公开(公告)号：US20240342307A1

公开(公告)日：2024-10-17

申请号：US18431597

申请日：2024-02-02

申请人： Translate Bio, Inc.

发明人： Braydon Charles Guild ,  Frank DeRosa ,  Michael Heartlein

IPC分类号： A61K48/00 ,  A61K9/00 ,  A61K9/127 ,  A61K31/7105 ,  C07J43/00 ,  C12N15/67

CPC分类号： A61K48/0033 ,  A61K9/0019 ,  A61K9/1271 ,  A61K9/1272 ,  A61K31/7105 ,  A61K48/0008 ,  A61K48/005 ,  C07J43/003 ,  C12N15/67

摘要： Disclosed herein are compositions and methods of modulating the expression of gene or the production of a protein by transfecting target cells with nucleic acids. The compositions disclosed herein demonstrate a high transfection efficacy and are capable of ameliorating diseases associated with protein or enzyme deficiencies.

公开(公告)号：US11975110B2

公开(公告)日：2024-05-07

申请号：US16966214

申请日：2019-02-01

申请人： Translate Bio, Inc.

发明人： Frank DeRosa ,  Michael Heartlein ,  Shrirang Karve ,  Zarna Patel

IPC分类号： C08G63/685 ,  A61K9/00 ,  A61K9/14 ,  A61K9/51 ,  A61K38/17 ,  A61K38/45 ,  A61K48/00 ,  C07D319/12 ,  C08G63/78 ,  C12N15/113

CPC分类号： 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

摘要： 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.

公开(公告)号：US11951180B2

公开(公告)日：2024-04-09

申请号：US18295152

申请日：2023-04-03

申请人： Translate Bio, Inc.

发明人： Frank DeRosa ,  Braydon Charles Guild ,  Michael Heartlein

IPC分类号： C12Q1/00 ,  A61K9/00 ,  A61K9/127 ,  A61K9/51 ,  A61K31/7105 ,  A61K31/713 ,  A61K38/18 ,  A61K38/47 ,  A61K38/48 ,  A61K38/57 ,  A61K48/00 ,  C07K14/505 ,  C07K14/81 ,  C12N9/40 ,  C12N9/64 ,  C12N15/52 ,  C12N15/85

CPC分类号： A61K48/0008 ,  A61K9/0019 ,  A61K9/0073 ,  A61K9/1271 ,  A61K9/1272 ,  A61K9/5123 ,  A61K31/713 ,  A61K38/1816 ,  A61K38/47 ,  A61K38/4846 ,  A61K38/57 ,  A61K48/0075 ,  A61K48/0091 ,  C07K14/505 ,  C07K14/8125 ,  C12N9/2465 ,  C12N9/644 ,  C12N15/52 ,  C12N15/85 ,  C12Y302/01022 ,  C12Y304/21022 ,  A61K48/00

摘要： 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.

公开(公告)号：US11920182B2

公开(公告)日：2024-03-05

申请号：US17325092

申请日：2021-05-19

申请人： Translate Bio, Inc.

发明人： Michael Heartlein ,  Frank DeRosa ,  Anusha Dias

IPC分类号： C12Q1/68 ,  C12Q1/6804

CPC分类号： C12Q1/6804 ,  C12Q1/6804 ,  C12Q2522/101 ,  C12Q2525/186 ,  C12Q2537/125 ,  C12Q2563/125

摘要： The present invention provides, among other things, methods of quantitating mRNA capping efficiency, particularly mRNA synthesized in vitro. In some embodiments, methods according to the present invention comprise providing an mRNA sample containing capped and uncapped mRNA, providing a cap specific binding substance under conditions that permit the formation of a complex between the cap specific binding substance and the capped mRNA, and quantitatively determining the amount of the complex as compared to a control, thereby quantifying mRNA capping efficiency.

公开(公告)号：US20240009281A1

公开(公告)日：2024-01-11

申请号：US18207609

申请日：2023-06-08

申请人： Translate Bio, Inc.

