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公开(公告)号：US20230000781A1
公开(公告)日：2023-01-05
申请号：US17745428
申请日：2022-05-16
Applicant: Translate Bio, Inc.
Inventor： Frank DeRosa , Michael Heartlein , Shrirang Karve , Zarna Patel , Ashish Sarode
Abstract: The present invention provides stable, dry powder messenger RNA formulations for therapeutic use, and methods of making and using the same.
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公开(公告)号：US20220411791A1
公开(公告)日：2022-12-29
申请号：US17572260
申请日：2022-01-10
Applicant: Translate Bio, Inc.
Inventor： Michael Heartlein , Braydon Charles Guild , Frank DeRosa
IPC: C12N15/11 , C07H21/02 , C07H21/00 , C07K14/505
Abstract: The invention provides, among other things, methods of stabilizing mRNA and nuclease resistant mRNA prepared in accordance with such methods. hi 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：US20220364145A1
公开(公告)日：2022-11-17
申请号：US17746657
申请日：2022-05-17
Applicant: Translate Bio, Inc.
Inventor： Frank DeRosa , Anusha Dias , Michael Heartlein
IPC: C12Q1/68 , C12Q1/6823 , C07K14/505 , C12N9/02
Abstract: The present invention provides, among other things, methods of quantitating mRNA capping efficiency, particularly for mRNA synthesized in vitro. In some embodiments, the methods comprise chromatographic methods of quantifying capping efficiency and methylation status of the caps.
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公开(公告)号：US20220323355A1
公开(公告)日：2022-10-13
申请号：US17054886
申请日：2019-05-15
Applicant: Translate Bio, Inc.
Inventor： Frank DeRosa , Shrirang Karve , Yi Zhang , 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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公开(公告)号：US11427535B2
公开(公告)日：2022-08-30
申请号：US17172640
申请日：2021-02-10
Applicant: Translate Bio, Inc.
Inventor： Michael Heartlein , Frank DeRosa , Shrirang Karve
IPC: C07C305/06 , C07C317/18 , A61K9/127 , A61K47/20 , A61K47/54 , A61K47/58 , A61K31/7105
Abstract: Disclosed are dendritic anionic lipids which are compounds of Formula (I): wherein R and R1 are non-polar groups, L is a linking moiety, and Dm is a dendritic moiety of m generations, each as defined herein. These dendritic anionic lipids are useful for delivery and expression of m RNA 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：US20220249699A1
公开(公告)日：2022-08-11
申请号：US17472622
申请日：2021-09-11
Applicant: Translate Bio, Inc.
Inventor： Braydon Charles Guild , Frank DeRosa , Michael Heartlein
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：US20220111070A1
公开(公告)日：2022-04-14
申请号：US17556573
申请日：2021-12-20
Applicant: Translate Bio, Inc.
Inventor： Braydon Charles Guild , Frank DeRosa , Michael Heartlein
IPC: A61K48/00 , A61K31/713 , A61K38/48 , A61K9/127 , A61K38/47 , A61K38/57 , C07K14/81 , A61K38/18 , A61K9/00 , C12N15/52 , C12N9/40 , A61K9/51 , C12N9/64 , C12N15/85 , C07K14/505
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：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：US11253605B2
公开(公告)日：2022-02-22
申请号：US15907131
申请日：2018-02-27
Applicant: Translate Bio, Inc.
Inventor： Anusha Dias , Jonathan Abysalh , Brian Bettencourt , Frank DeRosa , Michael Heartlein
IPC: A61K47/69 , A61K47/64 , A61K9/127 , C12N15/10 , A61K38/17 , C07K14/47 , A61P11/00 , A61K9/00 , A61K31/7105
Abstract: The present invention provides, among other things, improved methods and pharmaceutical compositions for treating cystic fibrosis based on codon optimized mRNA encoding a Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) protein.
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公开(公告)号：US20220010347A1
公开(公告)日：2022-01-13
申请号：US17217752
申请日：2021-03-30
Applicant: Translate Bio, Inc.
Inventor： Bettina Strack-Logue , Frank DeRosa , Michael Heartlein
Abstract: The present invention provides, among other things, methods and compositions for large-scale production of capped mRNA using SUMO-Guanylyl Transferase fusion protein.
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