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.

公开(公告)号：US20180263918A1

公开(公告)日：2018-09-20

申请号：US15979054

申请日：2018-05-14

Applicant: Translate Bio, Inc.

Inventor： Frank DeRosa ,  Shrirang Karve ,  Michael Heartlein

IPC: A61K9/50 ,  A61K9/127 ,  A61K31/713

CPC classification number: A61K9/5089 ,  A61K9/1271 ,  A61K9/1272 ,  A61K9/1277 ,  A61K9/5015 ,  A61K31/713

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 mRNA solution and a lipid solution, wherein the mRNA solution and/or the lipid solution are at a pre-determined temperature greater than ambient temperature.

公开(公告)号：US20180125989A1

公开(公告)日：2018-05-10

申请号：US15809605

申请日：2017-11-10

Applicant: Translate Bio, Inc.

Inventor： Frank DeRosa ,  Michael Heartlein ,  Shrirang Karve

IPC: A61K47/54 ,  A61K9/00 ,  A61K48/00 ,  A61K38/17 ,  A61K47/60 ,  A61K47/69 ,  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：US20180028445A1

公开(公告)日：2018-02-01

申请号：US15676570

申请日：2017-08-14

Applicant: Translate Bio, Inc.

Inventor： Frank DeRosa ,  Michael Heartlein ,  Shrirang Karve

IPC: A61K9/127 ,  A61K38/17 ,  A61K48/00 ,  C12N15/88

Abstract: The present invention provides, among other things, methods and compositions for effective delivery of messenger RNA (mRNA) to the central nervous system (CNS). In particular, the present invention provides methods and compositions for administering intrathecally to a subject in need of delivery a composition comprising an mRNA encoding a protein, encapsulated within a liposome, such that the administering of the composition results in the intracellular delivery of mRNA in neurons in the brain and/or spinal cord. The present invention is particularly useful for the treatment of CNS diseases, disorders or conditions, such as spinal muscular atrophy.

公开(公告)号：US09850269B2

公开(公告)日：2017-12-26

申请号：US14696140

申请日：2015-04-24

Applicant: TRANSLATE BIO, INC.

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

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

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：US20250057765A1

公开(公告)日：2025-02-20

申请号：US18782767

申请日：2024-07-24

Applicant: Translate Bio, Inc.

Inventor： Christian Cobaugh ,  Richard Wooster ,  Frank DeRosa ,  Anusha Dias ,  Shrirang Karve

IPC: A61K9/127 ,  A61K9/00 ,  A61K38/17 ,  A61K38/19 ,  A61K38/20 ,  A61K45/06 ,  A61P35/00

Abstract: The present invention provides, among other things, methods and compositions for cancer treatment. The methods and compositions disclosed herein are particularly effective in reducing the size/volume of a tumor and inhibiting tumor growth.

公开(公告)号：US12201677B2

公开(公告)日：2025-01-21

申请号：US17094327

申请日：2020-11-10

Applicant: Translate Bio, Inc.

Inventor： Frank DeRosa ,  Michael Heartlein ,  Lianne Smith ,  Shrirang Karve

IPC: A61K38/45 ,  A61K9/00 ,  A61K9/127 ,  A61K48/00 ,  C12N9/10

Abstract: The present invention provides, among other things, methods of treating ornithine transcarbamylase (OTC) deficiency, including administering to a subject in need of treatment a composition comprising an mRNA encoding an ornithine transcarbamylase (OTC) protein at an effective dose and an administration interval such that at least one symptom or feature of the OTC deficiency is reduced in intensity, severity, or frequency or has delayed 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：US20240408013A1

公开(公告)日：2024-12-12

申请号：US18664925

申请日：2024-05-15

Applicant: Translate Bio, Inc.

Inventor： Frank DeRosa ,  Michael Heartlein ,  Shrirang Karve

IPC: A61K9/127 ,  A61K38/17 ,  A61K48/00 ,  A61P25/00 ,  C07H21/02 ,  C12N15/88

Abstract: The present invention provides, among other things, methods and compositions for effective delivery of messenger RNA (mRNA) to the central nervous system (CNS). In particular, the present invention provides methods and compositions for administering intrathecally to a subject in need of delivery a composition comprising an mRNA encoding a protein, encapsulated within a liposome, such that the administering of the composition results in the intracellular delivery of mRNA in neurons in the brain and/or spinal cord. The present invention is particularly useful for the treatment of CNS diseases, disorders or conditions, such as spinal muscular atrophy.

公开(公告)号：US12144871B2

公开(公告)日：2024-11-19

申请号：US16936909

申请日：2020-07-23

Applicant: Translate Bio, Inc.

Inventor： Shrirang Karve ,  Frank DeRosa ,  Michael Heartlein ,  Natalia Vargas Montoya ,  Priyal Patel ,  Ashish Sarode

IPC: A61K48/00 ,  A61K9/107 ,  A61K47/32 ,  A61K47/34

Abstract: The present invention provides an improved compositions and processes for preparing mRNA-loaded lipid nanoparticles (mRNA-LNPs). In some embodiments, the present invention provides mRNA-LNPs with exceptional stability and is particularly useful in cases where LNPs comprising low or no PEG-modified lipids are desired.