公开(公告)号：US10172808B2

公开(公告)日：2019-01-08

申请号：US15995889

申请日：2018-06-01

Applicant: ModernaTX, Inc.

Inventor： Joshua Frederick ,  Ailin Bai ,  Vladimir Presnyak ,  Stephen Hoge ,  Kerry Benenato ,  Iain McFadyen ,  Ellalahewage Sathyajith Kumarasinghe ,  Susannah Hewitt

IPC: A61K48/00 ,  A61K31/7115 ,  A61K9/51 ,  A61K38/20 ,  A61K38/17 ,  C07K14/54 ,  C07K14/705 ,  A61K39/395 ,  A61K9/00 ,  A61P35/00 ,  A61K39/00

Abstract: The disclosure relates to compositions and methods for the preparation, manufacture and therapeutic use of combinations of immunomodulatory polynucleotides (e.g., mRNAs) encoding an immune response primer polypeptide (e.g., an interleukin 23 (IL-23) polypeptide or an interleukin 36γ (IL-36-gamma) polypeptide), and an immune response co-stimulatory signal polypeptide (e.g., an OX40L polypeptide).

公开(公告)号：US20180371047A1

公开(公告)日：2018-12-27

申请号：US16025302

申请日：2018-07-02

Applicant: ModernaTX, Inc.

Inventor： Barry Ticho ,  Nadege Briancon-Eris ,  Zhinan Xia ,  Athanasios Dousis ,  Seymour de Picciotto ,  Vladimir Presnyak ,  Stephen Hoge ,  Iain Mcfadyen ,  Kerry Benenato ,  Ellalahewage Sathyajith Kumarasinghe

IPC: C07K14/64 ,  A61P9/00 ,  C12N15/62 ,  C12N15/52 ,  A61K47/69 ,  C07K16/26 ,  A61K38/17

Abstract: The invention relates to mRNA therapy for the treatment of fibrosis and/or cardiovascular disease. mRNAs for use in the invention, when administered in vivo, encode human relaxin, isoforms thereof, functional fragments thereof, and fusion proteins comprising relaxin. mRNAs of the invention are preferably encapsulated in lipid nanoparticles (LNPs) to effect efficient delivery to cells and/or tissues in subjects, when administered thereto. mRNA therapies of the invention increase and/or restore deficient levels of relaxin expression and/or activity in subjects. mRNA therapies of the invention further decrease levels of toxic metabolites associated with deficient relaxin activity in subjects.

公开(公告)号：US12252704B2

公开(公告)日：2025-03-18

申请号：US17154309

申请日：2021-01-21

Applicant: ModernaTX, Inc.

Inventor： Paolo Martini ,  Stephen Hoge ,  Kerry Benenato ,  Vladimir Presnyak ,  Iain McFadyen ,  Ellalahewage Sathyajith Kumarasinghe ,  Ding An ,  Staci Sabnis

IPC: A61K9/51 ,  A61K48/00 ,  B82Y5/00 ,  C07K14/14 ,  C12N9/10 ,  C12N9/12 ,  C12N15/88 ,  G01N33/68

Abstract: The invention relates to mRNA therapy for the treatment of galactosemia type 1 (Gal-1). mRNAs for use in the invention, when administered in vivo, encode human galactose-1-phosphate uridylyltransferase (GALT), isoforms thereof, functional fragments thereof, and fusion proteins comprising GALT. mRNAs of the invention are preferably encapsulated in lipid nanoparticles (LNPs) to effect efficient delivery to cells and/or tissues in subjects, when administered thereto. mRNA therapies of the invention increase and/or restore deficient levels of GALT expression and/or activity in subjects. mRNA therapies of the invention further decrease levels of toxic metabolites associated with deficient GALT activity in subjects, namely galactose-1-phosphate (Gal-1-P).

公开(公告)号：US12241063B2

公开(公告)日：2025-03-04

申请号：US17933500

申请日：2022-09-20

Applicant: ModernaTX, Inc.

