公开(公告)号：US10144942B2

公开(公告)日：2018-12-04

申请号：US15294249

申请日：2016-10-14

Applicant: Translate Bio, Inc.

Inventor： Bettina Strack-Logue ,  Frank DeRosa ,  Michael Heartlein

IPC: C12P19/34 ,  C12N9/12 ,  C07K14/47 ,  C12N15/62

Abstract: The present invention provides, among other things, methods and compositions for large-scale production of capped mRNA using SUMO-Guanylyl Transferase fusion protein.

公开(公告)号：US10137087B2

公开(公告)日：2018-11-27

申请号：US15878137

申请日：2018-01-23

Applicant: TRANSLATE BIO, INC.

Inventor： Frank DeRosa ,  Braydon Charles Guild ,  Michael Heartlein

IPC: A61K9/12 ,  A61K9/127 ,  C12N15/11 ,  A61K38/44 ,  A61K47/28 ,  A61K48/00

Abstract: Disclosed herein are novel lipids and liposomal compositions prepared using such compounds and related methods of neutralizing or otherwise modifying such liposomal compositions. The lipids described herein are useful for example, as liposomal vehicles to facilitate the delivery of encapsulated polynucleotides to target cells and the subsequent transfection of such target cells. In certain embodiments, one or more of the compounds that comprise the liposomal delivery vehicle may be neutralized or further modified such that the properties of the liposomal delivery vehicle are modified.

公开(公告)号：US20180272005A1

公开(公告)日：2018-09-27

申请号：US16002903

申请日：2018-06-07

Applicant: Translate Bio, Inc.

Inventor： Frank DeRosa ,  Michael Heartlein

IPC: A61K48/00 ,  C07D319/12 ,  A61K9/127 ,  A61K38/48 ,  A61K38/18

CPC classification number: A61K48/0033 ,  A61K9/1272 ,  A61K38/1816 ,  A61K38/4846 ,  A61K48/0025 ,  A61K48/0041 ,  A61K48/005 ,  C07D319/12 ,  C12N15/111 ,  C12N2310/11 ,  C12N2310/14 ,  C12N2310/141 ,  C12N2320/32 ,  C12Y304/21022

Abstract: The present invention provides, in part, a biodegradable compound of formula I, and sub-formulas thereof: Formula (I) or a pharmaceutically acceptable salt thereof, where each X independently is O or S, each Y independently is O or S, and each R1 independently is defined herein; and a liposome composition comprising the cationic lipid of formula I or a sub-formula thereof, and methods of delivering agents, such as nucleic acids including mRNA, in vivo, by administering to a subject the liposome comprising the cationic lipid of formula I or a sub-formula thereof, where the agent is encapsulated within the liposome.

公开(公告)号：US20180272003A1

公开(公告)日：2018-09-27

申请号：US16002889

申请日：2018-06-07

Applicant: Translate Bio, Inc.

Inventor： Frank DeRosa ,  Michael Heartlein

IPC: A61K48/00 ,  C07D319/12 ,  A61K9/127 ,  A61K38/48 ,  A61K38/18

CPC classification number: A61K48/0033 ,  A61K9/1272 ,  A61K38/1816 ,  A61K38/4846 ,  A61K48/0025 ,  A61K48/0041 ,  A61K48/005 ,  C07D319/12 ,  C12N15/111 ,  C12N2310/11 ,  C12N2310/14 ,  C12N2310/141 ,  C12N2320/32 ,  C12Y304/21022

Abstract: The present invention provides, in part, a biodegradable compound of formula I, and sub-formulas thereof: Formula (I) or a pharmaceutically acceptable salt thereof, where each X independently is O or S, each Y independently is O or S, and each R1 independently is defined herein; and a liposome composition comprising the cationic lipid of formula I or a sub-formula thereof, and methods of delivering agents, such as nucleic acids including mRNA, in vivo, by administering to a subject the liposome comprising the cationic lipid of formula I or a sub-formula thereof, where the agent is encapsulated within the liposome.

