公开(公告)号：US20240299489A9

公开(公告)日：2024-09-12

申请号：US17449679

申请日：2021-10-01

申请人： Translate Bio, Inc.

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

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

CPC分类号： A61K38/1709 ,  A61K9/0078 ,  A61K9/127 ,  A61K48/0075 ,  A61P11/12 ,  C12N15/88

摘要： 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：US12076438B2

公开(公告)日：2024-09-03

申请号：US17066919

申请日：2020-10-09

申请人： Translate Bio, Inc.

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

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

CPC分类号： A61K9/127 ,  A61K9/0019 ,  A61K9/007 ,  A61K9/0078 ,  A61K38/1709 ,  A61K38/19 ,  A61K38/193 ,  A61K38/208 ,  A61K45/06 ,  A61P35/00

摘要： 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：US11884692B2

公开(公告)日：2024-01-30

申请号：US17811022

申请日：2022-07-06

申请人： Translate Bio, Inc.

发明人： Frank DeRosa ,  Anusha Dias ,  Michael Heartlein ,  Shrirang Karve

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

CPC分类号： C07H1/06 ,  C07H21/00 ,  C07H21/02 ,  C12N15/1017

摘要： 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：US20240018514A1

公开(公告)日：2024-01-18

申请号：US18065952

申请日：2022-12-14

申请人： Translate Bio, Inc.

发明人： Jonathan Abysalh ,  Daniel Crawford ,  Frank DeRosa ,  Anusha Dias ,  Michael Heartlein

IPC分类号： C12N15/10 ,  C12P19/34 ,  C12N9/12 ,  G01N27/447

CPC分类号： C12N15/1096 ,  C12Y207/07006 ,  G01N27/44778 ,  C12N9/1247 ,  C12N15/1068 ,  C12P19/34

摘要： The present invention provides methods for large-scale production of a composition enriched for full-length mRNA molecules using an SP6 RNA polymerase and compositions produced using such methods and uses thereof.

公开(公告)号：US20220411781A1

公开(公告)日：2022-12-29

申请号：US17835725

申请日：2022-06-08

申请人： Translate Bio, Inc.

发明人： Frank DeRosa ,  Anusha Dias ,  Michael Heartlein ,  Shrirang Karve

IPC分类号： C12N15/10 ,  C07H21/02

摘要： 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：US20220402958A1

公开(公告)日：2022-12-22

申请号：US17811022

申请日：2022-07-06

申请人： Translate Bio, Inc.

发明人： Frank DeRosa ,  Anusha Dias ,  Michael Heartlein ,  Shrirang Karve

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

摘要： 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：US20220160827A1

公开(公告)日：2022-05-26

申请号：US17449679

申请日：2021-10-01

申请人： Translate Bio, Inc.

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

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

摘要： 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：US20220136022A1

公开(公告)日：2022-05-05

申请号：US17502816

申请日：2021-10-15

申请人： Translate Bio, Inc.

发明人： Jonathan Abysalh ,  Anusha Dias ,  Jorel E. Vargas ,  Dustin Cooper ,  Frank DeRosa

IPC分类号： C12P19/34 ,  A61K31/713

摘要： The present invention provides, among other things, methods for large-scale production of a composition comprising full-length messenger RNA product that is substantially free of double-stranded RNA, and compositions produced using such methods and uses thereof. The present invention is based, in part, on the surprising discovery that mRNA product produced by in vitro transcription using an SP6 RNA polymerase is substantially free of double-stranded RNA. In one aspect, the present invention provides methods of generating large-scale mRNA product for mRNA therapy without need for a chromatography step.

公开(公告)号：US20210324368A1

公开(公告)日：2021-10-21

申请号：US17103439

申请日：2020-11-24

申请人： Translate Bio, Inc.

发明人： Michael Heartlein ,  Frank DeRosa ,  Anusha Dias ,  Shrirang Karve

IPC分类号： C12N15/10 ,  C07H21/02

摘要： 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：US20210206846A1

公开(公告)日：2021-07-08

申请号：US17126833

申请日：2020-12-18

申请人： Translate Bio, Inc.

发明人： Michael Heartlein ,  Frank DeRosa ,  Anusha Dias ,  Braydon Charles Guild

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

摘要： 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.