WO2024241176 - RECOMBINANT AAV VECTORS AND METHODS FOR TREATMENT OF DISEASES WITH CENTRAL NERVOUS SYSTEM DISORDERS
National phase entry is expected:
Publication Number
WO/2024/241176
Publication Date
28.11.2024
International Application No.
PCT/IB2024/054820
International Filing Date
17.05.2024
Title **
[English]
RECOMBINANT AAV VECTORS AND METHODS FOR TREATMENT OF DISEASES WITH CENTRAL NERVOUS SYSTEM DISORDERS
[French]
VECTEURS AAV RECOMBINÉS ET PROCÉDÉS POUR LE TRAITEMENT DE MALADIES AVEC TROUBLES DU SYSTÈME NERVEUX CENTRAL
Applicants **
JCR PHARMACEUTICALS CO., LTD.
3-19 Kasuga-cho
Ashiya, Hyogo 659-0021, JP
Inventors
JACOBO, Sarah Melissa
c/o Takeda Development Center Americas, Inc.
35 Landsdowne Street
Cambridge, Massachusetts 02139, US
CHOI, Vivian
c/o Takeda Development Center Americas, Inc.
35 Landsdowne Street
Cambridge, Massachusetts 02139, US
RUANGSIRILUK, Wanida
c/o Takeda Development Center Americas, Inc.
35 Landsdowne Street
Cambridge, Massachusetts 02139, US
ISLAM, Rizwana
c/o Takeda Development Center Americas, Inc.
35 Landsdowne Street
Cambridge, Massachusetts 02139, US
ONOUCHI, Takashi
c/o JCR Pharmaceuticals Co., Ltd.
2-2-9, Murotani, Nishi-ku
Kobe, Hyogo 651-2241, JP
TAKAHASHI, Kenichi
c/o JCR Pharmaceuticals Co., Ltd.
2-2-9, Murotani, Nishi-ku
Kobe, Hyogo 651-2241, JP
TAKAGI, Haruna
c/o JCR Pharmaceuticals Co., Ltd.
2-2-9, Murotani, Nishi-ku
Kobe, Hyogo 651-2241, JP
Priority Data
63/468,775
24.05.2023
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
* |
| 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.
** 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 | 3124 | |
| EPO | Filing, Examination | 36494 | |
| Japan | Filing | 593 | |
| South Korea | Filing | 575 | |
| USA | Filing, Examination | 16835 |
Total: 57621 USD
The term for entry into the National Phase has expired. This quotation is for informational purposes only
Abstract[English]
The present invention provides gene therapy transgenes encoding a fusion protein comprising at least one anti-hTfR1 VHH and a human SGSH protein. Provided herein includes a recombinant AAV (rAAV) comprising a AAV9 capsid and a transgene encoding a fusion protein comprising at least one anti-hTfR1 VHH and a human SGSH protein. The present invention also provides methods for treating MPS IIIA in a patient using such rAAV vectors and compositions thereof.[French]
La présente invention concerne des transgènes de thérapie génique codant pour une protéine de fusion comprenant au moins un VHH anti-hTfR1 et une protéine SGSH humaine. La présente invention comprend un AAV recombiné (rAAV) comprenant une capside AAV9 et un transgène codant pour une protéine de fusion comprenant au moins un VHH anti-hTfR1 et une protéine SGSH humaine. La présente invention concerne également des procédés de traitement de la MPS IIIA chez un patient par l'utilisation de tels vecteurs rAAV et de compositions associées.