公开(公告)号：US20250090684A1

公开(公告)日：2025-03-20

申请号：US18292058

申请日：2022-07-26

Applicant: ModernaTX, Inc.

Inventor： Husain Attarwala ,  Jingsong Cao ,  Patrick Finn ,  Simone Mori

IPC: A61K48/00 ,  A61K38/46 ,  A61P3/08 ,  C12N9/16

Abstract: This disclosure relates to mRNA therapy for the treatment of glycogen storage disease type 1a (GSD1a). mRNAs for use in the invention, when administered in vivo, encode glucose-6-phosphatase (G6PC). mRNA therapies of the disclosure increase and/or restore deficient levels of G6PC expression and/or activity in subjects.

公开(公告)号：US11939601B2

公开(公告)日：2024-03-26

申请号：US16765656

申请日：2018-11-21

Applicant: ModernaTX, Inc.

Inventor： Raj Rajendran ,  Patrick Finn ,  Paolo G. V. Martini ,  Ding An ,  Athanasios Dousis ,  Kanchana Ravichandran

IPC: A61P3/00 ,  A61K9/00 ,  A61K9/51 ,  A61K48/00 ,  C12N9/02

CPC classification number: C12N9/0071 ,  A61K9/0019 ,  A61K9/5123 ,  A61K48/0075 ,  A61P3/00 ,  C12Y114/16001

Abstract: This disclosure relates to mRNA therapy for the treatment of hyperphenylalaninemias such as phenylketonuria (PKU). mRNAs for use in the invention, when administered in vivo, encode human phenylalanine hydroxylase (PAH), functional fragments thereof (e.g., those comprising the catalytic domain or the catalytic domain and the tetramerization domains), and fusion proteins comprising PAH. 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 PAH expression and/or activity in subjects. mRNA therapies of the invention further decrease abnormal accumulation of phenylalanine associated with deficient PAH activity in subjects.

公开(公告)号：US11859215B2

公开(公告)日：2024-01-02

申请号：US16765604

申请日：2018-11-21

Applicant: ModernaTX, Inc.

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

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

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：US20230235298A1

公开(公告)日：2023-07-27

申请号：US17928680

申请日：2021-06-01

Applicant: ModernaTX, Inc.

Inventor： Lisa M. Rice ,  Patrick Finn ,  Athanasios Dousis

IPC: C12N9/02 ,  C12N15/63 ,  A61P3/00 ,  A61K9/127

CPC classification number: C12N9/0071 ,  C12Y114/16001 ,  C12N15/63 ,  A61P3/00 ,  A61K9/1271

Abstract: Variant phenylalanine hydroxylase polypeptides having substitutions at selected amino acid residues are disclosed. Also disclosed are methods of using variant phenylalanine hydroxylase polypeptides, or polynucleotides encoding variant phenylalanine hydroxylase polypeptides, to treat disorders such as phenylketonuria.

公开(公告)号：US20200299652A1

公开(公告)日：2020-09-24

申请号：US16765604

申请日：2018-11-21

Applicant: ModernaTX, Inc.

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

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

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：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：US20210040456A1

公开(公告)日：2021-02-11

申请号：US16765656

申请日：2018-11-21

Applicant: ModernaTX, Inc.

Inventor： Raj Rajendran ,  Patrick Finn ,  Paolo G.V. Martini ,  Ding An ,  Athanasios Dousis ,  Kanchana Ravichandran

IPC: C12N9/02 ,  A61K9/00 ,  A61K48/00 ,  A61K9/51 ,  A61P3/00

Abstract: This disclosure relates to mRNA therapy for the treatment of hyperphenylalaninemias such as phenylketonuria (PKU). mRNAs for use in the invention, when administered in vivo, encode human phenylalanine hydroxylase (PAH), functional fragments thereof (e.g., those comprising the catalytic domain or the catalytic domain and the tetramerization domains), and fusion proteins comprising PAH. 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 PAH expression and/or activity in subjects. mRNA therapies of the invention further decrease abnormal accumulation of phenylalanine associated with deficient PAH activity in subjects.