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公开(公告)号:US20220111075A1
公开(公告)日:2022-04-14
申请号:US17560195
申请日:2021-12-22
Applicant: Translate Bio, Inc.
Inventor: Braydon Charles Guild , Frank DeRosa , Michael Heartlein
Abstract: Disclosed herein are compositions and methods of modulating the expression of gene or the production of a protein by transfecting target cells with nucleic acids. The compositions disclosed herein demonstrate a high transfection efficacy and are capable of ameliorating diseases associated with protein or enzyme deficiencies.
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公开(公告)号:US11254936B2
公开(公告)日:2022-02-22
申请号:US15983635
申请日:2018-05-18
Applicant: Translate Bio, Inc.
Inventor: Michael Heartlein , Braydon Charles Guild , Frank DeRosa
IPC: C12N15/11 , C07H21/02 , C07H21/00 , C07K14/505 , C12P21/02
Abstract: The invention provides, among other things, methods of stabilizing mRNA and nuclease resistant mRNA prepared in accordance with such methods. In certain embodiments, the nuclease resistant mRNA encodes a functional protein, such as enzyme, and is characterized by its resistance to nuclease digestion, increased half-life and/or its ability to produce increased amounts of the functional protein (e.g., enzyme) encoded thereby.
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公开(公告)号:US11234936B2
公开(公告)日:2022-02-01
申请号:US16790407
申请日:2020-02-13
Applicant: Translate Bio, Inc.
Inventor: Braydon Charles Guild , Michael Heartlein , Frank DeRosa , Jerry Chi Zhang
IPC: C07C323/25 , C07C323/44 , C12N15/88 , A61K38/45 , A61K38/46 , C07C323/27 , C07J41/00 , A61K9/51 , A61K9/19 , C07D233/64 , A61K9/127 , A61K38/47 , A61K38/50 , A61K31/7088 , C07J43/00
Abstract: Disclosed herein are novel compounds, pharmaceutical compositions comprising such compounds and related methods of their use. The compounds described herein are useful, e.g., as liposomal delivery vehicles to facilitate the delivery of encapsulated polynucleotides to target cells and subsequent iransfection of said target cells, and in certain embodiments are characterized as having one or more properties that afford such compounds advantages relative to other similarly classified lipids.
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公开(公告)号:US11090264B2
公开(公告)日:2021-08-17
申请号:US16258191
申请日:2019-01-25
Applicant: Translate Bio, Inc. , Ethris GmbH
Inventor: Michael Heartlein , Braydon Charles Guild , Frank DeRosa , Carsten Rudolph , Christian Plank
Abstract: Compositions comprising mRNA formulated for pulmonary administration and related method for delivery of the mRNA and/or encoded protein to a non-lung cell or tissue. The compositions and methods may be used to prevent or ameliorate the symptoms of diseases associated with the mRNA encoded protein.
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公开(公告)号:US20200085973A1
公开(公告)日:2020-03-19
申请号:US16681565
申请日:2019-11-12
Applicant: Translate Bio, Inc.
Inventor: Braydon Charles Guild , Frank DeRosa , Michael Heartlein
IPC: A61K48/00 , A61K9/51 , C12N15/85 , C12N15/52 , C12N9/64 , C12N9/40 , C07K14/81 , C07K14/505 , A61K9/127 , A61K9/00 , A61K31/713 , A61K38/57 , A61K38/48 , A61K38/47 , A61K38/18
Abstract: Disclosed herein are compositions and methods for modulating the production of a protein in a target cell. The compositions and methods disclosed herein are capable of ameliorating diseases associated with protein or enzyme deficiencies.
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公开(公告)号:US20190216843A1
公开(公告)日:2019-07-18
申请号:US16157050
申请日:2018-10-10
Applicant: Translate Bio, Inc.
Inventor: Frank DeRosa , Lianne Smith , Michael Heartlein , Braydon Charles Guild
IPC: A61K31/7105 , A61K48/00 , A61K9/14 , A61K9/127
CPC classification number: A61K31/7105 , A61K9/0019 , A61K9/0085 , A61K9/1271 , A61K9/1272 , A61K9/145 , A61K9/146 , A61K48/0025 , A61K48/0033
Abstract: Disclosed herein are pharmaceutical compositions that comprise “blends” of lipid nanoparticles and related methods of using such blended compositions to deliver polynucleotides to one or more target cells, tissues or organs. The blended compositions are generally characterized as being able to efficiently deliver polynucleotides to target cells and by their ability to enhance the expression of such polynucleotides and the production of functional proteins by target cells.
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公开(公告)号:US10350303B1
公开(公告)日:2019-07-16
申请号:US16286400
申请日:2019-02-26
Applicant: Translate Bio, Inc.
Inventor: Braydon Charles Guild , Frank DeRosa , Michael Heartlein
IPC: A61K48/00 , C07K14/81 , A61K38/18 , A61K38/47 , A61K38/48 , A61K38/57 , A61K9/00 , A61K9/51 , C12N15/85 , C12N15/52 , C12N9/64 , C12N9/40 , C07K14/505 , A61K9/127 , A61K31/713
Abstract: Disclosed herein are compositions and methods for modulating the production of a protein in a target cell. The compositions and methods disclosed herein are capable of ameliorating diseases associated with protein or enzyme deficiencies.
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公开(公告)号:US10130649B2
公开(公告)日:2018-11-20
申请号:US14775818
申请日:2014-03-14
Applicant: TRANSLATE BIO, INC.
Inventor: Frank Derosa , Lianne Smith , Michael Heartlein , Braydon Charles Guild
IPC: A61K9/127 , A61K31/7105 , A61K9/14 , A61K48/00 , A61K9/00
Abstract: Disclosed herein are pharmaceutical compositions that comprise “blends” of lipid nanoparticles and related methods of using such blended compositions to deliver polynucleotides to one or more target cells, tissues or organs. The blended compositions are generally characterized as being able to efficiently deliver polynucleotides to target cells and by their ability to enhance the expression of such polynucleotides and the production of functional proteins by target cells.
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公开(公告)号:US10065919B2
公开(公告)日:2018-09-04
申请号:US15368280
申请日:2016-12-02
Applicant: TRANSLATE BIO, INC.
Inventor: Frank DeRosa , Braydon Charles Guild , Michael Heartlein
Abstract: Disclosed herein are novel compounds, pharmaceutical compositions comprising such compounds and related methods of their use. The compounds described herein are useful, e.g., as liposomal delivery vehicles to facilitate the delivery of encapsulated polynucleotides to target cells and subsequent transfection of said target cells, and in certain embodiments are characterized as having one or more properties that afford such compounds advantages relative to other similarly classified lipids.
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公开(公告)号:US20250162977A1
公开(公告)日:2025-05-22
申请号:US19029962
申请日:2025-01-17
Applicant: Translate Bio, Inc.
Inventor: Frank DeRosa , Braydon Charles Guild , Michael Heartlein
IPC: C07C211/21 , A61K9/1271 , A61K9/1272 , A61K9/50 , A61K38/18 , A61K38/47 , C12N15/85
Abstract: Disclosed herein are novel compounds, pharmaceutical compositions comprising such compounds and related methods of their use. The compounds described herein are useful, e.g., as liposomal delivery vehicles to facilitate the delivery of encapsulated polynucleotides to target cells and subsequent transfection of said target cells, and in certain embodiments are characterized as having one or more properties that afford such compounds advantages relative to other similarly classified lipids.
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