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：US11013812B2

公开(公告)日：2021-05-25

申请号：US16599928

申请日：2019-10-11

Applicant: Translate Bio, Inc.

Inventor： Frank DeRosa ,  Michael Heartlein ,  Shrirang Karve

IPC: A61K47/54 ,  A61K9/00 ,  A61K48/00 ,  A61K47/69 ,  A61K47/60 ,  A61K38/17 ,  A61P11/12 ,  A61K9/127 ,  A61K47/28

Abstract: The present invention provides, among other things, compositions and methods of formulating nucleic acid-containing nanoparticles comprising no more than three distinct lipids components, one distinct lipid component being a sterol-based cationic lipid. In some embodiments, the present invention provides compositions and methods in which the lipid nanoparticles further comprise helper lipids and PEG-modified lipids. The resulting formulation comprises a high encapsulation percentage for nucleic acids.

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：US10940207B2

公开(公告)日：2021-03-09

申请号：US16773638

申请日：2020-01-27

Applicant: Translate Bio, Inc.

Inventor： Frank DeRosa ,  Michael Heartlein ,  Shrirang Karve

IPC: A61K47/54 ,  A61K38/17 ,  A61K47/28 ,  A61K47/60 ,  A61K47/69 ,  A61K48/00 ,  A61K9/00 ,  A61K9/127 ,  A61P11/12

Abstract: The present invention provides, among other things, compositions and methods of formulating nucleic acid-containing nanoparticles comprising no more than three distinct lipids components, one distinct lipid component being a sterol-based cationic lipid. In some embodiments, the present invention provides compositions and methods in which the lipid nanoparticles further comprise helper lipids and PEG-modified lipids. The resulting formulation comprises a high encapsulation percentage for nucleic acids.

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：US20200016274A1

公开(公告)日：2020-01-16

申请号：US16425693

申请日：2019-05-29

Applicant: Translate Bio, Inc.

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

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

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：US20190249191A1

公开(公告)日：2019-08-15

申请号：US16280772

申请日：2019-02-20

Applicant: Translate Bio, Inc.

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

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

CPC classification number: C12N15/85 ,  A61K31/7115 ,  A61K31/7125 ,  A61K38/177 ,  A61K38/1816 ,  A61K38/44 ,  A61K38/45 ,  A61K48/005 ,  C07H21/02 ,  C07K14/505 ,  C07K14/705 ,  C12N9/0071 ,  C12N9/1018 ,  C12N15/67 ,  C12N2830/50 ,  C12P21/00 ,  C12Y114/16001 ,  C12Y201/03003

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：US10138213B2

公开(公告)日：2018-11-27

申请号：US14749027

申请日：2015-06-24

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：US12280147B2

公开(公告)日：2025-04-22

申请号：US17291919

申请日：2019-11-07

Applicant: Translate Bio, Inc.

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

IPC: A61K9/127 ,  A61K9/1273 ,  A61K38/17 ,  A61K38/45 ,  C08G65/48

Abstract: 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.

公开(公告)号：US12268754B2

公开(公告)日：2025-04-08

申请号：US17448996

申请日：2021-09-27

Applicant: Translate Bio, Inc.

Inventor： Kim Askew ,  Jou-Ku Chung ,  Frank DeRosa ,  Michael Heartlein ,  Shrirang Karve ,  Thomas McCauley ,  Lianne Smith ,  Ann J. Barbier

IPC: A61K48/00 ,  A61K9/00 ,  A61K9/127 ,  A61K9/1272 ,  A61K38/45 ,  A61K47/69 ,  A61P3/00 ,  C12N15/52 ,  C12N15/85

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