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公开(公告)号：US20210187122A1
公开(公告)日：2021-06-24
申请号：US17016860
申请日：2020-09-10
申请人： Translate Bio, Inc.
发明人： Michael Heartlein , Frank DeRosa , Jonathan Cherry , Paula Lewis , Shrirang Karve , Zarna Patel , Darshan Parekh , Susan Wood , Chi-Sung Chiu , Caroline J. Woo
IPC分类号： A61K48/00 , C12N15/62 , A61K38/02 , A61K9/127 , A61K38/17 , A61P21/00 , C12N15/52 , A61K9/00 , A61P43/00 , C12N15/88 , A61K31/7088 , A61K9/107
摘要： The present invention provides methods and compositions of treating Friedreich's ataxia (FRDA) based on administering an mRNA encoding a frataxin protein.
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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
申请人： Translate Bio, Inc.
发明人： Anusha Dias , Darshan Parekh , Jeffrey S. Dubins , Christian Cobaugh , Shrirang Karve , Zarna Patel , Sara J. Dunaj , Frank DeRosa , Michael Heartlein
摘要： 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：US10780183B2
公开(公告)日：2020-09-22
申请号：US16012138
申请日：2018-06-19
申请人： Translate Bio, Inc.
发明人： Michael Heartlein , Frank DeRosa , Jonathan Cherry , Paula Lewis , Shrirang Karve , Zarna Patel , Darshan Parekh , Susan Wood , Chi-Sung Chiu , Caroline J. Woo
IPC分类号： A61P21/00 , A61K48/00 , A61K38/17 , A61K9/127 , C12N15/62 , A61K38/02 , C12N15/52 , A61K9/00 , A61P43/00 , C12N15/88 , A61K31/7088 , A61K9/107
摘要： The present invention provides methods and compositions of treating Friedreich's ataxia (FRDA) based on administering an mRNA encoding a frataxin protein.
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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
申请人： Translate Bio, Inc.
发明人： Anusha Dias , Darshan Parekh , Jeffrey S. Dubins , Christian Cobaugh , Shrirang Karve , Zarna Patel , Sara J. Dunaj , Frank DeRosa , Michael Heartlein
IPC分类号： A61K9/127 , A61K31/711 , A61P11/00
摘要： 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：US20230226147A1
公开(公告)日：2023-07-20
申请号：US18062435
申请日：2022-12-06
申请人： Translate Bio, Inc.
发明人： Anusha Dias , Darshan Parekh , Jeffrey S. Dubins , Christian Cobaugh , Shrirang Karve , Zarna Patel , Sara J. Dunaj , Frank DeRosa , Michael Heartlein
IPC分类号： A61K38/17 , A61K31/7105 , A61K9/00 , A61P11/00 , A61K31/711 , A61K9/127
CPC分类号： A61K38/1709 , A61K31/7105 , A61K9/0053 , A61P11/00 , A61K31/711 , A61K9/1272
摘要： 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
申请人： Translate Bio, Inc.
发明人： Anusha Dias , Darshan Parekh , Jeffrey S. Dubins , Christian Cobaugh , Shrirang Karve , Zarna Patel , Sara J. Dunaj , Frank DeRosa , Michael Heartlein
IPC分类号： A61K9/127 , A61K31/7105 , A61K38/17 , A61K9/00 , A61P11/00 , A61K31/711
摘要： 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：US20180369413A1
公开(公告)日：2018-12-27
申请号：US16012138
申请日：2018-06-19
申请人： Translate Bio, Inc.
发明人： Michael Heartlein , Frank DeRosa , Jonathan Cherry , Paula Lewis , Shrirang Karve , Zarna Patel , Darshan Parekh , Susan Wood , Chi-Sung Chiu , Caroline J. Woo
摘要： The present invention provides methods and compositions of treating Friedreich's ataxia (FRDA) based on administering an mRNA encoding a frataxin protein.
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