公开(公告)号：US20240408020A1

公开(公告)日：2024-12-12

申请号：US18815955

申请日：2024-08-27

Applicant: Translate Bio, Inc.

Inventor： Braydon Charles Guild ,  Michael Heartlein ,  Frank DeRosa ,  Jerry Chi Zhang

IPC: A61K9/19 ,  A61K9/127 ,  A61K9/51 ,  A61K31/7088 ,  A61K38/45 ,  A61K38/46 ,  A61K38/47 ,  A61K38/50 ,  C07C323/25 ,  C07C323/27 ,  C07C323/44 ,  C07D233/64 ,  C07J41/00 ,  C07J43/00 ,  C12N15/88

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：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.

公开(公告)号：US20220378939A1

公开(公告)日：2022-12-01

申请号：US17832196

申请日：2022-06-03

Applicant: Translate Bio, Inc.

Inventor： Frank DeRosa ,  Braydon Charles Guild ,  Michael Heartlein

IPC: 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

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：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.

公开(公告)号：US20220111074A1

公开(公告)日：2022-04-14

申请号：US17558555

申请日：2021-12-21

Applicant: TRANSLATE BIO, INC.

Inventor： Braydon Charles Guild ,  Frank DeRosa ,  Michael Heartlein

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

Abstract: 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：US20220111072A1

公开(公告)日：2022-04-14

申请号：US17558310

申请日：2021-12-21

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.

公开(公告)号：US11135274B2

公开(公告)日：2021-10-05

申请号：US15937359

申请日：2018-03-27

Applicant: Translate Bio, Inc.

Inventor： Braydon Charles Guild ,  Michael Heartlein ,  Frank DeRosa

IPC: A61K38/47 ,  A61K48/00 ,  C12N9/10 ,  C07K14/505 ,  C07K14/61 ,  C12N15/67 ,  C12N9/40 ,  A61K38/27 ,  A61K38/45 ,  A61K38/18

Abstract: Compositions for modulating the expression of a protein in a target cell comprising at least one RNA molecule which comprises at least one modification 5 conferring stability to the RNA, as well as related methods, are disclosed.

公开(公告)号：US20210206846A1

公开(公告)日：2021-07-08

申请号：US17126833

申请日：2020-12-18

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：US20200338214A1

公开(公告)日：2020-10-29

申请号：US16931231

申请日：2020-07-16

Applicant: Translate Bio, Inc.

Inventor： Braydon Charles Guild ,  Frank DeRosa ,  Michael Heartlein

IPC: 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

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：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.