WO2025219952 - 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/2025/219952
Publication Date
23.10.2025
International Application No.
PCT/IB2025/054079
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 PROCÉDÉS DE RÉDUCTION DE TAUX DE BIOMARQUEURS LIÉS À UNE MALADIE CHEZ DES PATIENTS ATTEINTS DE TROUBLES DU STOCKAGE DES GANGLIOSIDES
Applicants **
GENZYME CORPORATION
Inventors
BATSU, Isabela
FISCHER, Tanya
KISSELL, Julie
MININI, Pascal
ZHENG, Riliang
Priority Data
63/636,318
19.04.2024
US
Application details
| Total Number of Claims/PCT | * |
| Number of Independent Claims | * |
| Number of Priorities | * |
| Number of Multi-Dependent Claims | * |
| Number of Drawings | * |
| Pages for Publication | * |
| Number of Pages with Drawings | * |
| Pages of Specification | * |
| * | |
| Number of Office Actions | * |
| * | |
International Searching Authority |
EPO
* |
| Recordal of a Change of the Applicant's Name/Address |
Change of Applicant's Name and Address
* |
| Type of Assignment |
The Standard Agent's Assignment
* |
| Applicant's Legal Status |
Legal Entity
* |
| * | |
| * | |
| * | |
| * | |
| * | |
| Entry into National Phase under |
Chapter I
* |
| Patent Delivery |
Send the Letters Patent by Courier
* |
| Translation |
|
* 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 | 2309 | |
| EPO | Filing, Examination, Granting | 12434 | |
| Japan | Filing, Examination, Granting | 2399 | |
| South Korea | Filing, Examination, Granting | 2573 | |
| USA | Filing, Examination, Granting | 5340 |
Total:
25,055
Contact Us
Abstract[English]
Provided herein is a method of stabilizing the 9-HPT, 25-FWT, and/or FARS-neuro test score (a measure of neurologic impairment) in a human subject suffering from GM1 gangliosidosis, juvenile GM2 gangliosidosis, or sialidosis. The method comprises administering to the subject an effective amount of venglustat or a pharmaceutically acceptable salt thereof.[French]
Sont proposés dans la description un procédé de stabilisation du score de test 9-HPT, 25-FWT et/ou FARS-neuro (une mesure de déficience neurologique) chez un sujet humain souffrant de gangliosidose à GM1, de gangliosidose à GM2 juvénile ou de sialidose. Le procédé consiste à administrer au sujet une quantité efficace de venglustat ou d'un sel pharmaceutiquement acceptable de celui-ci.