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Last Updated: March 28, 2024

Claims for Patent: 9,884,058


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Summary for Patent: 9,884,058
Title:Use of Hsp70 as a regulator of enzymatic activity
Abstract: The present invention concerns a method for modulating the enzymatic activity of an enzyme, wherein said enzyme interacts with BMP, said method comprising the step of administering or inducing Hsp70, or a functional fragment or variant thereof, in a form suitable for allowing interaction between BMP and Hsp70, or said functional fragment or variant thereof, and thereby modulating the enzymatic activity of an enzyme interacting with BMP.
Inventor(s): Jensen; Thomas Kirkegaard (Frederiksberg C, DK), Jaattela; Marja H. (Kobenhavn O, DK)
Assignee: Orphazyme ApS (Kobenhavn N, DK)
Application Number:15/048,483
Patent Claims:1. A method for treatment of a lipid storage disorder (lipidosis) comprising administering to an individual in need thereof a bioactive agent capable of increasing the intracellular concentration of Hsp70 by amplifying Hsp70 gene expression, wherein said bioactive agent is a hydroxylamine derivative.

2. The method according to claim 1, wherein said treatment is curative or ameliorating.

3. The method according to claim 1, wherein said hydroxylamine derivative is bimoclomol, or a structural analogue thereof.

4. The method according to claim 1, wherein said hydroxylamine derivative is selected from the group consisting of bimoclomol, arimoclomol, BRX-220, BRX-345 and BGP-15.

5. The method according to claim 1, wherein said hydroxylamine derivative is arimoclomol.

6. The method according to claim 1, wherein said hydroxylamine derivative is BRX-345.

7. The method according to claim 1, wherein said lipid storage disorder is a sphingolipidosis.

8. The method according to claim 1, wherein said bioactive agent is administered in combination with at least one other treatment modality.

9. The method according to claim 8, wherein said at least one other treatment modality is individually selected from the group consisting of enzyme replacement therapy (ERT), pain relievers, corticosteroids, a transplantation, substrate reduction therapy, Hsp70 protein, a functional fragment of Hsp70 having at least 95% sequence identity to Hsp70, and a functional variant of Hsp70 having at least 95% sequence identity to Hsp70.

10. The method according to claim 1, wherein said bioactive agent is administered in combination with enzyme replacement therapy (ERT).

11. The method according to claim 10, wherein said enzyme replacement therapy is selected from the group consisting of imiglucerase, Miglustat, agalsidase beta and agalsidase alpha.

12. The method according to claim 9, wherein said functional fragment or variant of Hsp70 comprises all or part of the ATPase domain of Hsp70.

13. The method according to claim 9, wherein said Hsp70, or a functional fragment or variant thereof, is recombinant Hsp70 (rHsp70).

Details for Patent 9,884,058

Applicant Tradename Biologic Ingredient Dosage Form BLA Approval Date Patent No. Expiredate
Genzyme Corporation CEREZYME imiglucerase For Injection 020367 05/23/1994 ⤷  Try a Trial 2028-06-26
Genzyme Corporation CEREZYME imiglucerase For Injection 020367 09/22/1999 ⤷  Try a Trial 2028-06-26
Genzyme Corporation FABRAZYME agalsidase beta For Injection 103979 04/24/2003 ⤷  Try a Trial 2028-06-26
Genzyme Corporation FABRAZYME agalsidase beta For Injection 103979 10/10/2003 ⤷  Try a Trial 2028-06-26
>Applicant >Tradename >Biologic Ingredient >Dosage Form >BLA >Approval Date >Patent No. >Expiredate

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