公开(公告)号：US10584165B2

公开(公告)日：2020-03-10

申请号：US16129412

申请日：2018-09-12

Applicant: Translate Bio, Inc.

Inventor： Michael Heartlein ,  Frank DeRosa ,  Anusha Dias ,  Braydon Charles Guild

IPC: C07K16/24 ,  C07K16/22 ,  C12N15/88 ,  A61K9/00 ,  A61K9/127 ,  A61K47/69

Abstract: The present invention provides, among other things, methods and compositions for delivering an antibody in vivo by administering to a subject in need thereof one or more mRNAs encoding a heavy chain and a light chain of an antibody, and wherein the antibody is expressed systemically in the subject. In some embodiments, the one or more mRNAs comprise a first mRNA encoding the heavy chain and a second mRNA encoding the light chain of the antibody.

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.

公开(公告)号：US20190263850A1

公开(公告)日：2019-08-29

申请号：US16282106

申请日：2019-02-21

Applicant: TRANSLATE BIO, INC.

Inventor： Frank DeRosa ,  Michael Heartlein

IPC: C07H21/02 ,  C12N15/67

Abstract: Disclosed are messenger RNA molecules and related compositions incorporating a 4′-thio modification in the furanose ring of at least one nucleotide residue, and methods of using these mRNAs to produce an encoded therapeutic protein in vivo and to treat or prevent diseases or disorders. In certain embodiments, the 4′-thio modified mRNA provides for enhanced stability and/or reduced immunogenicity in in vivo therapies.

公开(公告)号：US20190175761A1

公开(公告)日：2019-06-13

申请号：US16233031

申请日：2018-12-26

Applicant: Translate Bio, Inc.

Inventor： Braydon Charles Guild ,  Frank DeRosa ,  Michael Heartlein

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

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

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：US20190144480A1

公开(公告)日：2019-05-16

申请号：US16196664

申请日：2018-11-20

Applicant: Translate Bio, Inc.

Inventor： Frank DeRosa ,  Anusha Dias ,  Shrirang Karve ,  Michael Heartlein

IPC: C07H1/06 ,  C12N15/10 ,  C07H21/02 ,  C07H21/00

Abstract: The present invention provides, among other things, methods of purifying messenger RNA (mRNA) including the steps of (a) precipitating mRNA from an impure preparation; (b) subjecting the impure preparation comprising precipitated mRNA to a purification process involving membrane filtration such that the precipitated mRNA is captured by a membrane; and (c) eluting the captured precipitated mRNA from the membrane by re-solubilizing the mRNA, thereby resulting in a purified mRNA solution. In some embodiments, a purification process involving membrane filtration suitable for the present invention is tangential flow filtration.

公开(公告)号：US10286082B2

公开(公告)日：2019-05-14

申请号：US16002889

申请日：2018-06-07

Applicant: Translate Bio, Inc.

Inventor： Frank DeRosa ,  Michael Heartlein

IPC: C07D319/12 ,  A61K48/00 ,  C12N15/11 ,  A61K9/127 ,  A61K38/18 ,  A61K38/48

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：US20190136283A1

公开(公告)日：2019-05-09

申请号：US16165372

申请日：2018-10-19

Applicant: Translate Bio, Inc.

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

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

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：US10266843B2

公开(公告)日：2019-04-23

申请号：US15482431

申请日：2017-04-07

Applicant: Translate Bio, Inc.

Inventor： Frank DeRosa ,  Michael Heartlein ,  Daniel Crawford ,  Shrirang Karve

IPC: C07H21/02 ,  C07H21/04 ,  C12N15/85 ,  C07K14/505 ,  C12N9/10 ,  C12N9/02 ,  C07K14/705 ,  A61K48/00 ,  A61K38/17 ,  A61K38/44 ,  A61K38/45 ,  A61K38/18 ,  A61K31/7115 ,  A61K31/7125 ,  C12P21/00 ,  C12N15/67 ,  A01K67/00

Abstract: The present invention provides, among other things, multimeric coding nucleic acids that exhibit superior stability for in vivo and in vitro use. In some embodiments, a multimeric coding nucleic acid (MCNA) comprises two or more encoding polynucleotides linked via 3′ ends such that the multimeric coding nucleic acid compound comprises two or more 5′ ends.

公开(公告)号：US10266559B2

公开(公告)日：2019-04-23

申请号：US14776506

申请日：2014-03-14

Applicant: TRANSLATE BIO, INC.

Inventor： Frank DeRosa ,  Michael Heartlein

IPC: C12N15/67 ,  C07H21/02

Abstract: Disclosed are messenger RNA molecules and related compositions incorporating a 4′-thio modification in the furanose ring of at least one nucleotide residue, and methods of using these mRNAs to produce an encoded therapeutic protein in vivo and to treat or prevent diseases or disorders. In certain embodiments, the 4′-thio modified mRNA provides for enhanced stability and/or reduced immunogenicity in in vivo therapies.

公开(公告)号：US20190100753A1

公开(公告)日：2019-04-04

申请号：US15983635

申请日：2018-05-18

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. In 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.