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公开(公告)号：US20230302039A1
公开(公告)日：2023-09-28
申请号：US18049210
申请日：2022-10-24
Applicant: Translate Bio, Inc. , Ethris GmbH
Inventor： Michael Heartlein , Braydon Charles Guild , Frank DeRosa , Carsten Rudolph , Christian Plank , Lianne Smith
IPC: A61K31/713 , A61K31/7105 , A61K48/00 , A61K31/7115 , A61K9/00 , A61K9/51 , A61K47/69 , C07K14/705 , A61K9/127 , C12N9/14
CPC classification number: A61K31/713 , A61K31/7105 , A61K48/005 , A61K31/7115 , A61K9/0078 , A61K9/5123 , A61K47/6935 , C07K14/705 , A61K9/0073 , A61K9/1271 , C12N9/14 , C12N15/63
Abstract: Materials, formulations, production methods, and methods for delivery of CFTR mRNA for induction of CFTR expression, including in the mammalian lung are provided. The present invention is particularly useful for treating cystic fibrosis.
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公开(公告)号：US11510937B2
公开(公告)日：2022-11-29
申请号：US16540960
申请日：2019-08-14
Applicant: Translate Bio, Inc. , Ethris GmbH
Inventor： Michael Heartlein , Braydon Charles Guild , Frank DeRosa , Carsten Rudolph , Christian Plank , Lianne Smith
IPC: A61K31/713 , C07H21/04 , A61K31/7105 , A61K48/00 , A61K31/7115 , A61K9/00 , A61K9/51 , A61K47/69 , C07K14/705 , A61K9/127 , C12N9/14 , C12N15/63 , C07H21/02 , C07K14/47 , C12N15/88
Abstract: The present disclosure relates to materials, formulations, production methods, and methods for delivery of CFTR mRNA, including but not limited to chemically modified mRNA for induction of CFTR expression, including in the mammalian lung. The present invention is particularly useful for treating cystic fibrosis, but is also useful in the treatment of diseases related to CFTR gene.
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公开(公告)号：US10646504B2
公开(公告)日：2020-05-12
申请号：US16157050
申请日：2018-10-10
Applicant: Translate Bio, Inc.
Inventor： Frank DeRosa , Lianne Smith , Michael Heartlein , Braydon Charles Guild
IPC: A61K31/7105 , A61K9/127 , A61K48/00 , A61K9/14 , A61K9/00
Abstract: Disclosed herein are pharmaceutical compositions that comprise “blends” of lipid nanoparticles and related methods of using such blended compositions to deliver polynucleotides to one or more target cells, tissues or organs. The blended compositions are generally characterized as being able to efficiently deliver polynucleotides to target cells and by their ability to enhance the expression of such polynucleotides and the production of functional proteins by target cells.
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公开(公告)号：US20200046752A1
公开(公告)日：2020-02-13
申请号：US16540960
申请日：2019-08-14
Applicant: Translate Bio, Inc. , Ethris GmbH
Inventor： Michael Heartlein , Braydon Charles Guild , Frank DeRosa , Carsten Rudolph , Christian Plank , Lianne Smith
IPC: A61K31/713 , C12N9/14 , A61K9/127 , A61K9/00 , C07K14/705 , A61K47/69 , A61K9/51 , A61K31/7115 , A61K48/00 , A61K31/7105
Abstract: Materials, formulations, production methods, and methods for delivery of CFTR mRNA for induction of CFTR expression, including in the mammalian lung are provided. The present invention is particularly useful for treating cystic fibrosis.
