公开(公告)号：US10519455B2

公开(公告)日：2019-12-31

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: C12N15/11 ,  C12N15/67 ,  A61K9/51 ,  C12N9/40 ,  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：US11649461B2

公开(公告)日：2023-05-16

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: A61K48/00 ,  C12N15/67 ,  A61K9/51 ,  C12N9/40 ,  A61P3/00 ,  A61K38/47

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