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：US20200085916A1

公开(公告)日：2020-03-19

申请号：US16302339

申请日：2017-05-18

Applicant: ModernaTX, Inc. ,  Fundacion Para La Investigacion Medica Aplicada

Inventor： Paolo Martini ,  Stephen Hoge ,  Kerry Benenato ,  Vladimir Presnyak ,  Lei Jiang ,  Iain McFadyen ,  Ellalahewage Sathyajith Kumarasinghe ,  Antonio Fontanellas Roma ,  Pedro Berraondo Lopez ,  Matias Antonio Avila Zaragoza ,  Lin Tung Guey ,  Staci Sabnis

IPC: A61K38/45 ,  C12N9/10 ,  C12N15/88 ,  A61K48/00 ,  A61P7/08

Abstract: The invention relates to mRNA therapy for the treatment of Acute Intermittent Porphyria (AIP). mRNAs for use in the invention, when administered in vivo, encode human porphobilinogen deaminase (PBGD), isoforms thereof, functional fragments thereof, and fusion proteins comprising PBGD. mRNAs of the invention are preferably encapsulated in lipid nanoparticles (LNPs) to affect efficient delivery to cells and/or tissues in subjects, when administered thereto. mRNA therapies of the invention increase and/or restore deficient levels of PBGD expression and/or activity in subjects. mRNA therapies of the invention further decrease levels of toxic metabolites associated with deficient PBGD activity in subjects, namely porphobilinogen and aminolevulinate (PBG and ALA).

公开(公告)号：US20240316165A1

公开(公告)日：2024-09-26

申请号：US18578052

申请日：2022-07-12

Applicant: ModernaTX, Inc.

Inventor： Husain Attarwala ,  Lei Jiang ,  Matthew Lumley

IPC: A61K38/53 ,  A61K9/51 ,  A61K31/167 ,  A61K31/192 ,  A61K47/24 ,  A61K48/00 ,  A61P3/00

CPC classification number: A61K38/53 ,  A61K9/5123 ,  A61K31/167 ,  A61K31/192 ,  A61K47/24 ,  A61K48/005 ,  A61P3/00

Abstract: This disclosure relates to mRNA therapy for the treatment of propionic acidemia. mRNAs for use in the invention, when administered in vivo, encode propionyl-CoA carboxy lase alpha (PCCA) and propionyl-CoA carboxy lase beta (PCCB). mRNA therapies of the disclosure increase and/or restore deficient levels of PCCA and/or PCCB expression and/or activity in subjects.

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.