THERAPEUTIC REGIMEN FOR THE TREATMENT OF FABRY USING STABILIZED ALPHA-GALACTOSIDASE

    公开(公告)号:US20250134968A1

    公开(公告)日:2025-05-01

    申请号:US19015746

    申请日:2025-01-10

    Applicant: Protalix Ltd.

    Abstract: Methods of treating Fabry disease via administration of stabilized plant recombinant human alpha galactosidase protein comprising at least two alpha-galactosidase monomers being covalently linked to one another via a linking moiety, and unit dosages of protein are disclosed herein. The disclosed protocols are safe, have greater than 2 week intervals between administrations and exhibit important improvement in patient's disease parameters, in terms of reduced Gb3 accumulation, pain and GI parameters, kidney and cardiac stabilization in the clinical setting.

    MODIFIED URICASE AND USES THEREOF
    2.
    发明公开

    公开(公告)号:US20240002814A1

    公开(公告)日:2024-01-04

    申请号:US18035149

    申请日:2021-11-03

    Applicant: Protalix Ltd.

    CPC classification number: C12N9/0048 A61K38/44 C12Y107/03003

    Abstract: A modified uricase is described herein, as well as a method of reducing a level of uric acid by contacting a medium with the modified uricase. The modified uricase comprises a uricase polypeptide crosslinked by at least one bifunctional linking moiety that comprises a poly(alkylene glycol) moiety. A molecular weight of the bifunctional linking moiety is from about 1.5 kDa to about 4 kDa, and/or the modified uricase comprises a plurality of polypeptides having the amino acid sequence SEQ ID NO: 2. Further described is a polypeptide having the amino acid sequence SEQ ID NO: 2. A process of preparing the modified uricase is also described, comprising contacting the polypeptide with a crosslinking agent that comprises a poly(alkylene glycol) moiety and at least two aldehyde groups, to obtain a conjugate; and contacting the conjugate with a reducing agent.

    THERAPEUTIC REGIMEN FOR THE TREATMENT OF FABRY USING STABILIZED ALPHA-GALACTOSIDASE

    公开(公告)号:US20200155654A1

    公开(公告)日:2020-05-21

    申请号:US16476084

    申请日:2018-01-05

    Applicant: Protalix Ltd.

    Abstract: Methods of treating Fabry disease via administration of stabilized plant recombinant human alpha galactosidase protein comprising at least two alpha-galactosidase monomers being covalently linked to one another via a linking moiety, and unit dosages of protein are disclosed herein. The disclosed protocols are safe, have greater than 2 week intervals between administrations and exhibit important improvement in patient's disease parameters, in terms of reduced Gb3 accumulation, pain and GI parameters, kidney and cardiac stabilization in the clinical setting.

    Plant cell culture expressing human lysosomal proteins and uses thereof
    10.
    发明授权
    Plant cell culture expressing human lysosomal proteins and uses thereof 有权
    表达人溶酶体蛋白质的植物细胞培养物及其用途

    公开(公告)号:US09220737B2

    公开(公告)日:2015-12-29

    申请号:US14292966

    申请日:2014-06-02

    Applicant: Protalix Ltd.

    Abstract: A device, system and method for producing glycosylated proteins in plant culture, particularly proteins having a high mannose glycosylation, while targeting such proteins with an ER signal and/or by-passing the Golgi. The invention further relates to vectors and methods for expression and production of enzymatically active high mannose lysosomal enzymes using transgenic plant root, particularly carrot cells. More particularly, the invention relates to host cells, particularly transgenic suspended carrot cells, vectors and methods for high yield expression and production of biologically active high mannose Glucocerebrosidase (GCD). The invention further provides for compositions and methods for the treatment of lysosomal storage diseases.

    Abstract translation: 用于在植物培养物中生产糖基化蛋白质的装置,系统和方法,特别是具有高甘露糖糖基化的蛋白质,同时用ER信号靶向这种蛋白质和/或旁路高尔基体。 本发明进一步涉及使用转基因植物根特别是胡萝卜细胞表达和产生酶活性高甘露糖溶酶体酶的载体和方法。 更具体地,本发明涉及宿主细胞,特别是转基因悬浮的胡萝卜细胞,用于高产量表达和生产生物活性高甘露糖葡糖脑苷脂酶(GCD)的载体和方法。 本发明还提供了用于治疗溶酶体贮积病的组合物和方法。

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