公开(公告)号：US20210009503A1

公开(公告)日：2021-01-14

申请号：US16983121

申请日：2020-08-03

Applicant: Translate Bio, Inc.

Inventor： Frank DeRosa ,  Braydon Charles Guild ,  Michael Heartlein

IPC: C07C211/21 ,  A61K9/127 ,  C12N15/85 ,  A61K9/50 ,  A61K38/18 ,  A61K38/47

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：US10822368B2

公开(公告)日：2020-11-03

申请号：US16282106

申请日：2019-02-21

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：US10786455B2

公开(公告)日：2020-09-29

申请号：US16182090

申请日：2018-11-06

Applicant: TRANSLATE BIO, INC.

Inventor： Frank DeRosa ,  Braydon Charles Guild ,  Michael Heartlein

IPC: 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：US20200237671A1

公开(公告)日：2020-07-30

申请号：US16790407

申请日：2020-02-13

Applicant: Translate Bio, Inc.

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

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

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 iransfection 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：US10576166B2

公开(公告)日：2020-03-03

申请号：US15092226

申请日：2016-04-06

Applicant: TRANSLATE BIO, INC.

Inventor： 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：US20200022921A1

公开(公告)日：2020-01-23

申请号：US16520000

申请日：2019-07-23

Applicant: Translate Bio, Inc.

Inventor： Shrirang Karve ,  Frank DeRosa ,  Michael Heartlein ,  Zarna Patel ,  Ashish Sarode

IPC: A61K9/50 ,  A61P11/00 ,  A61K38/17 ,  A61K9/00 ,  A61K9/48 ,  A61K48/00

Abstract: The present invention provides stable, dry powder messenger RNA formulations for therapeutic use, and methods of making and using the same.

公开(公告)号：US20190388563A1

公开(公告)日：2019-12-26

申请号：US16422403

申请日：2019-05-24

Applicant: Translate Bio, Inc.

Inventor： Michael Heartlein

IPC: A61K48/00

Abstract: Disclosed are compositions and methods for producing therapeutic fusion proteins in vivo. The compositions and methods disclosed herein are capable of ameliorating diseases by providing therapeutic protein delivery.

公开(公告)号：US20190321489A1

公开(公告)日：2019-10-24

申请号：US16502672

申请日：2019-07-03

Applicant: Translate Bio, Inc.

Inventor： Braydon Charles Guild ,  Frank DeRosa ,  Michael Heartlein

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

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：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.

公开(公告)号：US20190216730A1

公开(公告)日：2019-07-18

申请号：US16258191

申请日：2019-01-25

Applicant: Translate Bio, Inc. ,  Ethris GmbH

Inventor： Michael Heartlein ,  Braydon Charles Guild ,  Frank DeRoas ,  Carsten Rudolph ,  Christian Plank

IPC: A61K9/00 ,  A61M16/14 ,  A61M15/00 ,  A61K31/7088 ,  C12N15/88

CPC classification number: A61K9/0073 ,  A61K31/7088 ,  A61K48/0075 ,  A61M15/009 ,  A61M16/14 ,  C12N15/88

Abstract: Compositions comprising mRNA formulated for pulmonary administration and related method for delivery of the mRNA and/or encoded protein to a non-lung cell or tissue. The compositions and methods may be used to prevent or ameliorate the symptoms of diseases associated with the mRNA encoded protein.