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    TREATMENT OF CYSTIC FIBROSIS BY DELIVERY OF NEBULIZED mRNA ENCODING CFTR
    4.
    发明申请
    TREATMENT OF CYSTIC FIBROSIS BY DELIVERY OF NEBULIZED mRNA ENCODING CFTR 有权

    公开(公告)号：US20220323542A1

    公开(公告)日：2022-10-13

    申请号：US17631322

    申请日：2020-07-30

    Applicant: Translate Bio, Inc.

    Inventor： Ann Barbier ,  Michael Heartlein ,  Frank DeRoss ,  Jonathan Abysalh ,  Anusha Dias ,  Shrirang Karve ,  Zama Patel

    IPC: A61K38/17 ,  A61P11/00 ,  A61K9/00 ,  A61P43/00 ,  A61K31/47 ,  A61K31/443

    Abstract: The present invention provides, among other things, an improved method of treating cystic fibrosis (CF) in a human subject. The method comprises administration of a composition comprising an mRNA encoding a Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) protein by nebulization at a dose between 7 mg and 25 mg. A suitable dose for use in the method of the invention is selected on the basis that it provides the human subject with at least a 3% increase in absolute change in ppFEV1 (percent predicted forced expiratory volume in one second) from baseline ppFEV1 at two days following the administration. In addition or alternatively, the dose is selected to provide the human subject with at least a 2% increase in absolute change in ppFEV1 from baseline ppFEV1 at one week following the administration. In addition or alternatively, the dose is selected to provide the human subject with at least a 4% maximum increase in absolute change in ppFEV1 from baseline ppFEV1 through one week following administration.

    Methods and Compositions for Messenger RNA Purification
    5.
    发明申请
    Methods and Compositions for Messenger RNA Purification 有权

    公开(公告)号：US20210388338A1

    公开(公告)日：2021-12-16

    申请号：US17291797

    申请日：2019-11-08

    Applicant: Translate Bio, Inc.

    Inventor： Jonathan Abysalh ,  Jorel E. Vargas ,  Frank DeRosa ,  Anusha Dias

    IPC: C12N15/10

    Abstract: The present invention provides, among other things, methods for purifying mRNA, which involves removing impurities from a messenger RNA preparation synthesized by large scale in vitro transcription process (IVT), by precipitating the IVT-synthesized mRNA in a buffer comprising a denaturing salt in combination with a reducing agent, followed by capturing the precipitated mRNA and dissolving the captured mRNA into a solution to obtain purified mRNA.

    METHODS FOR PURIFICATION OF MESSENGER RNA
    8.
    发明申请
    METHODS FOR PURIFICATION OF MESSENGER RNA 有权

    公开(公告)号：US20210002635A1

    公开(公告)日：2021-01-07

    申请号：US16875418

    申请日：2020-05-15

    Applicant: Translate Bio, Inc.

    Inventor： Jonathan Abysalh ,  Frank DeRosa ,  Jorel Vargas ,  Cameron M. Smith

    IPC: C12N15/10

    Abstract: The present invention provides, among other things, methods for purifying high quality messenger RNA (mRNA) suitable for clinical use, without using any caustic or flammable solvents. The present invention is, in part, based on surprising discovery that mRNA can be successfully purified by selective precipitation and washing without using ethanol while maintaining integrity and high purity of mRNA. Thus, the present invention provides an effective, reliable, and safer method of purifying RNA from large scale manufacturing process therapeutic applications without using any caustic or inflammable solvents.

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