WO2025219951 - VENGLUSTAT FOR USE IN METHODS FOR REDUCING DISEASE RELATED BIOMARKER LEVELS IN PATIENTS WITH GANGLIOSIDE STORAGE DISORDERS
National phase entry is expected:
Publication Number
WO/2025/219951
Publication Date
23.10.2025
International Application No.
PCT/IB2025/054078
International Filing Date
17.04.2025
Title **
[English]
VENGLUSTAT FOR USE IN METHODS FOR REDUCING DISEASE RELATED BIOMARKER LEVELS IN PATIENTS WITH GANGLIOSIDE STORAGE DISORDERS
[French]
VENGLUSTAT DESTINÉ À ÊTRE UTILISÉ DANS DES MÉTHODES DE RÉDUCTION DES TAUX DE BIOMARQUEURS LIÉS À UNE MALADIE CHEZ DES PATIENTS ATTEINTS DE MALADIES DE SURCHARGE DES GANGLIOSIDES
Applicants **
GENZYME CORPORATION
Inventors
BATSU, Isabela
LEVIT, Mikhail Nakhimovitch
SARDI, Sergio Pablo
ZHENG, Riliang
Priority Data
63/636,315
19.04.2024
US
Application details
| Total Number of Claims/PCT | * |
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International Searching Authority |
EPO
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| Recordal of a Change of the Applicant's Name/Address |
Change of Applicant's Name and Address
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| Type of Assignment |
The Standard Agent's Assignment
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| Applicant's Legal Status |
Legal Entity
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| Entry into National Phase under |
Chapter I
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| Patent Delivery |
Send the Letters Patent by Courier
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| Translation |
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* The data is based on automatic recognition. Please verify and amend if necessary.
** IP-Coster compiles data from publicly available sources. If this data includes your personal information, you can contact us to request its removal.
Quotation for National Phase entry
| Country | Stages | Total | |
|---|---|---|---|
| China | Filing, Examination, Granting | 2506 | |
| EPO | Filing, Examination, Granting | 12828 | |
| Japan | Filing, Examination, Granting | 2389 | |
| South Korea | Filing, Examination, Granting | 2474 | |
| USA | Filing, Examination, Granting | 6340 |
Total:
26,537
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Abstract[English]
Provided herein is a method in which venglustat is administered to a patient with a ganglioside storage disorder, e.g. a GM2 gangliosidosis such as Sandhoff disease or Tay-Sachs disease, GM1 gangliosidosis, or sialidosis. The method is designed to reduce the levels of disease related biomarkers in the patient.[French]
L'invention propose une méthode dans laquelle du venglustat est administré à un patient avec une maladie de surcharge des gangliosides, par exemple une gangliosidose à GM2 telle que la maladie de Sandhoff ou la maladie de Tay-Sachs, la gangliosidose à GM1 ou la sialidose. La méthode est conçue pour réduire les niveaux de biomarqueurs associés à une maladie chez le patient.