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公开(公告)号:US20220087935A1
公开(公告)日:2022-03-24
申请号:US17420346
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 , A61K31/711 , A61P11/00
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:US12076438B2
公开(公告)日:2024-09-03
申请号:US17066919
申请日:2020-10-09
Applicant: Translate Bio, Inc.
Inventor: Christian Cobaugh , Richard Wooster , Frank DeRosa , Anusha Dias , Shrirang Karve
CPC classification number: A61K9/127 , A61K9/0019 , A61K9/007 , A61K9/0078 , A61K38/1709 , A61K38/19 , A61K38/193 , A61K38/208 , A61K45/06 , A61P35/00
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:US20210106527A1
公开(公告)日:2021-04-15
申请号:US17066919
申请日:2020-10-09
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: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: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: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:US20210145860A1
公开(公告)日:2021-05-20
申请号:US17076195
申请日:2020-10-21
Applicant: Translate Bio, Inc.
Inventor: Richard Wooster , Anusha Dias , Dustin Cooper , Christian Cobaugh , Frank DeRosa , Tim Efthymiou , Jeffrey S. Dubins
IPC: A61K31/7105 , A61K47/54 , A61K47/60 , A61K47/69
Abstract: The present invention provides, among other things, methods and compositions for selective degradation of proteins. In some aspects, messenger RNAs (mRNAs) are described that encode a ubiquitin pathway moiety and a binding peptide that binds a target protein, wherein the mRNA is encapsulated within a lipid nanoparticle. Also provided herein are mRNAs that encode at least two binding peptides, wherein a first binding peptide binds a ubiquitin pathway moiety and a second binding peptide binds a target protein, and wherein the mRNA is encapsulated within a lipid nanoparticle.
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公开(公告)号:US20230145188A1
公开(公告)日:2023-05-11
申请号:US17275803
申请日:2019-09-13
Applicant: Translate Bio, Inc.
Inventor: Lianne Boeglin , Christian Cobaugh , Frank DeRosa , Michael Heartlein , Kim Askew
CPC classification number: A61K48/005 , A61K9/1271 , C12N15/88 , C12N15/52 , A61P3/00 , C12Y504/99002
Abstract: The present invention provides, among other things, methods and compositions for treating methylmalonic academia (MMA) based on mRNA therapy. The compositions used in treatment of MMA comprise an mRNA comprising a methymalonyl-CoA mutase (MUT) coding sequence and are administered at an effective dose and an administration interval such that at least one symptom or feature of MMA is reduced in intensity, severity, or frequency or has a delayed onset. mRNAs with optimized MUT 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:US20200215157A1
公开(公告)日:2020-07-09
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
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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