WO2024105638 - RECOMBINANT AAV VECTORS AND METHODS FOR TREATMENT OF HUNTER SYNDROME

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Publication Number WO/2024/105638
Publication Date 23.05.2024
International Application No. PCT/IB2023/061655
International Filing Date 17.11.2023
Title [English] RECOMBINANT AAV VECTORS AND METHODS FOR TREATMENT OF HUNTER SYNDROME [French] VECTEURS AAV RECOMBINANTS ET MÉTHODES DE TRAITEMENT DE LA MALADIE DE HUNTER
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 6512241, JP TAKAHASHI, Kenichi c/o JCR Pharmaceuticals Co., Ltd. 2-2-9, Murotani, Nishi-ku Kobe, Hyogo 6512241, JP TAKAGI, Haruna c/o JCR Pharmaceuticals Co., Ltd. 2-2-9, Murotani, Nishi-ku Kobe, Hyogo 6512241, JP
Priority Data 63/426,576  18.11.2022  US 63/502,461  16.05.2023  US
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Abstract [English] The present invention discloses gene therapy vectors for treatment of Hunter Syndrome. The gene therapy vector expresses a fusion protein comprising at least one anti-hTfR1 VHH antibody fused to a human iduronate -2-sulfatase. [French] La présente invention concerne des vecteurs de thérapie génique pour le traitement de la maladie de Hunter. Le vecteur de thérapie génique exprime une protéine de fusion comprenant au moins un anticorps anti-hTfR1 VHH fusionné à une iduronate-2-sulfatase humaine.