公开(公告)号：US12257305B2

公开(公告)日：2025-03-25

申请号：US17295621

申请日：2019-11-20

Applicant: Translate Bio, Inc.

Inventor： Frank DeRosa ,  Shrirang Karve ,  Yi Zhang

IPC: C07C215/24 ,  A61K9/1272 ,  A61K47/14 ,  A61K47/54 ,  C07C217/08 ,  C07C229/12

Abstract: The compounds disclosed herein compound of Formula (I), substructures thereof, and pharmaceutically acceptable salts thereof. 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：US20250025419A1

公开(公告)日：2025-01-23

申请号：US18762242

申请日：2024-07-02

Applicant: Translate Bio, Inc.

Inventor： Shrirang Karve ,  Zarna Patel ,  Yi Zhang ,  Ashish Sarode ,  Rebecca L. Goldman ,  Frank DeRosa ,  Michael Heartlein

IPC: A61K9/127 ,  A61K9/14 ,  A61K9/51 ,  A61K31/7105

Abstract: The present invention provides an improved process for lipid nanoparticle formulation and mRNA encapsulation. In some embodiments, the present invention provides a process of encapsulating messenger RNA (mRNA) in lipid nanoparticles comprising a step of mixing a solution of pre-formed lipid nanoparticles and mRNA at a low concentration.

公开(公告)号：US12195505B2

公开(公告)日：2025-01-14

申请号：US17693120

申请日：2022-03-11

Applicant: Translate Bio, Inc.

Inventor： Shrirang Karve ,  Frank DeRosa ,  Michael Heartlein ,  Zarna Patel

IPC: C07H21/04 ,  A61K9/00 ,  A61K9/127 ,  A61K9/19 ,  A61K9/51 ,  A61K45/06 ,  A61K47/26 ,  A61P11/00 ,  B82Y5/00 ,  C07K14/47 ,  A61K38/00

Abstract: The present invention provides, among other things, an improved method of treating cystic fibrosis (CF) in a human subject. The method comprises administering a composition comprising an mRNA encoding a Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) protein at a concentration of 0.5 mg/mL or greater to a human subject via nebulization. The composition is aerosolized using a nebulizer and a nominal dose of the mRNA is administered to the human subject via the nebulizer over a period of time, typically at least 30 minutes, at a suitable nebulization rate, e.g., at least 0.2 mL/minute.

公开(公告)号：US20240398720A1

公开(公告)日：2024-12-05

申请号：US18625655

申请日：2024-04-03

Applicant: Translate Bio, Inc.

Inventor： Frank DeRosa ,  Michael Heartlein ,  Shrirang Karve ,  Zarna Patel

IPC: A61K9/51 ,  A61K9/00 ,  A61K9/14 ,  A61K38/17 ,  A61K38/45 ,  C07D319/12 ,  C08G63/685 ,  C08G63/78 ,  C12N15/113

Abstract: Disclosed are cationic polymers comprising monomers such as those described in Formula (I), Such polymers can be useful for the preparation of therapeutic compositions (e.g., compositions comprising nucleic acids such as mRNA). Additionally, therapeutic compositions comprising these cationic, biodegradable polymers can have improved properties and reduced toxicity.

公开(公告)号：US11969480B2

公开(公告)日：2024-04-30

申请号：US17185587

申请日：2021-02-25

Applicant: Translate Bio, Inc.

Inventor： Shrirang Karve ,  Ashish Sarode ,  Frank DeRosa

IPC: C07H21/02 ,  A61K9/51 ,  A61K47/60 ,  A61K47/69 ,  A61K48/00 ,  B01F23/451 ,  B01F33/301 ,  B01J13/20 ,  B01F101/22

CPC classification number: A61K48/0091 ,  A61K9/5123 ,  A61K47/60 ,  A61K47/6925 ,  A61K48/0033 ,  B01F23/451 ,  B01F33/3017 ,  B01J13/203 ,  B01F2101/22

Abstract: The present invention provides an improved process for lipid nanoparticle formulation and mRNA encapsulation. In some embodiments, the present invention provides a process of encapsulating messenger RNA (mRNA) in lipid nanoparticles comprising a step of mixing a suspension of preformed lipid nanoparticles and mRNA.

公开(公告)号：US20240041789A1

公开(公告)日：2024-02-08

申请号：US18328206

申请日：2023-06-02

Applicant: Translate Bio, Inc.

Inventor： Shrirang Karve ,  Frank DeRosa ,  Zarna Bhavsar ,  Michael Heartlein

IPC: A61K9/51 ,  A61K38/45 ,  A61K38/18 ,  A61K38/17 ,  A61K38/44 ,  A61K38/53 ,  A61K9/127 ,  A61K47/22 ,  C12N15/88 ,  A61K47/28

CPC classification number: A61K9/5192 ,  A61K9/5123 ,  A61K38/45 ,  C12Y201/03003 ,  A61K38/1816 ,  C12Y603/04005 ,  A61K38/177 ,  A61K38/44 ,  C12Y114/16001 ,  A61K38/53 ,  A61K9/1272 ,  A61K47/22 ,  C12N15/88 ,  A61K47/28

Abstract: The present invention provides an improved process for lipid nanoparticle formulation and mRNA encapsulation. In some embodiments, the present invention provides a process of encapsulating messenger RNA (mRNA) in lipid nanoparticles comprising a step of mixing a solution of pre-formed lipid nanoparticles and mRNA.

公开(公告)号：US11692189B2

公开(公告)日：2023-07-04

申请号：US17835725

申请日：2022-06-08

Applicant: Translate Bio, Inc.

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

IPC: C12N15/10 ,  C07H21/02

CPC classification number: C12N15/10 ,  C07H21/02 ,  C12N15/1017

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 mRNA to a denaturing condition, and purifying the mRNA 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：US20230092238A1

公开(公告)日：2023-03-23

申请号：US17619040

申请日：2020-06-19

Applicant: Translate Bio, Inc.

Inventor： Shrirang Karve ,  Frank DeRosa

IPC: A61K9/127 ,  A61K38/52 ,  A61K38/45 ,  A61K48/00 ,  C07C229/12 ,  C07C233/18

Abstract: Disclosed are cationic lipids which are compounds of Formula (A). 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：US20230050672A1

公开(公告)日：2023-02-16

申请号：US17295621

申请日：2019-11-20

Applicant: Translate Bio, Inc.

Inventor： Frank DeRosa ,  Shrirang Karve ,  Yi Zhang

IPC: A61K47/14 ,  C07C229/12 ,  C07C215/24 ,  C07C217/08 ,  A61K47/54 ,  A61K9/127

Abstract: The compounds disclosed herein compound of Formula (I), substructures thereof, and pharmaceutically acceptable salts thereof. 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：US20230000781A1

公开(公告)日：2023-01-05

申请号：US17745428

申请日：2022-05-16

Applicant: Translate Bio, Inc.

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

IPC: A61K9/50 ,  A61P11/00 ,  A61K9/00 ,  A61K9/48 ,  A61K38/17 ,  A61K48/00 ,  A61K9/51 ,  A61K38/45

Abstract: The present invention provides stable, dry powder messenger RNA formulations for therapeutic use, and methods of making and using the same.