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：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.

公开(公告)号：US20220218612A1

公开(公告)日：2022-07-14

申请号：US17611020

申请日：2020-05-14

Applicant: Translate Bio, Inc.

Inventor： Shrirang Karve ,  Frank DeRosa ,  Michael Hearlein ,  Ashish Sarode ,  Zarna Patel ,  Rebecca L. Ball ,  Natalia Vargas Montoya ,  Priyal Patel ,  Asad Khanmohammed

IPC: A61K9/127 ,  A61K9/00 ,  A61K31/7088 ,  A61K9/16

Abstract: The present invention provides an improved process for lipid nanoparticle formulation and mRNA encapsulation. In some embodiments, the present invention provides a process for enhanced encapsulation of messenger RNA (mRNA) in lipid nanoparticles comprising a step of heating the mRNA-encapsulated lipid nanoparticles in a drug product formulation solution.

公开(公告)号：US20220087935A1

公开(公告)日：2022-03-24

申请号：US17420346

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: A61K9/127 ,  A61K31/711 ,  A61P11/00

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：US20200022921A1

公开(公告)日：2020-01-23

申请号：US16520000

申请日：2019-07-23

Applicant: Translate Bio, Inc.

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

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

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

公开(公告)号：US20180333457A1

公开(公告)日：2018-11-22

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

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

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.