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：US20240299489A9

公开(公告)日：2024-09-12

申请号：US17449679

申请日：2021-10-01

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 ,  A61K48/00 ,  A61P11/12

CPC classification number: A61K38/1709 ,  A61K9/0078 ,  A61K9/127 ,  A61K48/0075 ,  A61P11/12 ,  C12N15/88

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：US20220395459A1

公开(公告)日：2022-12-15

申请号：US17737712

申请日：2022-05-05

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：US20220160827A1

公开(公告)日：2022-05-26

申请号：US17449679

申请日：2021-10-01

Applicant: Translate Bio, Inc.

Inventor： Michael Heartlein ,  Frank DeRosa ,  Alan Kimura ,  Jonathan Abysalh ,  Anusha Dias ,  Shrirang Karve ,  Zarna Patel

IPC: A61K38/17 ,  A61P11/12 ,  A61K9/127 ,  A61K9/00 ,  A61K48/00

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：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：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：US12064515B2

公开(公告)日：2024-08-20

申请号：US17737712

申请日：2022-05-05

Applicant: Translate Bio, Inc.

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

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

CPC classification number: A61K9/1277 ,  A61K9/141 ,  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：US20220347307A1

公开(公告)日：2022-11-03

申请号：US17705803

申请日：2022-03-28

Applicant: Translate Bio, Inc.

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

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

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.