Claims for Patent: 10,675,328

Summary for Patent: 10,675,328

Title:	Compositions and methods for treatment of Gcase related disease states
Abstract:	Disclosed are compositions and methods of treating a neurodegenerative disease in an individual. The methods disclose administration of an Integrin .alpha.4.beta.1, Very Late Antigen-4 positive neural precursor cell (\"VLA4+ NPC\") transfected with a lentivirus overexpressing wild type GCase to an individual having a neurodegenerative disorder. The neurodegenerative disease may include lipid storage diseases, for example Gaucher disease, Parkinson\'s disease (PD), Dementia with Lewy bodies.
Inventor(s):	Sun; Ying (Mason, OH)
Assignee:	Children\'s Hospital Medical Center (Cincinnati, OH)
Application Number:	15/896,423
Patent Claims:	1. A method of treating Gaucher disease in an individual in need thereof, comprising administering intravenously to said individual a composition comprising a Very Late Antigen-4 positive neural precursor cell ("VLA4+NPC") having a glucocerebrosidase (GCase) gene cloned into a lentiviral expression vector under the control of a promoter; wherein said GCase gene encodes for a functional GCase enzyme; wherein said VLA4+NPC cells are derived from an induced pluripotent stem cell (iPSC); and wherein said VLA4+NPC cells comprise at least 90% of total neural precursor cells (NPC) in said composition. 2. The method of claim 1, wherein said Gaucher disease is in an individual having a mutation in glucocerebrosidase gene GBA1. 3. The method of claim 1, wherein said Gaucher disease is type II neuronopathic Gaucher disease nGD. 4. The method of claim 1, wherein said Gaucher disease is type III neuronopathic Gaucher disease nGD. 5. The method of claim 1 wherein said lentivirus comprises a human elongation factor 1 alpha ("EF1.alpha.") promoter or an Ubiquitin C promoter (UbC). 6. The method of claim 1 wherein said VLA4+NPC is co-administered with a chaperone molecule. 7. The method of claim 1 wherein said VLA4+NPC is co-administered with a chaperone molecule selected from Dantrolene, Ambroxol, or a combination thereof. 8. The method of claim 1 wherein said VLA4+NPC is delivered to said individual until one or more parameters selected from neurological pathology, survival, brain inflammation, brain neurodegeneration, GCase activity, GCase substrate level, mitochondrial function, neurotropic factor expression, or combinations thereof, is improved. 9. The method of claim 1, wherein said VLA4+NPC is co-administered with an agent selected from imiglucerase, velaglucerase alfa, taliglucerase alpha, eligustat, miglustat, ibiglustat, or combinations thereof.

Details for Patent 10,675,328

Applicant	Tradename	Biologic Ingredient	Dosage Form	BLA	Approval Date	Patent No.	Expiredate
Genzyme Corporation	CEREZYME	imiglucerase	For Injection	020367	May 23, 1994	10,675,328	2037-02-14
Genzyme Corporation	CEREZYME	imiglucerase	For Injection	020367	September 22, 1999	10,675,328	2037-02-14
Takeda Pharmaceuticals U.s.a., Inc.	VPRIV	velaglucerase alfa	For Injection	022575	February 26, 2010	10,675,328	2037-02-14