公开(公告)号：US12239742B2

公开(公告)日：2025-03-04

申请号：US17154325

申请日：2021-01-21

Applicant: ModernaTX, Inc.

Inventor： Paolo Martini ,  Stephen Hoge ,  Kerry Benenato ,  Vladimir Presnyak ,  Iain McFadyen ,  Ellalahewage Sathyajith Kumarasinghe ,  Jingsong Cao ,  Lin Tung Guey ,  Staci Sabnis

IPC: C12N15/11 ,  A61K9/00 ,  A61K9/51 ,  A61P13/00 ,  C07K14/47 ,  A61K38/00

Abstract: The invention relates to mRNA therapy for the treatment of Citrullinemia Type 2 (“CTLN2”). mRNAs for use in the invention, when administered in vivo, encode human Citrin, isoforms thereof, functional fragments thereof, and fusion proteins comprising Citrin. 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 Citrin expression and/or activity in subjects. mRNA therapies of the invention further decrease levels of biomarkers associated with deficient Citrin activity in subjects, namely ammonia and/or triglycerides.

公开(公告)号：US20240247239A1

公开(公告)日：2024-07-25

申请号：US18510740

申请日：2023-11-16

Applicant: ModernaTX, Inc.

Inventor： Zhijian Zhuo ,  Andrea Lea Frassetto ,  Paolo G.V. Martini ,  Vladimir Presnyak ,  Patrick Finn

IPC: C12N9/10 ,  A61K9/00 ,  A61K9/51 ,  A61P7/00

CPC classification number: C12N9/1018 ,  A61K9/0019 ,  A61K9/5123 ,  A61P7/00 ,  C12Y201/03003

Abstract: This disclosure relates mRNA therapy for the treatment of ornithine transcarbamylase deficiency (OTCD). mRNAs for use in the invention, when administered in vivo, encode human ornithine transcarbamylase (OTC), isoforms thereof, functional fragments thereof, and fusion proteins comprising OTC. 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 OTC expression and/or activity in subjects. mRNA therapies of the invention further decrease levels of toxic ammonia associated with deficient OTC activity in subjects.

公开(公告)号：US11801227B2

公开(公告)日：2023-10-31

申请号：US16302370

申请日：2017-05-18

Applicant: ModernaTX, Inc.

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

IPC: A61K38/18 ,  A61K9/51 ,  A61K9/00 ,  A61K9/127 ,  C07K14/705 ,  C07K14/47 ,  A61P11/00 ,  A61K38/17 ,  C12N15/88

CPC classification number: A61K9/5123 ,  A61K9/0043 ,  A61K9/0053 ,  A61K9/0073 ,  A61K38/177 ,  A61P11/00 ,  C07K14/47 ,  C12N15/88 ,  A61K9/1271

Abstract: The invention relates to mRNA therapy for the treatment of cystic fibrosis. mRNAs for use in the invention, when administered in vivo, encode cystic fibrosis transmembrane conductance regulator (CFTR), isoforms thereof, functional fragments thereof, and fusion proteins comprising CFTR. 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 CFTR expression and/or activity in subjects. mRNA therapies of the invention further decrease levels of toxic metabolites associated with deficient CFTR activity in subjects.

公开(公告)号：US11596609B2

公开(公告)日：2023-03-07

申请号：US17930369

申请日：2022-09-07

Applicant: ModernaTX, Inc.

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

IPC: A61K9/51 ,  A61K48/00 ,  A61K38/17 ,  A61K38/20 ,  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：US20220265856A1

公开(公告)日：2022-08-25

申请号：US16765632

申请日：2018-11-21

Applicant: ModernaTX, Inc.

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

IPC: A61K48/00 ,  A61K38/53 ,  A61K9/127 ,  A61P3/00

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：US20220243182A1

公开(公告)日：2022-08-04

申请号：US17275082

申请日：2019-09-12

Applicant: ModernaTX, Inc.

Inventor： Paolo G. V. Martini ,  Vladimir Presnyak ,  Paloma Hoban Giangrande ,  Kimberly Ann Coughlan

IPC: C12N9/02 ,  A61K9/51 ,  A61P3/00 ,  C12N9/10

Abstract: This disclosure relates to mRNA therapy for the treatment of maple syrup urine disease (MSUD). mRNAs for use in the invention, when administered in vivo, encode branched chain α-ketoacid dehydrogenase complex (BCKDC) E1α, E1β, or E2mRNA therapies of the disclosure increase and/or restore deficient levels of E1α, E1β, or E2 expression and/or BCKDC activity in subjects. mRNA therapies of the invention further decrease abnormal accumulation of branched chain amino acids associated with deficient BCKDC activity in subjects.

公开(公告)号：US20210228707A1

公开(公告)日：2021-07-29

申请号：US17000215

申请日：2020-08-21

Applicant: ModernaTX, Inc.

Inventor： Mihir Metkar ,  Vladimir Presnyak ,  Guillaume Stewart-Jones

IPC: A61K39/215 ,  C07H21/02 ,  A61K9/16

Abstract: The disclosure relates to coronavirus ribonucleic acid (RNA) vaccines as well as methods of using the vaccines and compositions comprising the vaccines.

公开(公告)号：US10322091B2

公开(公告)日：2019-06-18

申请号：US16219418

申请日：2018-12-13

Applicant: ModernaTX, Inc.

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

IPC: A61K48/00 ,  A61K9/51 ,  A61K38/17 ,  A61K38/20 ,  A61K9/00 ,  A61K39/39 ,  A61K45/06 ,  A61K9/127 ,  C07K14/54 ,  C07K14/545 ,  C07K14/705 ,  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：US20190105281A1

公开(公告)日：2019-04-11

申请号：US16222155

申请日：2018-12-17

Applicant: ModernaTX, Inc.

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

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

CPC classification number: A61K9/5123 ,  A61K9/00 ,  A61K9/0019 ,  A61K9/1272 ,  A61K9/5146 ,  A61K38/177 ,  A61K38/1774 ,  A61K38/20 ,  A61K38/2006 ,  A61K38/208 ,  A61K38/2086 ,  A61K39/39 ,  A61K39/3955 ,  A61K45/06 ,  A61K48/00 ,  A61K48/005 ,  A61K2039/505 ,  A61K2039/51 ,  A61K2039/53 ,  A61K2039/54 ,  A61K2039/545 ,  A61K2039/585 ,  A61P35/00 ,  B82Y5/00 ,  C07K14/54 ,  C07K14/5434 ,  C07K14/5443 ,  C07K14/5446 ,  C07K14/545 ,  C07K14/70575 ,  C07K2319/00 ,  C12N15/88 ,  A61K2300/00

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：US20190022019A1

公开(公告)日：2019-01-24

申请号：US16113814

申请日：2018-08-27

Applicant: ModernaTX, Inc.

Inventor： Paolo Martini ,  Vladimir Presnyak

IPC: A61K9/51 ,  A61P3/00 ,  C12N9/90

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.