claim 17 , wherein the post-treatment HJHS 2.1 score is lower than the pre-treatment baseline HJHS 2.1 score.
19 . The method of claim 17 , wherein the post-treatment HJHS 2.1 score is taken at least 1 year after the administration of the gene therapy vehicle for use or the pharmaceutical composition for use.