公开(公告)号：US20180258423A1

公开(公告)日：2018-09-13

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 ,  G01N27/447

CPC classification number: C12N15/1096 ,  C12N9/1247 ,  C12N15/1068 ,  C12P19/34 ,  C12Y207/07006 ,  G01N27/44778

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.

公开(公告)号：US20180256741A1

公开(公告)日：2018-09-13

申请号：US15907131

申请日：2018-02-27

Applicant: Translate Bio, Inc.

Inventor： Anusha Dias ,  Jonathan Abysalh ,  Brian Bettencourt ,  Frank DeRosa ,  Michael Heartlein

IPC: A61K47/64 ,  A61P11/00 ,  A61K31/7105 ,  A61K47/69 ,  A61K9/127 ,  A61K9/00

Abstract: The present invention provides, among other things, improved methods and pharmaceutical compositions for treating cystic fibrosis based on codon optimized mRNA encoding a Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) protein.

公开(公告)号：US20180251755A1

公开(公告)日：2018-09-06

申请号：US15906864

申请日：2018-02-27

Applicant: Translate Bio, Inc.

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

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

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：US20180251754A1

公开(公告)日：2018-09-06

申请号：US15907086

申请日：2018-02-27

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：US20250101481A1

公开(公告)日：2025-03-27

申请号：US18794053

申请日：2024-08-05

Applicant: Translate Bio, Inc.

Inventor： Joseph Parella ,  Kimberly Gillis ,  Jonathan Abysalh ,  Travis Jeannotte ,  Erik Held ,  Frank DeRosa ,  Michael Heartlein ,  Rebecca Powell

IPC: C12P19/34 ,  B01D25/00 ,  B01D37/00 ,  C07H1/06 ,  C07H21/02 ,  C07H21/04 ,  C12N15/10 ,  C12N15/52

Abstract: The present invention provides, among other things, methods for purifying mRNA based on normal flow filtration for therapeutic use.

公开(公告)号：US20240299489A9

公开(公告)日：2024-09-12

申请号：US17449679

申请日：2021-10-01

Applicant: Translate Bio, Inc.

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

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

CPC classification number: A61K38/1709 ,  A61K9/0078 ,  A61K9/127 ,  A61K48/0075 ,  A61P11/12 ,  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：US20240018514A1

公开(公告)日：2024-01-18

申请号：US18065952

申请日：2022-12-14

Applicant: Translate Bio, Inc.

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

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

CPC classification number: C12N15/1096 ,  C12Y207/07006 ,  G01N27/44778 ,  C12N9/1247 ,  C12N15/1068 ,  C12P19/34

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.

公开(公告)号：US11453877B2

公开(公告)日：2022-09-27

申请号：US16875418

申请日：2020-05-15

Applicant: Translate Bio, Inc.

Inventor： Jonathan Abysalh ,  Frank DeRosa ,  Jorel E. Vargas ,  Cameron M. Smith

IPC: C12N15/10

Abstract: The present invention provides, among other things, methods for purifying high quality messenger RNA (mRNA) suitable for clinical use, without using any caustic or flammable solvents. The present invention is, in part, based on surprising discovery that mRNA can be successfully purified by selective precipitation and washing without using ethanol while maintaining integrity and high purity of mRNA. Thus, the present invention provides an effective, reliable, and safer method of purifying RNA from large scale manufacturing process therapeutic applications without using any caustic or inflammable solvents.

公开(公告)号：US20220160827A1

公开(公告)日：2022-05-26

申请号：US17449679

申请日：2021-10-01

Applicant: Translate Bio, Inc.

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

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

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：US20220136022A1

公开(公告)日：2022-05-05

申请号：US17502816

申请日：2021-10-15

Applicant: Translate Bio, Inc.

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

IPC: C12P19/34 ,  A61K31/713

Abstract: 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.