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公开(公告)号:US11253605B2
公开(公告)日:2022-02-22
申请号:US15907131
申请日:2018-02-27
Applicant: Translate Bio, Inc.
Inventor: Anusha Dias , Jonathan Abysalh , Brian Bettencourt , Frank DeRosa , Michael Heartlein
IPC: A61K47/69 , A61K47/64 , A61K9/127 , C12N15/10 , A61K38/17 , C07K14/47 , A61P11/00 , A61K9/00 , A61K31/7105
Abstract: The present invention provides, among other things, improved methods and pharmaceutical compositions for treating cystic fibrosis based on codon optimized mRNA encoding a Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) protein.
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公开(公告)号:US10975369B2
公开(公告)日:2021-04-13
申请号:US15907086
申请日:2018-02-27
Applicant: Translate Bio, Inc.
Inventor: Frank DeRosa , Michael Heartlein , Jonathan Abysalh , Daniel Crawford , Anusha Dias , Shrirang Karve
Abstract: The present invention relates, in part, to methods for large-scale purification of mRNA. The method includes, at least, a step of centrifuging an mRNA suspension in a centrifuge comprising a porous substrate at a speed sufficient to remove process contaminants and to precipitate purified mRNA composition onto the porous substrate.
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公开(公告)号:US20180333457A1
公开(公告)日:2018-11-22
申请号:US15981757
申请日:2018-05-16
Applicant: Translate Bio, Inc.
Inventor: Michael Heartlein , Frank DeRosa , Alan Kimura , Jonathan Abysalh , Anusha Dias , Shrirang Karve , Zarna Patel
CPC classification number: A61K38/1709 , A61K9/0078 , A61K9/127 , A61K48/0075 , A61P11/12
Abstract: The present invention provides, among other things, methods of treating cystic fibrosis, comprising a step of administering to a subject in need of treatment a composition comprising an mRNA encoding a Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) protein, wherein the mRNA encoding the CFTR protein comprises a polynucleotide sequence at least 80% identical to SEQ ID NO: 1, wherein the mRNA is at a concentration of at least 0.4 mg/mL, and wherein the step of administering comprises inhalation.
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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
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.
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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.
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公开(公告)号:US20210180041A1
公开(公告)日:2021-06-17
申请号:US17130114
申请日:2020-12-22
Applicant: Translate Bio, Inc.
Inventor: Frank DeRosa , Michael Heartlein , Jonathan Abysalh , Daniel Crawford , Anusha Dias , Shrirang Karve
Abstract: The present invention relates, in part, to methods for large-scale purification of mRNA. The method includes, at least, a step of centrifuging an mRNA suspension in a centrifuge comprising a porous substrate at a speed sufficient to remove process contaminants and to precipitate purified mRNA composition onto the porous substrate.
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公开(公告)号:US20210123041A1
公开(公告)日:2021-04-29
申请号:US17070877
申请日:2020-10-14
Applicant: Translate Bio, Inc.
Inventor: Jonathan Abysalh , Daniel Crawford , Frank DeRosa , Shrirang Karve , Anusha Dias , Michael Heartlein
IPC: C12N15/10 , B01L3/00 , C12Q1/6806
Abstract: The present invention relates, in part, to methods for large-scale purification of mRNA. The method includes, at least, steps of forming an mRNA slurry, stirring the slurry, and vacuum or pressure filtering the slurry.
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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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公开(公告)号:US20220193247A1
公开(公告)日:2022-06-23
申请号:US17570352
申请日:2022-01-06
Applicant: Translate Bio, Inc.
Inventor: Anusha Dias , Jonathan Abysalh , Brian Bettencourt , Frank DeRosa , Michael Heartlein
IPC: A61K47/64 , A61K9/127 , C12N15/10 , A61K38/17 , C07K14/47 , A61K47/69 , A61P11/00 , A61K9/00 , A61K31/7105
Abstract: The present invention provides, among other things, improved methods and pharmaceutical compositions for treating cystic fibrosis based on codon optimized mRNA encoding a Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) protein.
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公开(公告)号:US10808241B2
公开(公告)日:2020-10-20
申请号:US15906864
申请日:2018-02-27
Applicant: Translate Bio, Inc.
Inventor: Jonathan Abysalh , Daniel Crawford , Frank DeRosa , Shrirang Karve , Anusha Dias , Michael Heartlein
IPC: C12N15/10 , B01L3/00 , C12Q1/6806
Abstract: The present invention relates, in part, to methods for large-scale purification of mRNA. The method includes, at least, steps of forming an mRNA slurry, stirring the slurry, and vacuum or pressure filtering the slurry.
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