WO2026028089 - COMPOSITIONS AND METHODS FOR THE TREATMENT OF LYSOSOMAL ACID LIPASE DEFICIENCY (LAL-D)
National phase entry is expected:
Publication Number
WO/2026/028089
Publication Date
05.02.2026
International Application No.
PCT/IB2025/057651
International Filing Date
28.07.2025
Title **
[English]
COMPOSITIONS AND METHODS FOR THE TREATMENT OF LYSOSOMAL ACID LIPASE DEFICIENCY (LAL-D)
[French]
COMPOSITIONS ET PROCÉDÉS POUR LE TRAITEMENT D'UN DÉFICIT EN LIPASE D'ACIDE LYSOSOMALE (LAL-D)
Applicants **
NOVARTIS AG
Lichtstrasse 35
4056 Basel, CH
Inventors
CHEN, Feng Graf
NOVARTIS INSTITUTES FOR BIOMEDICAL RESEARCH, INC.
250 MASSACHUSETTS AVENUE
CAMBRIDGE, Massachusetts 02139, US
FLYNN, Jonathan Robert
NOVARTIS INSTITUTES FOR BIOMEDICAL RESEARCH, INC.
250 MASSACHUSETTS AVENUE
CAMBRIDGE, Massachusetts 02139, US
LIN, Amy Hsiu-Ti
NOVARTIS INSTITUTES FOR BIOMEDICAL RESEARCH, INC.
250 MASSACHUSETTS AVENUE
CAMBRIDGE, Massachusetts 02139, US
MAO, Xiaochong
NOVARTIS INSTITUTES FOR BIOMEDICAL RESEARCH, INC.
250 MASSACHUSETTS AVENUE
CAMBRIDGE, Massachusetts 02139, US
WEINMANN, Jonas
NOVARTIS PHARMA AG
Lichtstrasse 35
4056 Basel, CH
Priority Data
63/676,736
29.07.2024
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 | * |
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International Searching Authority |
EPO
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| Applicant's Legal Status |
Legal Entity
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| * | |
| * | |
| * | |
| * | |
| Entry into National Phase under |
Chapter I
* |
| Translation |
|
Recalculate
* The data is based on automatic recognition. Please verify and amend if necessary.
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Quotation for National Phase entry
| Country | Stages | Total | |
|---|---|---|---|
| China | Filing | 5534 | |
| EPO | Filing, Examination | 31798 | |
| Japan | Filing | 589 | |
| South Korea | Filing | 575 | |
| USA | Filing, Examination | 18010 |
Total: 56506 USD
Abstract[English]
The present disclosure relates to gene therapy compositions and methods for the preparation and use thereof, for the treatment of Lysosomal Acid Lipase Deficiency (LAL-D) disorders, such as Wolman Disease and cholesterol ester storage disease (CESD).[French]
La présente divulgation concerne des compositions de thérapie génique et leurs procédés de préparation et d'utilisation, pour le traitement de troubles liés au déficit en lipase acide lysosomale (LAL-D), tels que la maladie de Wolman et la maladie de stockage des esters du cholestérol (CESD).