公开(公告)号：US20180153822A1

公开(公告)日：2018-06-07

申请号：US15809680

申请日：2017-11-10

Applicant: Translate Bio, Inc.

Inventor： Shrirang Karve ,  Frank DeRosa ,  Zarna Bhavsar ,  Michael Heartlein

IPC: A61K9/51 ,  A61K38/53 ,  A61K38/44 ,  A61K38/18 ,  A61K38/17 ,  A61K38/45

CPC classification number: A61K9/5192 ,  A61K9/1272 ,  A61K9/5123 ,  A61K38/177 ,  A61K38/1816 ,  A61K38/44 ,  A61K38/45 ,  A61K38/53 ,  A61K47/22 ,  A61K47/28 ,  C12N15/88 ,  C12Y114/16001 ,  C12Y201/03003 ,  C12Y603/04005

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.

公开(公告)号：US09943595B2

公开(公告)日：2018-04-17

申请号：US14959453

申请日：2015-12-04

Applicant: TRANSLATE BIO, INC.

Inventor： Frank DeRosa ,  Michael Heartlein

IPC: A61K39/395 ,  A61K38/17 ,  A61K9/127 ,  C07K16/00 ,  A61K48/00

CPC classification number: A61K39/3955 ,  A61K9/1272 ,  A61K38/177 ,  A61K48/00 ,  A61K48/0025 ,  A61K48/005 ,  A61K48/0075 ,  C07K16/00

Abstract: The present invention provides, among other things, a method of intra-articular delivery of messenger RNA (mRNA), comprising administering into a joint of a subject in need of delivery a composition comprising an mRNA encoding a protein, such that the administering of the composition results in expression of the protein encoded by the mRNA in the joint.

公开(公告)号：US20250162977A1

公开(公告)日：2025-05-22

申请号：US19029962

申请日：2025-01-17

Applicant: Translate Bio, Inc.

Inventor： Frank DeRosa ,  Braydon Charles Guild ,  Michael Heartlein

IPC: C07C211/21 ,  A61K9/1271 ,  A61K9/1272 ,  A61K9/50 ,  A61K38/18 ,  A61K38/47 ,  C12N15/85

Abstract: Disclosed herein are novel compounds, pharmaceutical compositions comprising such compounds and related methods of their use. The compounds described herein are useful, e.g., as liposomal delivery vehicles to facilitate the delivery of encapsulated polynucleotides to target cells and subsequent transfection of said target cells, and in certain embodiments are characterized as having one or more properties that afford such compounds advantages relative to other similarly classified lipids.

公开(公告)号：US20250152735A1

公开(公告)日：2025-05-15

申请号：US19027351

申请日：2025-01-17

Applicant: Translate Bio, Inc.

Inventor： Frank DeRosa ,  Braydon Charles Guild ,  Michael Heartlein

IPC: A61K48/00 ,  A61K9/00 ,  A61K9/1271 ,  A61K9/1272 ,  A61K9/51 ,  A61K31/713 ,  A61K38/18 ,  A61K38/47 ,  A61K38/48 ,  A61K38/57 ,  C07K14/505 ,  C07K14/81 ,  C12N9/40 ,  C12N9/64 ,  C12N15/52 ,  C12N15/85

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.

公开(公告)号：US12280147B2

公开(公告)日：2025-04-22

申请号：US17291919

申请日：2019-11-07

Applicant: Translate Bio, Inc.

Inventor： Shrirang Karve ,  Yi Zhang ,  Frank DeRosa ,  Michael Heartlein

IPC: A61K9/127 ,  A61K9/1273 ,  A61K38/17 ,  A61K38/45 ,  C08G65/48

Abstract: The compounds disclosed herein (e.g., compounds having a structure according to Formula (I/(II)/(III)/(IV), and (V)) are polymers wherein an organic polymeric segment (e.g., a polyethylene glycol (PEG) group) comprises covalent attachments to two or more lipid substructures, and each lipid substructure independently comprises a hydrophobic moiety and a hydrophilic moiety. The compounds 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.

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.