公开(公告)号：US20190000933A1

公开(公告)日：2019-01-03

申请号：US16110833

申请日：2018-08-23

Applicant: ModernaTX, Inc.

Inventor： Paolo MARTINI ,  Stephen G. HOGE ,  Kerry BENENATO ,  Vladimir PRESNYAK ,  Iain MCFADYEN ,  Ellalahewage Sathyajith KUMARASINGHE ,  Xuling ZHU ,  Lin Tung GUEY ,  Staci SABNIS

IPC: A61K38/47 ,  A61K9/51 ,  A61P3/00

Abstract: The invention relates to mRNA therapy for the treatment of Fabry disease. mRNAs for use in the invention, when administered in vivo, encode human the α-galactosidase A (GLA), isoforms thereof, functional fragments thereof, and fusion proteins comprising GLA. 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 GLA expression and/or activity in subjects. mRNA therapies of the invention further decrease levels of toxic metabolites associated with deficient GLA activity in subjects, namely Gb3 and lyso-Gb3.

公开(公告)号：US20190002890A1

公开(公告)日：2019-01-03

申请号：US16110309

申请日：2018-08-23

Applicant: ModernaTX, Inc.

Inventor： Paolo MARTINI ,  Stephen G. HOGE ,  Kerry BENENATO ,  Vladimir PRESNYAK ,  Iain MCFADYEN ,  Ellalahewage Sathyajith KUMARASINGHE ,  Xuling ZHU ,  Lin Tung GUEY ,  Staci SABNIS

IPC: C12N15/67 ,  C12N9/40 ,  A61K9/51

Abstract: The invention relates to mRNA therapy for the treatment of Fabry disease. mRNAs for use in the invention, when administered in vivo, encode human the α-galactosidase A (GLA), isoforms thereof, functional fragments thereof, and fusion proteins comprising GLA. 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 GLA expression and/or activity in subjects. mRNA therapies of the invention further decrease levels of toxic metabolites associated with deficient GLA activity in subjects, namely Gb3 and lyso-Gb3.

公开(公告)号：US20200149052A1

公开(公告)日：2020-05-14

申请号：US16570351

申请日：2019-09-13

Applicant: ModernaTX, Inc.

Inventor： Paolo MARTINI ,  Stephen G. HOGE ,  Kerry BENENATO ,  Vladimir PRESNYAK ,  Iain MCFADYEN ,  Ellalahewage Sathyajith KUMARASINGHE ,  Xuling ZHU ,  Lin Tung GUEY ,  Staci SABNIS

IPC: C12N15/67 ,  A61K38/47 ,  A61P3/00 ,  C12N9/40 ,  A61K9/51

Abstract: The invention relates to mRNA therapy for the treatment of Fabry disease. mRNAs for use in the invention, when administered in vivo, encode human the α-galactosidase A (GLA), isoforms thereof, functional fragments thereof, and fusion proteins comprising GLA. 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 GLA expression and/or activity in subjects. mRNA therapies of the invention further decrease levels of toxic metabolites associated with deficient GLA activity in subjects, namely Gb3 and lyso-Gb3.

公开(公告)号：US20230323371A1

公开(公告)日：2023-10-12

申请号：US18296596

申请日：2023-04-06

Applicant: ModernaTX, Inc.

Inventor： Paolo MARTINI ,  Stephen G. HOGE ,  Kerry BENENATO ,  Vladimir PRESNYAK ,  Iain MCFADYEN ,  Ellalahewage Sathyajith KUMARASINGHE ,  Xuling ZHU ,  Lin Tung GUEY ,  Staci SABNIS

IPC: C12N15/67 ,  A61K9/51 ,  C12N9/40 ,  A61P3/00 ,  A61K38/47

CPC classification number: C12N15/67 ,  A61K9/5123 ,  C12N9/2465 ,  C12Y302/01022 ,  A61P3/00 ,  A61K38/47 ,  A61K48/00

Abstract: The invention relates to mRNA therapy for the treatment of Fabry disease. mRNAs for use in the invention, when administered in vivo, encode human the α-galactosidase A (GLA), isoforms thereof, functional fragments thereof, and fusion proteins comprising GLA. 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 GLA expression and/or activity in subjects. mRNA therapies of the invention further decrease levels of toxic metabolites associated with deficient GLA activity in subjects, namely Gb3 and lyso-Gb3.

公开(公告)号：US20190000932A1

公开(公告)日：2019-01-03

申请号：US16110788

申请日：2018-08-23

Applicant: ModernaTX, Inc.

Inventor： Paolo MARTINI ,  Stephen G. HOGE ,  Kerry BENENATO ,  Vladimir PRESNYAK ,  Iain MCFADYEN ,  Ellalahewage Sathyajith KUMARASINGHE ,  Xuling ZHU ,  Lin Tung GUEY ,  Staci SABNIS

IPC: A61K38/47 ,  A61K9/51 ,  A61P3/00

Abstract: The invention relates to mRNA therapy for the treatment of Fabry disease. mRNAs for use in the invention, when administered in vivo, encode human the α-galactosidase A (GLA), isoforms thereof, functional fragments thereof, and fusion proteins comprising GLA. 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 GLA expression and/or activity in subjects. mRNA therapies of the invention further decrease levels of toxic metabolites associated with deficient GLA activity in subjects, namely Gb3 and lyso-Gb3.