公开(公告)号：US20220348899A1

公开(公告)日：2022-11-03

申请号：US17835738

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 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：US20220348898A1

公开(公告)日：2022-11-03

申请号：US17835719

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 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：US20220347307A1

公开(公告)日：2022-11-03

申请号：US17705803

申请日：2022-03-28

Applicant: Translate Bio, Inc.

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

IPC: A61K47/64 ,  A61K47/54 ,  A61K47/69 ,  A61K31/7105 ,  C12N15/85

Abstract: The present invention provides, among other things, methods and compositions of formulating nucleic acid-containing nanoparticles for efficient delivery of payload in vivo such that the method and compositions can be used to generate mRNA vaccines.

公开(公告)号：US20220160633A1

公开(公告)日：2022-05-26

申请号：US17522754

申请日：2021-11-09

Applicant: Translate Bio, Inc.

Inventor： John Androsavich ,  Lianne Boeglin ,  Shraddha Sharma ,  Gang Sun ,  Neha Kaushal ,  Shrirang Karve

IPC: A61K9/127 ,  A61K31/7105 ,  A61K9/00

Abstract: The present invention provides, among other things, improved pharmaceutical compositions comprising codon-optimized mRNA encoding a peptide or polypeptide encapsulated in a lipid nanoparticle comprising one or more of the cationic lipids that are particularly effective for pulmonary delivery.

公开(公告)号：US20220073944A1

公开(公告)日：2022-03-10

申请号：US17404283

申请日：2021-08-17

Applicant: TRANSLATE BIO, INC.

Inventor： Frank DeRosa ,  Michael Heartlein ,  Daniel Crawford ,  Shrirang Karve

IPC: C12N15/85 ,  C07K14/505 ,  C12N9/10 ,  C12N9/02 ,  C07K14/705 ,  A61K48/00 ,  A61K38/17 ,  A61K38/44 ,  A61K38/45 ,  A61K38/18 ,  A61K31/7115 ,  A61K31/7125 ,  C07H21/02 ,  C12N15/67 ,  C12P21/00

Abstract: The present invention provides, among other things, multimeric coding nucleic acids that exhibit superior stability for in vivo and in vitro use. In some embodiments, a multimeric coding nucleic acid (MCNA) comprises two or more encoding polynucleotides linked via 3′ ends such that the multimeric coding nucleic acid compound comprises two or more 5′ ends.

公开(公告)号：US20220008338A1

公开(公告)日：2022-01-13

申请号：US17057929

申请日：2019-05-23

Applicant: Translate Bio, Inc.

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

IPC: A61K9/127 ,  C07C327/22 ,  C07D257/00 ,  A61K38/52 ,  A61K38/45

Abstract: Disclosed are cationic lipids which are compounds of Formula (I), (II), (III), (IV), (V), or (VI). 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：US11173190B2

公开(公告)日：2021-11-16

申请号：US15981757

申请日：2018-05-16

Applicant: Translate Bio, Inc.

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

IPC: A61K48/00 ,  A61K38/17 ,  A61P11/12 ,  A61K9/127 ,  A61K9/00 ,  C12N15/88

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：US11104652B2

公开(公告)日：2021-08-31

申请号：US16178142

申请日：2018-11-01

Applicant: TRANSLATE BIO, INC.

Inventor： Frank DeRosa ,  Shrirang Karve ,  Michael Heartlein

IPC: A61K9/127 ,  C07D241/08 ,  A61K31/7088 ,  A61K47/22 ,  A61K31/495 ,  A61K31/7105

Abstract: Provided, in part, is a composition comprising one or more chemical entities of formula I, each of which is a compound of formula I: a pharmaceutically acceptable salt thereof, a solvate thereof, or a solvate of a pharmaceutically acceptable salt thereof, the composition characterized in that greater than a first threshold amount of the total amount of chemical entities of formula I in the composition: are chemical entities of formula I.a, wherein the first threshold amount is 50%; or are chemical entities of formula I.b.1, wherein the first threshold amount is 25%; or are chemical entities of formula I.b.2, wherein the first threshold amount is 25%, wherein the chemical entities of formula I.a, I.b.1, and I.b.2, are described herein, and methods of using such compositions, for example, for the delivery of a polynucleotide in vivo.

公开(公告)号：US20210205421A1

公开(公告)日：2021-07-08

申请号：US17094327

申请日：2020-11-10

Applicant: Translate Bio, Inc.

Inventor： Frank DeRosa ,  Michael Heartlein ,  Lianne Smith ,  Shrirang Karve

IPC: A61K38/45 ,  C12N9/10 ,  A61K48/00 ,  A61K9/00 ,  A61K9/127

Abstract: The present invention provides, among other things, methods of treating ornithine transcarbamylase (OTC) deficiency, including administering to a subject in need of treatment a composition comprising an mRNA encoding an ornithine transcarbamylase (OTC) protein at an effective dose and an administration interval such that at least one symptom or feature of the OTC deficiency is reduced in intensity, severity, or frequency or has delayed 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：US10780183B2

公开(公告)日：2020-09-22

申请号：US16012138

申请日：2018-06-19

Applicant: Translate Bio, Inc.

Inventor： Michael Heartlein ,  Frank DeRosa ,  Jonathan Cherry ,  Paula Lewis ,  Shrirang Karve ,  Zarna Patel ,  Darshan Parekh ,  Susan Wood ,  Chi-Sung Chiu ,  Caroline J. Woo

IPC: A61P21/00 ,  A61K48/00 ,  A61K38/17 ,  A61K9/127 ,  C12N15/62 ,  A61K38/02 ,  C12N15/52 ,  A61K9/00 ,  A61P43/00 ,  C12N15/88 ,  A61K31/7088 ,  A61K9/107

Abstract: The present invention provides methods and compositions of treating Friedreich's ataxia (FRDA) based on administering an mRNA encoding a frataxin protein.