公开(公告)号：US20220135608A1

公开(公告)日：2022-05-05

申请号：US17344535

申请日：2021-06-10

Applicant: Translate Bio, Inc.

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

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

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：US20210388338A1

公开(公告)日：2021-12-16

申请号：US17291797

申请日：2019-11-08

Applicant: Translate Bio, Inc.

Inventor： Jonathan Abysalh ,  Jorel E. Vargas ,  Frank DeRosa ,  Anusha Dias

IPC: C12N15/10

Abstract: The present invention provides, among other things, methods for purifying mRNA, which involves removing impurities from a messenger RNA preparation synthesized by large scale in vitro transcription process (IVT), by precipitating the IVT-synthesized mRNA in a buffer comprising a denaturing salt in combination with a reducing agent, followed by capturing the precipitated mRNA and dissolving the captured mRNA into a solution to obtain purified mRNA.

公开(公告)号：US11059841B2

公开(公告)日：2021-07-13

申请号：US16196664

申请日：2018-11-20

Applicant: Translate Bio, Inc.

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

IPC: C12Q1/68 ,  C07H21/00 ,  C07H1/06 ,  C12N15/10 ,  C07H21/02

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：US20210180041A1

公开(公告)日：2021-06-17

申请号：US17130114

申请日：2020-12-22

Applicant: Translate Bio, Inc.

Inventor： Frank DeRosa ,  Michael Heartlein ,  Jonathan Abysalh ,  Daniel Crawford ,  Anusha Dias ,  Shrirang Karve

IPC: C12N15/10 ,  C07H21/04

Abstract: The present invention relates, in part, to methods for large-scale purification of mRNA. The method includes, at least, a step of centrifuging an mRNA suspension in a centrifuge comprising a porous substrate at a speed sufficient to remove process contaminants and to precipitate purified mRNA composition onto the porous substrate.

公开(公告)号：US20210123041A1

公开(公告)日：2021-04-29

申请号：US17070877

申请日：2020-10-14

Applicant: Translate Bio, Inc.

Inventor： Jonathan Abysalh ,  Daniel Crawford ,  Frank DeRosa ,  Shrirang Karve ,  Anusha Dias ,  Michael Heartlein

IPC: C12N15/10 ,  B01L3/00 ,  C12Q1/6806

Abstract: The present invention relates, in part, to methods for large-scale purification of mRNA. The method includes, at least, steps of forming an mRNA slurry, stirring the slurry, and vacuum or pressure filtering the slurry.

公开(公告)号：US20200215157A1

公开(公告)日：2020-07-09

申请号：US16736417

申请日：2020-01-07

Applicant: Translate Bio, Inc.

Inventor： Anusha Dias ,  Darshan Parekh ,  Jeffrey S. Dubins ,  Christian Cobaugh ,  Shrirang Karve ,  Zarna Patel ,  Sara J. Dunaj ,  Frank DeRosa ,  Michael Heartlein

IPC: A61K38/17 ,  A61K9/127 ,  A61K9/00

Abstract: The present invention provides, among other things, methods and compositions for treating primary ciliary dyskinesia (PCD) based on mRNA therapy. The compositions used in treatment of PCD comprise an mRNA comprising a dynein axonemal heavy chain 5 (DNAH5) coding sequence and are administered at an effective dose and an administration interval such that at least one symptom or feature of PCD is reduced in intensity, severity, or frequency or has a delayed onset. mRNAs with optimized DNAH5 coding sequences are provided that can be administered without the need for modifying the nucleotides of the mRNA to achieve sustained in vivo function.

公开(公告)号：US10208295B2

公开(公告)日：2019-02-19

申请号：US15349720

申请日：2016-11-11

Applicant: TRANSLATE BIO, INC.

Inventor： Frank DeRosa ,  Michael Heartlein ,  Anusha Dias

IPC: A61K38/44 ,  C12N9/02 ,  A61K9/127 ,  A61K48/00

Abstract: The present invention provides, among other things, methods of treating phenylketonuria (PKU), including administering to a subject in need of treatment a composition comprising an mRNA encoding phenylalanine hydroxylase (PAH) at an effective dose and an administration interval such that at least one symptom or feature of PKU 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：US12060381B2

公开(公告)日：2024-08-13

申请号：US17811029

申请日：2022-07-06

Applicant: Translate Bio, Inc.

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

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

CPC classification number: C07H1/06 ,  C07H21/00 ,  C07H21/02 ,  C12N15/1017

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：US11976272B2

公开(公告)日：2024-05-07

申请号：US17070877

申请日：2020-10-14

Applicant: Translate Bio, Inc.

Inventor： Jonathan Abysalh ,  Daniel Crawford ,  Frank DeRosa ,  Shrirang Karve ,  Anusha Dias ,  Michael Heartlein

IPC: C12N15/10 ,  B01L3/00 ,  C12Q1/6806

CPC classification number: C12N15/1017 ,  B01L3/502753 ,  C12Q1/6806 ,  B01L2200/028 ,  B01L2200/16 ,  B01L2300/0681 ,  B01L2400/0409 ,  C12Q2523/113 ,  C12Q2523/308 ,  C12Y201/03003

Abstract: The present invention relates, in part, to methods for large-scale purification of mRNA. The method includes, at least, steps of forming an mRNA slurry, stirring the slurry, and vacuum or pressure filtering the slurry.

公开(公告)号：US11560562B2

公开(公告)日：2023-01-24

申请号：US15907163

申请日：2018-02-27

Applicant: Translate Bio, Inc.

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

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

Abstract: 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.