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：US20220160827A1

公开(公告)日：2022-05-26

申请号：US17449679

申请日：2021-10-01

Applicant: Translate Bio, Inc.

Inventor： Michael Heartlein ,  Frank DeRosa ,  Alan Kimura ,  Jonathan Abysalh ,  Anusha Dias ,  Shrirang Karve ,  Zarna Patel

IPC: A61K38/17 ,  A61P11/12 ,  A61K9/127 ,  A61K9/00 ,  A61K48/00

Abstract: The present invention provides, among other things, methods of treating cystic fibrosis, comprising a step of administering to a subject in need of treatment a composition comprising an mRNA encoding a Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) protein, wherein the mRNA encoding the CFTR protein comprises a polynucleotide sequence at least 80% identical to SEQ ID NO: 1, wherein the mRNA is at a concentration of at least 0.4 mg/mL, and wherein the step of administering comprises inhalation.

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：US20220071905A1

公开(公告)日：2022-03-10

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 ,  A61K38/45 ,  A61K38/17 ,  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：US20220016029A1

公开(公告)日：2022-01-20

申请号：US17291923

申请日：2019-11-07

Applicant: Translate Bio, Inc.

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

IPC: A61K9/127 ,  C08G65/333 ,  C08G65/332 ,  C08G65/335 ,  A61K45/06

Abstract: The compounds disclosed herein (e.g., compounds of Formula (I), (II), (III), and (IV)) comprise a lipid substructure comprising a hydrophobic moiety and a hydrophilic moiety; and two or more polymeric groups (e.g., two or more polyethylene glycol (PEG) groups). 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：US20210378977A1

公开(公告)日：2021-12-09

申请号：US17262421

申请日：2019-07-23

Applicant: Translate Bio, Inc.

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

IPC: A61K9/51 ,  A61K38/17 ,  A61K48/00 ,  A61K9/50 ,  A61K38/45 ,  A61K9/00

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：US11174500B2

公开(公告)日：2021-11-16

申请号：US16551340

申请日：2019-08-26

Applicant: Translate Bio, Inc.

Inventor： Joseph Parella ,  Kimberly Gillis ,  Jonathan Abysalh ,  Travis Jeannotte ,  Erik Held ,  Frank DeRosa ,  Michael Heartlein ,  Rebecca Powell

IPC: C12N15/10 ,  C12P19/34 ,  C07H21/02 ,  C07H1/06

Abstract: The present invention provides, among other things, methods for purifying mRNA based on normal flow filtration for therapeutic use.

公开(公告)号：US20210324368A1

公开(公告)日：2021-10-21

申请号：US17103439

申请日：2020-11-24

Applicant: Translate Bio, Inc.

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

IPC: C12N15/10 ,  C07H21/02

Abstract: The present invention provides, among other things, methods of purifying messenger RNA (mRNA) including the steps of subjecting an impure preparation comprising in vitro synthesized rnRNA to a denaturing condition, and purifying the rnRNA from the impure preparation from step (a) by tangential flow filtration, wherein the mRNA purified from step (b) is substantially free of prematurely aborted RNA sequences and/or enzyme reagents used in in vitro synthesis.