Inventor： Melissa J. Moore ,  Caroline Köhrer ,  Ruchi Jain ,  Vladimir Presnyak

IPC: C07H21/02 ,  A61K47/69 ,  C12N15/11 ,  C12N15/85 ,  C12N15/88

Abstract: The present disclosure provides messenger RNAs (mRNAs) having chemical and/or structural modifications, including RNA elements and/or modified nucleotides, which provide a desired translational regulatory activity to the mRNA.

公开(公告)号：US12103955B2

公开(公告)日：2024-10-01

申请号：US16944014

申请日：2020-07-30

Applicant: MODERNATX, INC.

Inventor： Barry Ticho ,  Nadege Briancon-Eris ,  Zhinan Xia ,  Athanasios Dousis ,  Seymour de Picciotto ,  Vladimir Presnyak ,  Stephen Hoge ,  Iain Mcfadyen ,  Kerry Benenato ,  Ellalahewage Sathyajith Kumarasinghe

IPC: C12N15/62 ,  A61K31/7105 ,  A61K38/17 ,  A61K47/14 ,  A61K47/69 ,  A61K48/00 ,  A61P9/00 ,  C07K14/64 ,  C07K16/26 ,  C12N15/52 ,  A61K9/00 ,  A61K9/127 ,  A61K38/00

CPC classification number: C07K14/64 ,  A61K31/7105 ,  A61K38/1796 ,  A61K47/6929 ,  A61P9/00 ,  C07K16/26 ,  C12N15/52 ,  C12N15/62 ,  A61K9/0019 ,  A61K9/127 ,  A61K38/00 ,  C07K2319/00 ,  C07K2319/31

Abstract: The invention relates to mRNA therapy for the treatment of fibrosis and/or cardiovascular disease. mRNAs for use in the invention, when administered in vivo, encode human relaxin, isoforms thereof, functional fragments thereof, and fusion proteins comprising relaxin. mRNAs of the invention are preferably encapsulated in lipid nanoparticles (LNPs) to effect efficient delivery to cells and/or tissues in subjects, when administered thereto. mRNA therapies of the invention increase and/or restore deficient levels of relaxin expression and/or activity in subjects. mRNA therapies of the invention further decrease levels of toxic metabolites associated with deficient relaxin activity in subjects.

公开(公告)号：US20240024506A1

公开(公告)日：2024-01-25

申请号：US18327728

申请日：2023-06-01

Applicant: ModernaTX, Inc.

Inventor： Lei Jiang ,  Lin Tung Guey ,  Paolo G. V. Martini ,  Vladimir Presnyak

IPC: C12N9/00

CPC classification number: C12N9/93 ,  C12Y604/01003

Abstract: This disclosure relates to mRNA therapy for the treatment of propionic acidemia (PA). mRNAs for use in the invention, when administered in vivo, encode human propionyl-CoA carboxylase alpha (PCCA) and/or human propionyl-CoA carboxylase beta (PCCB), and isoforms thereof, functional fragments thereof, and fusion proteins comprising PCCA and/or PCCB. mRNAs of the invention are preferably encapsulated in lipid nanoparticles (LNPs) to effect efficient delivery to cells and/or tissues in subjects, when administered thereto. mRNA therapies of the invention increase and/or restore deficient levels of propionyl-CoA carboxylase (PCC) expression and/or activity in subjects. mRNA therapies of the invention further decrease levels of disease-associated toxic metabolites associated with deficient PCCA or PCCB activity, in subjects.

公开(公告)号：US11504337B2

公开(公告)日：2022-11-22

申请号：US16515256

申请日：2019-07-18

Applicant: ModernaTX, Inc.

