公开(公告)号：US20210106527A1

公开(公告)日：2021-04-15

申请号：US17066919

申请日：2020-10-09

Applicant: Translate Bio, Inc.

Inventor： Christian Cobaugh ,  Richard Wooster ,  Frank DeRosa ,  Anusha Dias ,  Shrirang Karve

IPC: A61K9/127 ,  A61K38/20 ,  A61K38/17 ,  A61K38/19 ,  A61P35/00 ,  A61K9/00 ,  A61K45/06

Abstract: The present invention provides, among other things, methods and compositions for cancer treatment. The methods and compositions disclosed herein are particularly effective in reducing the size/volume of a tumor and inhibiting tumor growth.

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：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：US12234446B2

公开(公告)日：2025-02-25

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.

公开(公告)号：US12084711B2

公开(公告)日：2024-09-10

申请号：US17746657

申请日：2022-05-17

Applicant: Translate Bio, Inc.

Inventor： Frank DeRosa ,  Anusha Dias ,  Michael Heartlein

IPC: C12Q1/68 ,  C07K14/505 ,  C12N9/02 ,  C12Q1/6823

CPC classification number: C12Q1/68 ,  C07K14/505 ,  C12N9/0069 ,  C12Q1/6823 ,  C12Y113/12007 ,  C12Q1/6823 ,  C12Q2521/327 ,  C12Q2525/186 ,  C12Q2537/113 ,  C12Q2565/137

Abstract: The present invention provides, among other things, methods of quantitating mRNA capping efficiency, particularly for mRNA synthesized in vitro. In some embodiments, the methods comprise chromatographic methods of quantifying capping efficiency and methylation status of the caps.

公开(公告)号：US20240197825A1

公开(公告)日：2024-06-20

申请号：US17761893

申请日：2020-09-17

Applicant: Translate Bio, Inc.

Inventor： Richard Wooster ,  Frank DeRosa ,  Lianne Boeglin ,  Priyaanka Nanduri ,  Anusha Dias ,  Khang Anh Tran

IPC: A61K38/17 ,  A61K48/00

CPC classification number: A61K38/177 ,  A61K48/0033 ,  A61K48/005

Abstract: The present invention provides, among other things, improved methods and pharmaceutical compositions for treating cystic fibrosis based on codon optimized mRNA encoding an engineered or mutant Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) protein.

公开(公告)号：US20230407358A1

公开(公告)日：2023-12-21

申请号：US17800157

申请日：2021-02-18

Applicant: Translate Bio, Inc.

Inventor： Dustin Cooper ,  Frank DeRosa ,  Anusha Dias ,  Jeffrey S. Dubins ,  Xiaobo Gu ,  Khang Anh Tran

IPC: C12P19/34 ,  C12N9/12 ,  C12N15/10

CPC classification number: C12P19/34 ,  C12Y207/07006 ,  C12N9/1247 ,  C12N15/1068

Abstract: The present invention provides methods for preparing optimized DNA sequences as templates for in vitro transcription of mRNA. These DNA sequences are optimized to avoid premature termination of transcription by RNA polymerase. The invention also provides methods for preparing optimized DNA sequences that include one or more termination signal at their 3′ end to reduce or prevent non-templated “runoff” transcription.

公开(公告)号：US11820977B2

公开(公告)日：2023-11-21

申请号：US17835710

申请日：2022-06-08

Applicant: Translate Bio, Inc.

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

IPC: C12N5/10 ,  C07H21/02 ,  C12N15/10

CPC classification number: C12N15/10 ,  C07H21/02 ,  C12N15/1017

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 mRNA to a denaturing condition, and purifying the mRNA 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.

公开(公告)号：US20220389403A1

公开(公告)日：2022-12-08

申请号：US17835710

申请日：2022-06-08

Applicant: Translate Bio, Inc.

Inventor： Frank DeRosa ,  Anusha Dias ,  Michael Heartlein ,  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 mRNA to a denaturing condition, and purifying the mRNA 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.

公开(公告)号：US20220323542A1

公开(公告)日：2022-10-13

申请号：US17631322

申请日：2020-07-30

Applicant: Translate Bio, Inc.

Inventor： Ann Barbier ,  Michael Heartlein ,  Frank DeRoss ,  Jonathan Abysalh ,  Anusha Dias ,  Shrirang Karve ,  Zama Patel

IPC: A61K38/17 ,  A61P11/00 ,  A61K9/00 ,  A61P43/00 ,  A61K31/47 ,  A61K31/443

Abstract: The present invention provides, among other things, an improved method of treating cystic fibrosis (CF) in a human subject. The method comprises administration of a composition comprising an mRNA encoding a Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) protein by nebulization at a dose between 7 mg and 25 mg. A suitable dose for use in the method of the invention is selected on the basis that it provides the human subject with at least a 3% increase in absolute change in ppFEV1 (percent predicted forced expiratory volume in one second) from baseline ppFEV1 at two days following the administration. In addition or alternatively, the dose is selected to provide the human subject with at least a 2% increase in absolute change in ppFEV1 from baseline ppFEV1 at one week following the administration. In addition or alternatively, the dose is selected to provide the human subject with at least a 4% maximum increase in absolute change in ppFEV1 from baseline ppFEV1 through one week following administration.