公开(公告)号：US10350303B1

公开(公告)日：2019-07-16

申请号：US16286400

申请日：2019-02-26

申请人： Translate Bio, Inc.

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

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

摘要： 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：US10172924B2

公开(公告)日：2019-01-08

申请号：US15073163

申请日：2016-03-17

申请人： TRANSLATE BIO, INC.

发明人： Frank DeRosa ,  Michael Heartlein

IPC分类号： A61K38/47 ,  A61K9/00 ,  A61K9/127 ,  A61K48/00 ,  A61K31/7105 ,  A61K47/22 ,  C12N9/40

摘要： The present invention provides, among other things, methods of treating Pompe disease, including administering to a subject in need of treatment a composition comprising an mRNA encoding acid alpha-glucosidase (GAA) at an effective dose and an administration interval such that at least one symptom or feature of Pompe disease is reduced in intensity, severity, or frequency or has delayed in onset. In some embodiments, the mRNA is encapsulated in a liposome comprising one or more cationic lipids, one or more non-cationic lipids, one or more cholesterol-based lipids and one or more PEG-modified lipids.

公开(公告)号：US10130649B2

公开(公告)日：2018-11-20

申请号：US14775818

申请日：2014-03-14

申请人： TRANSLATE BIO, INC.

发明人： Frank Derosa ,  Lianne Smith ,  Michael Heartlein ,  Braydon Charles Guild

IPC分类号： A61K9/127 ,  A61K31/7105 ,  A61K9/14 ,  A61K48/00 ,  A61K9/00

摘要： Disclosed herein are pharmaceutical compositions that comprise “blends” of lipid nanoparticles and related methods of using such blended compositions to deliver polynucleotides to one or more target cells, tissues or organs. The blended compositions are generally characterized as being able to efficiently deliver polynucleotides to target cells and by their ability to enhance the expression of such polynucleotides and the production of functional proteins by target cells.

公开(公告)号：US10065919B2

公开(公告)日：2018-09-04

申请号：US15368280

申请日：2016-12-02

申请人： TRANSLATE BIO, INC.

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

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

摘要： 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：US09970047B2

公开(公告)日：2018-05-15

申请号：US14775846

申请日：2014-03-14

申请人： TRANSLATE BIO, INC.

发明人： Michael Heartlein ,  Frank DeRosa ,  Anusha Dias

IPC分类号： C12Q1/68 ,  C07K14/50 ,  C12N9/00 ,  C07K14/505 ,  C12N9/02

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

摘要： 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：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：US20240263217A1

公开(公告)日：2024-08-08

申请号：US18565849

申请日：2022-06-03

申请人： TRANSLATE BIO, INC.

发明人： Frank DEROSA ,  Anusha DIAS ,  Xiaobo GU

IPC分类号： C12Q1/6825 ,  C12N15/10

CPC分类号： C12Q1/6825 ,  C12N15/10

摘要： The invention relates to a method of quantifying capping efficiency in a sample from an in vitro transcription reaction mixture comprising a plurality of mRNA transcript, characterized by a step of contacting the mRNA transcripts with an oligonucleotide complementary to a sequence of nucleotides in the 5′ untranslated region of the mRNA transcripts to form an mRNA:DNA hybrid between the oligonucleotide and the sequence of nucleotides of the mRNA transcripts in order to release the first five, six, or seven nucleotides of the mRNA transcripts using nuclease (e.g., RNAse H) digestion.

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.