发明人： Frank DeRosa ,  Michael Heartlein

IPC分类号： A61K38/47 ,  A61K9/00 ,  A61K9/127 ,  A61K31/7105 ,  C12N9/40 ,  A61K48/00 ,  A61K47/22

CPC分类号： A61K38/47 ,  A61K9/0019 ,  A61K9/127 ,  A61K9/1271 ,  A61K9/1272 ,  A61K31/7105 ,  C12Y302/0102 ,  C12N9/2465 ,  A61K48/0058 ,  A61K47/22 ,  A61K48/0033

摘要： The present invention provides, among other things, methods of treating Pompe disease, including administering to a subject in need of treatment a composition comprising an mRNA encoding acid alpha-glucosidase (GAA) at an effective dose and an administration interval such that at least one symptom or feature of Pompe disease 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.

公开(公告)号：US20230293723A1

公开(公告)日：2023-09-21

申请号：US18295139

申请日：2023-04-03

申请人： Translate Bio, Inc.

发明人： Frank DeRosa ,  Braydon Charles Guild ,  Michael Heartlein

IPC分类号： A61K48/00 ,  A61K38/18 ,  A61K38/47 ,  A61K38/48 ,  A61K38/57 ,  A61K31/713 ,  A61K9/00 ,  A61K9/127 ,  C07K14/505 ,  C07K14/81 ,  C12N9/40 ,  C12N9/64 ,  C12N15/52 ,  C12N15/85 ,  A61K9/51

CPC分类号： A61K48/0008 ,  A61K38/1816 ,  A61K38/47 ,  A61K38/4846 ,  A61K38/57 ,  A61K31/713 ,  A61K9/0019 ,  A61K9/0073 ,  A61K9/1271 ,  A61K9/1272 ,  C07K14/505 ,  C07K14/8125 ,  C12N9/2465 ,  C12N9/644 ,  C12N15/52 ,  C12N15/85 ,  A61K9/5123 ,  A61K48/0075 ,  A61K48/0091 ,  C12Y302/01022 ,  C12Y304/21022 ,  A61K48/00

摘要： 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.

公开(公告)号：US11712463B2

公开(公告)日：2023-08-01

申请号：US17369783

申请日：2021-07-07

申请人： Translate Bio, Inc.

发明人： Frank DeRosa ,  Michael Heartlein

IPC分类号： A61K31/7105 ,  A61K47/22 ,  A61K38/47 ,  A61K9/00 ,  A61K9/127 ,  C12N9/40 ,  A61K48/00

CPC分类号： A61K38/47 ,  A61K9/0019 ,  A61K9/127 ,  A61K9/1271 ,  A61K9/1272 ,  A61K31/7105 ,  A61K47/22 ,  A61K48/0033 ,  A61K48/0058 ,  C12N9/2465 ,  C12Y302/0102

摘要： The present invention provides, among other things, methods of treating Pompe disease, including administering to a subject in need of treatment a composition comprising an mRNA encoding acid alpha-glucosidase (GAA) at an effective dose and an administration interval such that at least one symptom or feature of Pompe disease 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.

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.

公开(公告)号：US20230065040A1

公开(公告)日：2023-03-02

申请号：US17841194

申请日：2022-06-15

申请人： Translate Bio, Inc.

发明人： Shrirang Karve ,  Frank DeRosa ,  Zarna Bhavsar ,  Michael Heartlein

IPC分类号： A61K31/7105 ,  A61K9/127 ,  A61P3/00 ,  A61K38/45 ,  C12N9/26 ,  A61K9/00 ,  A61K47/69 ,  A61K38/47

摘要： 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.

公开(公告)号：US20230050301A1

公开(公告)日：2023-02-16

申请号：US17830102

申请日：2022-06-01

申请人： Translate Bio, Inc.

发明人： Frank DeRosa ,  Anusha Dias ,  Michael Heartlein

IPC分类号： C12N9/02 ,  A61K38/44 ,  A61K9/127 ,  A61K48/00

摘要： The present invention provides, among other things, methods of treating phenylketonuria (PKU), including administering to a subject in need of treatment a composition comprising an mRNA encoding phenylalanine hydroxylase (PAH) at an effective dose and an administration interval such that at least one symptom or feature of PKU 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.