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    EUGLOBULIN-BASED METHOD FOR DETERMINING THE BIOLOGICAL ACTIVITY OF DEFIBROTIDE
    64.
    发明申请
    EUGLOBULIN-BASED METHOD FOR DETERMINING THE BIOLOGICAL ACTIVITY OF DEFIBROTIDE 审中-公开

    公开(公告)号：US20200208148A1

    公开(公告)日：2020-07-02

    申请号：US16816741

    申请日：2020-03-12

    Applicant: GENTIUM S.R.L.

    Inventor： Terenzio IGNONI ,  Vijay KUMAR ,  Khalid ISLAM

    IPC: C12N15/11 ,  G01N33/86 ,  C12Q1/56 ,  C12Q1/37 ,  A61K31/711

    Abstract: It is disclosed a method for determining the biological activity of defibrotide, which comprises the steps of: a) bringing into contact defibrotide, mammalian euglobulin and a substrate specific for the plasmin which, by reaction with the plasmin, provides a measurable product; and b) measuring the amount of product formed at successive times, to thereby determine the biological activity of the defibrotide. Liquid defibrotide formulations are also disclosed, preferably water solutions, having a defined biological activity and, in particular, having an activity of 25 to 35 IU/mg of defibrotide, preferably from 27 to 32 IU/mg and, more preferably, from 28 to 32 IU/mg.

    Methods for modulating RNA splicing
    65.
    发明授权
    Methods for modulating RNA splicing 审中-公开

    公开(公告)号：US10668171B2

    公开(公告)日：2020-06-02

    申请号：US15577584

    申请日：2016-05-27

    Applicant: PTC Therapeutics, Inc.

    Inventor： Nikolai Naryshkin ,  Amal Dakka

    IPC: A61K48/00 ,  A61K31/519 ,  A61K31/711 ,  C07D239/70 ,  C12N15/11 ,  C12N15/85

    Abstract: In one aspect, described herein is a recognition element for splicing modifier (REMS) that can be recognized by a compound provided herein. In another aspect, described herein are methods for modulating the amount of a product of a gene, wherein a precursor RNA transcript transcribed from the gene contains a REMS, and the methods utilizing a compound described herein. More particularly, described herein are methods for modulating the amount of an RNA transcript or protein product encoded by a gene, wherein a precursor RNA transcript transcribed from the gene comprises a REMS, and the methods utilizing a compound described herein. In another aspect, provided herein are artificial gene constructs comprising a REMS, and uses of those artificial gene constructs to modulate functional protein production. In another aspect, provided herein are methods for altering endogenous genes to comprise a REMS, and the use of a compound described herein to modulate the functional protein produced from such altered endogenous genes.

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