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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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公开(公告)号：US20230065040A1
公开(公告)日：2023-03-02
申请号：US17841194
申请日：2022-06-15
Applicant: Translate Bio, Inc.
Inventor： Shrirang Karve , Frank DeRosa , Zarna Bhavsar , Michael Heartlein
Abstract: The present invention provides, among other things, methods of formulating nucleic acid-containing nanoparticles with an enzyme to afford efficient delivery of payload to a cell or tissue of interest via subcutaneous administration. In some embodiments, the present invention provides a process in which mRNA-loaded lipid nanoparticles are co-mixed with various amounts of hyaluronidase and administered via subcutaneous administration. The resulting payload can be efficiently delivered to the liver and other organs or tissues of a treated subject.
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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：US11547666B2
公开(公告)日：2023-01-10
申请号：US17058334
申请日：2019-05-30
Applicant: Translate Bio, Inc.
Inventor： Yi Zhang , Shrirang Karve , Frank DeRosa , Michael Heartlein
Abstract: Disclosed are cationic lipids which are compounds of Formula I. Cationic lipids provided herein can be useful for delivery and expression of mRNA and encoded protein, e.g., as a component of liposomal delivery vehicle, and accordingly can be useful for treating various diseases, disorders and conditions, such as those associated with deficiency of one or more proteins.
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Patent Agency Ranking
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：US20220287966A1
公开(公告)日：2022-09-15
申请号：US17534956
申请日：2021-11-24
Applicant: Translate Bio, Inc.
Inventor： Shrirang Karve , Ashish Sarode , Natalia Vargas Montoya , Priyal Patel , Frank DeRosa
Abstract: The present invention provides, among other things, a liquid lipid nanoparticle (LNP) formulation encapsulating mRNA encoding a peptide or polypeptide, that is resistant to aggregation and to mRNA degradation following multiple rounds of freezing at −20° C. and rethawing.
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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：US20220072152A1
公开(公告)日：2022-03-10
申请号：US17448996
申请日：2021-09-27
Applicant: Translate Bio, Inc.
Inventor： Kim Askew , Jou-Ku Chung , Frank DeRosa , Michael Heartlein , Shrirang Karve , Thomas McCauley , Lianne Smith , Ann J. Barbier
Abstract: The present invention provides, among other things, methods of treating ornithine transcarbamylase deficiency, including administering to a subject in need of treatment a composition comprising an mRNA encoding an ornithine transcarbamylase protein at a low dose and at an administration interval such that at least one symptom or feature of the OTC deficiency is reduced.
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公开(公告)号：US20210378962A1
公开(公告)日：2021-12-09
申请号：US17286113
申请日：2019-10-18
Applicant: Translate Bio, Inc.
Inventor： Shrirang Karve , Priyal Patel , Natalia Vargas Montoya , Frank DeRosa , Michael Heartlein
IPC: A61K9/127 , A61K31/7105
Abstract: The present invention provides, among other things, a process of encapsulating messenger RNA (mRNA) in liposomes comprising a. providing a first stream comprising an mRNA solution at a first controlled flow rate, b. providing a second stream comprising a lipid solution at a second controlled flow rate, and c. mixing the first stream and the second stream to form mRNA-encapsulated liposomes, wherein the first controlled flow rate and the second controlled flow rate are achieved without use of a pump.
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