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公开(公告)号:US10087247B2
公开(公告)日:2018-10-02
申请号:US14775835
申请日:2014-03-14
Applicant: TRANSLATE BIO, INC.
Inventor: Michael Heartlein , Frank Derosa , Anusha Dias , Braydon Charles Guild
Abstract: The present invention provides, among other things, methods and compositions for delivering an antibody in vivo by administering to a subject in need thereof one or more mRNAs encoding a heavy chain and a light chain of an antibody, and wherein the antibody is expressed systemically in the subject. In some embodiments, the one or more mRNAs comprise a first mRNA encoding the heavy chain and a second mRNA encoding the light chain of the antibody.
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公开(公告)号:US09957499B2
公开(公告)日:2018-05-01
申请号:US14775915
申请日:2014-03-14
Applicant: TRANSLATE BIO, INC.
Inventor: Michael Heartlein , Frank DeRosa , Anusha Dias , Shirang Karve
CPC classification number: C12N15/10 , C07H21/02 , C12N15/1017
Abstract: The present invention provides, among other things, methods of purifying messenger RNA (mRNA) including the steps of subjecting an impure preparation comprising in vitro synthesized mRNA to a denaturing condition, and purifying the mRNA from the impure preparation from step (a) by tangential flow filtration, wherein the mRNA purified from step (b) is substantially free of prematurely aborted RNA sequences and/or enzyme reagents used in in vitro synthesis.
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公开(公告)号:US09850269B2
公开(公告)日:2017-12-26
申请号:US14696140
申请日:2015-04-24
Applicant: TRANSLATE BIO, INC.
Inventor: Frank DeRosa , Anusha Dias , Shrirang Karve , Michael Heartlein
CPC classification number: C07H1/06 , C07H21/00 , C07H21/02 , C12N15/1017
Abstract: The present invention provides, among other things, methods of purifying messenger RNA (mRNA) including the steps of (a) precipitating mRNA from an impure preparation; (b) subjecting the impure preparation comprising precipitated mRNA to a purification process involving membrane filtration such that the precipitated mRNA is captured by a membrane; and (c) eluting the captured precipitated mRNA from the membrane by re-solubilizing the mRNA, thereby resulting in a purified mRNA solution. In some embodiments, a purification process involving membrane filtration suitable for the present invention is tangential flow filtration.
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公开(公告)号:US20250057765A1
公开(公告)日:2025-02-20
申请号:US18782767
申请日:2024-07-24
Applicant: Translate Bio, Inc.
Inventor: Christian Cobaugh , Richard Wooster , Frank DeRosa , Anusha Dias , Shrirang Karve
Abstract: The present invention provides, among other things, methods and compositions for cancer treatment. The methods and compositions disclosed herein are particularly effective in reducing the size/volume of a tumor and inhibiting tumor growth.
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公开(公告)号:US11920182B2
公开(公告)日:2024-03-05
申请号:US17325092
申请日:2021-05-19
Applicant: Translate Bio, Inc.
Inventor: Michael Heartlein , Frank DeRosa , Anusha Dias
IPC: C12Q1/68 , C12Q1/6804
CPC classification number: C12Q1/6804 , C12Q1/6804 , C12Q2522/101 , C12Q2525/186 , C12Q2537/125 , C12Q2563/125
Abstract: The present invention provides, among other things, methods of quantitating mRNA capping efficiency, particularly mRNA synthesized in vitro. In some embodiments, methods according to the present invention comprise providing an mRNA sample containing capped and uncapped mRNA, providing a cap specific binding substance under conditions that permit the formation of a complex between the cap specific binding substance and the capped mRNA, and quantitatively determining the amount of the complex as compared to a control, thereby quantifying mRNA capping efficiency.
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公开(公告)号:US20230226147A1
公开(公告)日:2023-07-20
申请号:US18062435
申请日:2022-12-06
Applicant: Translate Bio, Inc.
Inventor: Anusha Dias , Darshan Parekh , Jeffrey S. Dubins , Christian Cobaugh , Shrirang Karve , Zarna Patel , Sara J. Dunaj , Frank DeRosa , Michael Heartlein
IPC: A61K38/17 , A61K31/7105 , A61K9/00 , A61P11/00 , A61K31/711 , A61K9/127
CPC classification number: A61K38/1709 , A61K31/7105 , A61K9/0053 , A61P11/00 , A61K31/711 , A61K9/1272
Abstract: The present invention provides, among other things, methods and compositions for treating primary ciliary dyskinesia (PCD) based on mRNA therapy. The compositions used in treatment of PCD comprise an mRNA comprising a dynein axonemal heavy chain 5 (DNAH5) coding sequence and are administered at an effective dose and an administration interval such that at least one symptom or feature of PCD is reduced in intensity, severity, or frequency or has a delayed onset. mRNAs with optimized DNAH5 coding sequences are provided that can be administered without the need for modifying the nucleotides of the mRNA to achieve sustained in vivo function.
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公开(公告)号:US20230050301A1
公开(公告)日:2023-02-16
申请号:US17830102
申请日:2022-06-01
Applicant: Translate Bio, Inc.
Inventor: Frank DeRosa , Anusha Dias , Michael Heartlein
Abstract: The present invention provides, among other things, methods of treating phenylketonuria (PKU), including administering to a subject in need of treatment a composition comprising an mRNA encoding phenylalanine hydroxylase (PAH) at an effective dose and an administration interval such that at least one symptom or feature of PKU is reduced in intensity, severity, or frequency or has delayed in onset. In some embodiments, the mRNA is encapsulated in a liposome comprising one or more cationic lipids, one or more non-cationic lipids, one or more cholesterol-based lipids and one or more PEG-modified lipids.
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公开(公告)号:US11559561B2
公开(公告)日:2023-01-24
申请号:US16736417
申请日:2020-01-07
Applicant: Translate Bio, Inc.
Inventor: Anusha Dias , Darshan Parekh , Jeffrey S. Dubins , Christian Cobaugh , Shrirang Karve , Zarna Patel , Sara J. Dunaj , Frank DeRosa , Michael Heartlein
IPC: A61K9/127 , A61K31/7105 , A61K38/17 , A61K9/00 , A61P11/00 , A61K31/711
Abstract: The present invention provides, among other things, methods and compositions for treating primary ciliary dyskinesia (PCD) based on mRNA therapy. The compositions used in treatment of PCD comprise an mRNA comprising a dynein axonemal heavy chain 5 (DNAH5) coding sequence and are administered at an effective dose and an administration interval such that at least one symptom or feature of PCD is reduced in intensity, severity, or frequency or has a delayed onset. mRNAs with optimized DNAH5 coding sequences are provided that can be administered without the need for modifying the nucleotides of the mRNA to achieve sustained in vivo function.
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公开(公告)号:US11377642B2
公开(公告)日:2022-07-05
申请号:US16245828
申请日:2019-01-11
Applicant: Translate Bio, Inc.
Inventor: Frank DeRosa , Michael Heartlein , Anusha Dias
Abstract: The present invention provides, among other things, methods of treating phenylketonuria (PKU), including administering to a subject in need of treatment a composition comprising an mRNA encoding phenylalanine hydroxylase (PAH) at an effective dose and an administration interval such that at least one symptom or feature of PKU is reduced in intensity, severity, or frequency or has delayed in onset. In some embodiments, the mRNA is encapsulated in a liposome comprising one or more cationic lipids, one or more non-cationic lipids, one or more cholesterol-based lipids and one or more PEG-modified lipids.
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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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