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：US11964051B2

公开(公告)日：2024-04-23

申请号：US17054886

申请日：2019-05-15

申请人： Translate Bio, Inc.

发明人： Frank DeRosa ,  Shrirang Karve ,  Yi Zhang ,  Michael Heartlein

IPC分类号： A61K9/127 ,  A61K9/00 ,  A61K38/18 ,  C07H13/06

CPC分类号： A61K9/1272 ,  A61K9/0019 ,  A61K9/0078 ,  A61K38/1816 ,  C07H13/06

摘要： Disclosed are cationic lipids which are compounds of Formula (I′). Cationic lipids 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：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：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：US20220395459A1

公开(公告)日：2022-12-15

申请号：US17737712

申请日：2022-05-05

申请人： Translate Bio, Inc.

发明人： Shrirang Karve ,  Zarna Patel ,  Yi Zhang ,  Ashish Sarode ,  Rebecca L. Goldman ,  Frank DeRosa ,  Michael Heartlein

IPC分类号： A61K9/127 ,  A61K9/14 ,  A61K9/51 ,  A61K31/7105

摘要： The present invention provides an improved process for lipid nanoparticle formulation and mRNA encapsulation. In some embodiments, the present invention provides a process of encapsulating messenger RNA (mRNA) in lipid nanoparticles comprising a step of mixing a solution of pre-formed lipid nanoparticles and mRNA at a low concentration.

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：US20220133631A1

公开(公告)日：2022-05-05

申请号：US17450628

申请日：2021-10-12

申请人： Translate Bio, Inc.

发明人： Monic Shah ,  Travis Jeanotte ,  Yi Zhang ,  Shrirang Karve ,  Kimberly Gillis ,  Frank DeRosa

IPC分类号： A61K9/127 ,  A61K31/7105

摘要： The present invention provides an improved process for lipid nanoparticle formulation and mRNA encapsulation. In some embodiments, the present invention provides a process of encapsulating messenger RNA (mRNA) in lipid nanoparticles comprising a step of mixing an mRNA solution containing low citrate concentration and a lipid solution at an ambient temperature. Thus, the present invention provides an effective, reliable, energy-saving and cost-effective method of encapsulating mRNA into lipid nanoparticles.

公开(公告)号：US20220071905A1

公开(公告)日：2022-03-10

申请号：US17291919

申请日：2019-11-07

申请人： Translate Bio, Inc.

发明人： Shrirang Karve ,  Yi Zhang ,  Frank DeRosa ,  Michael Heartlein

IPC分类号： A61K9/127 ,  A61K38/45 ,  A61K38/17 ,  C08G65/48

摘要： 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.