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.

公开(公告)号：US20200268666A1

公开(公告)日：2020-08-27

申请号：US16621514

申请日：2018-06-13

Applicant: ModernaTX, Inc. ,  Seattle Children's Hospital d/b/a Seattle Children's Research Institute

Inventor： Raj Rajendran ,  Paolo G. V. Martini ,  Carol H. Miao

IPC: A61K9/127 ,  A61K9/16 ,  A61K38/37

Abstract: The invention relates to mRNA therapy for the treatment of Hemophilia A. mRNAs for use in the invention, when administered in vivo, encode Factor VIII, isoforms thereof, functional fragments thereof, and fusion proteins comprising Factor VIII. 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 Factor VIII expression and/or activity in subjects. mRNA therapies of the invention further decrease levels of toxic metabolites associated with deficient Factor VII I 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.