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公开(公告)号：US12268754B2
公开(公告)日：2025-04-08
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
IPC: A61K48/00 , A61K9/00 , A61K9/127 , A61K9/1272 , A61K38/45 , A61K47/69 , A61P3/00 , C12N15/52 , C12N15/85
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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Patent Agency Ranking
公开(公告)号：US10420791B2
公开(公告)日：2019-09-24
申请号：US15625648
申请日：2017-06-16
Applicant: Translate Bio, Inc. , Ethris GmbH
Inventor： Michael Heartlein , Braydon Charles Guild , Frank DeRosa , Carsten Rudolph , Christian Plank , Lianne Smith
IPC: A61K48/00 , C12N15/63 , C07H21/04 , A61K31/713 , C07K14/705 , A61K31/7105 , A61K31/7115 , A61K9/00 , A61K9/51 , A61K9/127 , C12N9/14 , A61K47/69 , C07H21/02 , C07K14/47 , C12N15/88
Abstract: The present invention is particularly useful for treating cystic fibrosis. The disclosure concerns method and compositions for treating cystic fibrosis. The method comprises inducing CFTR expression in epithelial cells in a lung of a mammal by contacting the epithelial cells in the lung of the mammal with a composition, where the composition is a pharmaceutical composition comprising in vitro transcribed mRNA.
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公开(公告)号：US11224642B2
公开(公告)日：2022-01-18
申请号：US14521351
申请日：2014-10-22
Applicant: TRANSLATE BIO, INC.
Inventor： Michael Heartlein , Frank DeRosa , Lianne Smith
IPC: A61K31/7088 , A61K9/127 , A61K38/53 , C12N9/00 , A61K48/00 , A61K9/51 , A61K31/7105
Abstract: The present invention provides, among other things, methods of treating Argininosuccinate Synthetase Deficiency (ASD), including administering to a subject in need of treatment a composition comprising an mRNA encoding argininosuccinate synthetase (ASS1) at an effective dose and an administration interval such that at least one symptom or feature of ASD is reduced in intensity, severity, or frequency or has delayed in onset. In some embodiments, the mRNA is encapsulated in a liposome comprising one or more cationic lipids, one or more non-cationic lipids, one or more cholesterol-based lipids and one or more PEG-modified lipids.
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公开(公告)号：US20210205421A1
公开(公告)日：2021-07-08
申请号：US17094327
申请日：2020-11-10
Applicant: Translate Bio, Inc.
Inventor： Frank DeRosa , Michael Heartlein , Lianne Smith , Shrirang Karve
Abstract: The present invention provides, among other things, methods of treating ornithine transcarbamylase (OTC) deficiency, including administering to a subject in need of treatment a composition comprising an mRNA encoding an ornithine transcarbamylase (OTC) protein at an effective dose and an administration interval such that at least one symptom or feature of the OTC deficiency is reduced in intensity, severity, or frequency or has delayed onset. In some embodiments, the mRNA is encapsulated in a liposome comprising one or more cationic lipids, one or more non-cationic lipids, one or more cholesterol-based lipids and one or more PEG-modified lipids.
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公开(公告)号：US20190192688A1
公开(公告)日：2019-06-27
申请号：US16228596
申请日：2018-12-20
Applicant: Translate Bio, Inc.
Inventor： Kim Askew , Jou-Ku Chung , Frank DeRosa , Michael Heartlein , Shrirang Karve , Thomas McCauley , Lianne Smith , Ann J. Barbier
CPC classification number: A61K48/005 , A61K9/0019 , A61K9/1272 , A61K38/45 , A61K47/6929 , A61K48/0025 , A61K48/0075 , A61K48/0083 , A61P3/00 , C12N15/52 , C12N15/85 , C12Y201/03003
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：US12201677B2
公开(公告)日：2025-01-21
申请号：US17094327
申请日：2020-11-10
Applicant: Translate Bio, Inc.
Inventor： Frank DeRosa , Michael Heartlein , Lianne Smith , Shrirang Karve
Abstract: The present invention provides, among other things, methods of treating ornithine transcarbamylase (OTC) deficiency, including administering to a subject in need of treatment a composition comprising an mRNA encoding an ornithine transcarbamylase (OTC) protein at an effective dose and an administration interval such that at least one symptom or feature of the OTC deficiency is reduced in intensity, severity, or frequency or has delayed onset. In some embodiments, the mRNA is encapsulated in a liposome comprising one or more cationic lipids, one or more non-cationic lipids, one or more cholesterol-based lipids and one or more PEG-modified lipids.
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