公开(公告)号：US20220016029A1

公开(公告)日：2022-01-20

申请号：US17291923

申请日：2019-11-07

Applicant: Translate Bio, Inc.

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

IPC: A61K9/127 ,  C08G65/333 ,  C08G65/332 ,  C08G65/335 ,  A61K45/06

Abstract: The compounds disclosed herein (e.g., compounds of Formula (I), (II), (III), and (IV)) comprise a lipid substructure comprising a hydrophobic moiety and a hydrophilic moiety; and two or more polymeric groups (e.g., two or more polyethylene glycol (PEG) groups). 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：US20210378977A1

公开(公告)日：2021-12-09

申请号：US17262421

申请日：2019-07-23

Applicant: Translate Bio, Inc.

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

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

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：US20210353556A1

公开(公告)日：2021-11-18

申请号：US17320946

申请日：2021-05-14

Applicant: Translate Bio, Inc.

Inventor： Shrirang Karve ,  Frank DeRosa ,  Natalia Vargas Montoya ,  Asad Khanmohammed

IPC: A61K9/51 ,  A61K31/713

Abstract: The present invention provides, among other things, methods of encapsulating messenger RNA in lipid nanoparticles without the use of flammable solvents, and compositions produced by these methods, for mRNA delivery in therapeutic use. The present invention is, in part, based on the surprising discovery that mRNA can be encapsulated with high efficiency, without using an ethanol solvent, in the presence of an amphiphilic polymer. Thus, the present invention provides safe, cost-effective, and efficient methods of producing LNP formulations from large scale manufacturing processes as well as in low volume formulations for therapeutic applications.

公开(公告)号：US20210324368A1

公开(公告)日：2021-10-21

申请号：US17103439

申请日：2020-11-24

Applicant: Translate Bio, Inc.

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

IPC: C12N15/10 ,  C07H21/02

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 rnRNA to a denaturing condition, and purifying the rnRNA 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：US20210220273A1

公开(公告)日：2021-07-22

申请号：US17058334

申请日：2019-05-30

Applicant: Translate Bio, Inc.

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

IPC: A61K9/127 ,  A61K9/00 ,  A61K39/385 ,  A61K38/45 ,  A61K38/52 ,  C07J41/00

Abstract: Disclosed are cationic lipids which are compounds of Formula I. 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：US20210196633A1

公开(公告)日：2021-07-01

申请号：US17002460

申请日：2020-08-25

Applicant: Translate Bio, Inc.

Inventor： Frank DeRosa ,  Michael Heartlein ,  Shrirang Karve

IPC: A61K9/127 ,  A61K48/00 ,  A61K38/17 ,  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：US20210187122A1

公开(公告)日：2021-06-24

申请号：US17016860

申请日：2020-09-10

Applicant: Translate Bio, Inc.

Inventor： Michael Heartlein ,  Frank DeRosa ,  Jonathan Cherry ,  Paula Lewis ,  Shrirang Karve ,  Zarna Patel ,  Darshan Parekh ,  Susan Wood ,  Chi-Sung Chiu ,  Caroline J. Woo

IPC: A61K48/00 ,  C12N15/62 ,  A61K38/02 ,  A61K9/127 ,  A61K38/17 ,  A61P21/00 ,  C12N15/52 ,  A61K9/00 ,  A61P43/00 ,  C12N15/88 ,  A61K31/7088 ,  A61K9/107

Abstract: The present invention provides methods and compositions of treating Friedreich's ataxia (FRDA) based on administering an mRNA encoding a frataxin protein.

公开(公告)号：US20210113483A1

公开(公告)日：2021-04-22

申请号：US16966214

申请日：2019-02-01

Applicant: Translate Bio, Inc.

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

IPC: A61K9/51 ,  C08G63/685 ,  C08G63/78 ,  C07D319/12 ,  A61K38/17 ,  A61K38/45 ,  A61K9/14 ,  A61K9/00 ,  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 m RNA). Additionally, therapeutic compositions comprising these cationic, biodegradable polymers can have improved properties and reduced toxicity.

公开(公告)号：US20210106527A1

公开(公告)日：2021-04-15

申请号：US17066919

申请日：2020-10-09

Applicant: Translate Bio, Inc.

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

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

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：US20200155691A1

公开(公告)日：2020-05-21

申请号：US16773638

申请日：2020-01-27

Applicant: Translate Bio, Inc.

Inventor： Frank DeRosa ,  Michael Heartlein ,  Shrirang Karve

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