Inventor： Paolo Martini ,  Vladimir Presnyak ,  Kerry Benenato ,  Stephen Hoge ,  Iain McFadyen ,  Ellalahewage Sathyajith Kumarasinghe

IPC: C12N15/11 ,  A61K9/51 ,  C12N9/90 ,  A61K9/127 ,  A61P3/00 ,  A61P43/00 ,  A61K38/52 ,  A61K48/00 ,  C12N15/52 ,  A61K38/00

Abstract: The disclosure relates to polynucleotides comprising an open reading frame of linked nucleosides encoding human methylmalonyl-CoA mutase precursor, human methylmalonyl-CoA mutase (MCM) mature form, or functional fragments thereof. In some embodiments, the disclosure includes methods of treating methylmalonic acidemia in a subject in need thereof comprising administering an mRNA encoding an MCM polypeptide.

公开(公告)号：US20220152225A1

公开(公告)日：2022-05-19

申请号：US17279697

申请日：2019-09-26

Applicant: ModernaTX, Inc.

Inventor： Paolo G. V. Martini ,  Vladimir Presnyak

IPC: A61K48/00 ,  A61P3/00

Abstract: This disclosure relates to mRNA therapy for the treatment of arginase deficiency (AD). mRNAs for use in the invention, when administered in vivo, encode arginase 1 (ARG1). mRNA therapies of the disclosure increase and/or restore deficient levels of ARG1 expression and/or activity in subjects. mRNA therapies of the disclosure further decrease abnormal accumulation of ammonia associated with deficient ARG1 activity in subjects.

公开(公告)号：US11185510B2

公开(公告)日：2021-11-30

申请号：US16543102

申请日：2019-08-16

Applicant: ModernaTX, Inc.

Inventor： Joshua P. Frederick ,  Ailin Bai ,  Vladimir Presnyak ,  Stephen G. Hoge ,  Kerry Benenato ,  Iain McFadyen ,  Ellalahewage Sathyajith Kumarasinghe ,  Susannah Hewitt

IPC: A61K38/20 ,  A61K9/51 ,  A61K48/00 ,  A61K38/17 ,  A61K9/00 ,  A61K39/39 ,  A61K45/06 ,  A61K9/127 ,  C07K14/54 ,  C07K14/545 ,  C07K14/705 ,  A61K31/7088 ,  A61K31/7115 ,  A61P35/00 ,  A61K39/395 ,  A61K39/00 ,  B82Y5/00 ,  C12N15/88

Abstract: The disclosure relates to compositions and methods for the preparation, manufacture and therapeutic use of combinations of immunomodulatory polynucleotides (e.g., mRNAs) encoding an immune response primer polypeptide (e.g., an interleukin 23 (IL-23) polypeptide or an interleukin 36γ (IL-36-gamma) polypeptide), and an immune response co-stimulatory signal polypeptide (e.g., an OX40L polypeptide).

公开(公告)号：US20210269830A1

公开(公告)日：2021-09-02

申请号：US17154309

申请日：2021-01-21

Applicant: ModernaTX, Inc.

Inventor： Paolo Martini ,  Stephen Hoge ,  Kerry Benenato ,  Vladimir Presnyak ,  Iain McFadyen ,  Ellalahewage Sathyajith Kumarasinghe ,  Ding An ,  Staci Sabnis

IPC: C12N15/88 ,  A61K9/51 ,  B82Y5/00 ,  C07K14/14 ,  C12N9/12 ,  G01N33/68 ,  A61K48/00

Abstract: The invention relates to mRNA therapy for the treatment of galactosemia type 1 (Gal-1). mRNAs for use in the invention, when administered in vivo, encode human galactose-1-phosphate uridylyltransferase (GALT), isoforms thereof, functional fragments thereof, and fusion proteins comprising GALT. mRNAs of the invention are preferably encapsulated in lipid nanoparticles (LNPs) to effect efficient delivery to cells and/or tissues in subjects, when administered thereto. mRNA therapies of the invention increase and/or restore deficient levels of GALT expression and/or activity in subjects. mRNA therapies of the invention further decrease levels of toxic metabolites associated with deficient GALT activity in subjects, namely galactose-1-phosphate (Gal-1-P).