Claims for Patent: 10,543,204

Summary for Patent: 10,543,204

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 K. (Frederiksberg C, DK), Jaattela; Marja H. (Kobenhavn O, DK)
Assignee:	Orphazyme A/S (Copenhagen N, DK)
Application Number:	15/854,352
Patent Claims:	1. A method for treatment of mucopolysaccaridoses, glycoproteinoses, and mucolipidoses, 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, 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 bioactive agent is administered in combination with at least one other treatment modality. 8. The method according to claim 7, wherein said at least one 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. 9. The method according to claim 1, wherein said bioactive agent is administered in combination with enzyme replacement therapy (ERT). 10. The method according to claim 9, wherein said enzyme replacement therapy is selected from the group consisting of imiglucerase, Miglustat, agalsidase beta and agalsidase alpha. 11. The method according to claim 8, wherein said functional fragment or variant of Hsp70 comprises all or part of the ATPase domain of Hsp70. 12. The method according to claim 8, wherein said Hsp70, or a functional fragment or variant thereof, is recombinant Hsp70 (rHsp70). 13. The method according to claim 1, wherein said mucopolysaccharidoses comprise a disorder selected from the group consisting of Hunter syndrome and Hurler disease. 14. The method according to claim 1, wherein said hydroxylamine derivative is BRX-220.

Details for Patent 10,543,204

Applicant	Tradename	Biologic Ingredient	Dosage Form	BLA	Approval Date	Patent No.	Expiredate
Genzyme Corporation	CEREZYME	imiglucerase	For Injection	020367	May 23, 1994		2037-12-26
Genzyme Corporation	CEREZYME	imiglucerase	For Injection	020367	September 22, 1999		2037-12-26
Genzyme Corporation	FABRAZYME	agalsidase beta	For Injection	103979	April 24, 2003		2037-12-26
Genzyme Corporation	FABRAZYME	agalsidase beta	For Injection	103979	October 10, 2003		2037-12-26