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公开(公告)号：US11173190B2
公开(公告)日：2021-11-16
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
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：US10780183B2
公开(公告)日：2020-09-22
申请号：US16012138
申请日：2018-06-19
Applicant: Translate Bio, Inc.
Inventor： Michael Heartlein , Frank DeRosa , Jonathan Cherry , Paula Lewis , Shrirang Karve , Zarna Patel , Darshan Parekh , Susan Wood , Chi-Sung Chiu , Caroline J. Woo
IPC: A61P21/00 , A61K48/00 , A61K38/17 , A61K9/127 , C12N15/62 , A61K38/02 , C12N15/52 , A61K9/00 , A61P43/00 , C12N15/88 , A61K31/7088 , A61K9/107
Abstract: 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：US20200085745A1
公开(公告)日：2020-03-19
申请号：US16553747
申请日：2019-08-28
Applicant: Translate Bio, Inc.
Inventor： Shrirang Karve , Zarna Patel , Yi Zhang , Ashish Sarode , Rebecca L. Ball , Frank DeRosa , Michael Heartlein
IPC: A61K9/127 , A61K9/14 , A61K9/51 , A61K31/7105
Abstract: The present invention provides an improved process for lipid nanoparticle formulation and mRNA encapsulation. In some embodiments, the present invention provides a process of encapsulating messenger RNA (mRNA) in lipid nanoparticles comprising a step of mixing a solution of pre-formed lipid nanoparticles and mRNA at a low concentration.
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公开(公告)号：US11975110B2
公开(公告)日：2024-05-07
申请号：US16966214
申请日：2019-02-01
Applicant: Translate Bio, Inc.
Inventor： Frank DeRosa , Michael Heartlein , Shrirang Karve , Zarna Patel
IPC: C08G63/685 , A61K9/00 , A61K9/14 , A61K9/51 , A61K38/17 , A61K38/45 , A61K48/00 , C07D319/12 , C08G63/78 , C12N15/113
CPC classification number: A61K9/5153 , A61K9/0019 , A61K9/14 , A61K9/5123 , A61K9/5146 , A61K38/1709 , A61K38/45 , C07D319/12 , C08G63/6852 , C08G63/78 , C12Y201/03003 , C12N15/113 , C12N2310/14 , C12N2310/141 , C12N2320/32
Abstract: Disclosed are cationic polymers comprising monomers such as those described in Formula (I). Such polymers can be useful for the preparation of therapeutic compositions [e.g., compositions comprising nucleic acids such as m RNA). Additionally, therapeutic compositions comprising these cationic, biodegradable polymers can have improved properties and reduced toxicity.
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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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Patent Agency Ranking
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：US20220324926A1
公开(公告)日：2022-10-13
申请号：US17693120
申请日：2022-03-11
Applicant: Translate Bio, Inc.
Inventor： Shrirang Karve , Frank DeRosa , Michael Heartlein , Zarna Patel
IPC: C07K14/47 , A61K9/00 , A61K9/51 , A61K9/127 , A61K9/19 , A61K47/26 , A61K45/06 , B82Y5/00 , A61P11/00
Abstract: The present invention provides, among other things, an improved method of treating cystic fibrosis (CF) in a human subject. The method comprises administering a composition comprising an mRNA encoding a Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) protein at a concentration of 0.5 mg/mL or greater to a human subject via nebulization. The composition is aerosolized using a nebulizer and a nominal dose of the mRNA is administered to the human subject via the nebulizer over a period of time, typically at least 30 minutes, at a suitable nebulization rate, e.g., at least 0.2 mL/minute.
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公开(公告)号：US11357726B2
公开(公告)日：2022-06-14
申请号：US16553747
申请日：2019-08-28
Applicant: Translate Bio, Inc.
Inventor： Shrirang Karve , Zarna Patel , Yi Zhang , Ashish Sarode , Rebecca L. Goldman , Frank DeRosa , Michael Heartlein
IPC: A61K9/12 , A61K9/127 , A61K9/14 , A61K9/51 , A61K31/7105
Abstract: The present invention provides an improved process for lipid nanoparticle formulation and mRNA encapsulation. In some embodiments, the present invention provides a process of encapsulating messenger RNA (mRNA) in lipid nanoparticles comprising a step of mixing a solution of pre-formed lipid nanoparticles and mRNA at a low concentration.
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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
Applicant: Translate Bio, Inc.
Inventor： Michael Heartlein , Frank DeRosa , Jonathan Cherry , Paula Lewis , Shrirang Karve , Zarna Patel , Darshan Parekh , Susan Wood , Chi-Sung Chiu , Caroline J. Woo
Abstract: 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：US20250025419A1
公开(公告)日：2025-01-23
申请号：US18762242
申请日：2024-07-02
Applicant: Translate Bio, Inc.
Inventor： Shrirang Karve , Zarna Patel , Yi Zhang , Ashish Sarode , Rebecca L. Goldman , Frank DeRosa , Michael Heartlein
IPC: A61K9/127 , A61K9/14 , A61K9/51 , A61K31/7105
Abstract: The present invention provides an improved process for lipid nanoparticle formulation and mRNA encapsulation. In some embodiments, the present invention provides a process of encapsulating messenger RNA (mRNA) in lipid nanoparticles comprising a step of mixing a solution of pre-formed lipid nanoparticles and mRNA at a low concentration.
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