Delivery System and Conjugates For Compound Delivery Via Naturally Occurring Intracellular Transport Routes
    1.
    发明申请
    Delivery System and Conjugates For Compound Delivery Via Naturally Occurring Intracellular Transport Routes 审中-公开
    通过自然发生的细胞内运输途径进行复合递送的递送系统和结合体

    公开(公告)号:US20120258104A1

    公开(公告)日:2012-10-11

    申请号:US13386347

    申请日:2010-07-22

    摘要: The present invention relates to a delivery system that comprises a conjugate that facilitates the delivery of a compound such as a biologically-active macromolecule, a nucleic acid or a peptide in particular, into a cell. The present invention also relates to said conjugate for delivery of a compound, such as a biologically-active macromolecule, a nucleic acid or a peptide, into a cell. The present invention further relates to a pharmaceutical composition comprising said conjugate and to its use. The present invention also relates to a method of delivering a compound to a cell or an organism, preferably a patient. The conjugates comprise: (a) at least one module that mediates cell targeting and facilitates cellular uptake, (b) at least one module that facilitates transport to the endoplasmic reticulum (ER), (c) at least one module that mediates translocation from the ER to the cytosol, and (d) at least one compound to be delivered wherein the modules (a) to (c) and the compound (d) are linked to each other in any arrangement.

    摘要翻译: 本发明涉及包含有助于将化合物(例如生物活性大分子,特别是核酸或肽)递送到细胞中的缀合物的递送系统。 本发明还涉及用于将化合物例如生物活性大分子,核酸或肽递送到细胞中的所述缀合物。 本发明还涉及包含所述缀合物及其用途的药物组合物。 本发明还涉及将化合物递送至细胞或生物体,优选患者的方法。 缀合物包括:(a)介导细胞靶向和促进细胞摄取的至少一个模块,(b)促进转运到内质网(ER)的至少一个模块,(c)至少一个介导来自 ER至胞质溶胶,和(d)至少一种待递送的化合物,其中模块(a)至(c)和化合物(d)以任何排列彼此连接。