公开(公告)号：US11801227B2

公开(公告)日：2023-10-31

申请号：US16302370

申请日：2017-05-18

Applicant: ModernaTX, Inc.

Inventor： Kerry Benenato ,  Stephen Hoge ,  Iain McFadyen ,  Vladimir Presnyak ,  Paolo Martini ,  Ellalahewage Sathyajith Kumarasinghe

IPC: A61K38/18 ,  A61K9/51 ,  A61K9/00 ,  A61K9/127 ,  C07K14/705 ,  C07K14/47 ,  A61P11/00 ,  A61K38/17 ,  C12N15/88

CPC classification number: A61K9/5123 ,  A61K9/0043 ,  A61K9/0053 ,  A61K9/0073 ,  A61K38/177 ,  A61P11/00 ,  C07K14/47 ,  C12N15/88 ,  A61K9/1271

Abstract: The invention relates to mRNA therapy for the treatment of cystic fibrosis. mRNAs for use in the invention, when administered in vivo, encode cystic fibrosis transmembrane conductance regulator (CFTR), isoforms thereof, functional fragments thereof, and fusion proteins comprising CFTR. 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 CFTR expression and/or activity in subjects. mRNA therapies of the invention further decrease levels of toxic metabolites associated with deficient CFTR activity in subjects.

公开(公告)号：US20190022019A1

公开(公告)日：2019-01-24

申请号：US16113814

申请日：2018-08-27

Applicant: ModernaTX, Inc.

Inventor： Paolo Martini ,  Vladimir Presnyak

IPC: A61K9/51 ,  A61P3/00 ,  C12N9/90

Abstract: The disclosure relates to polynucleotides comprising an open reading frame of linked nucleosides encoding human methylmalonyl-CoA mutase precursor, human methylmalonyl-CoA mutase (MCM) mature form, or functional fragments thereof. In some embodiments, the disclosure includes methods of treating methylmalonic acidemia in a subject in need thereof comprising administering an mRNA encoding an MCM polypeptide.

公开(公告)号：US20220071915A1

公开(公告)日：2022-03-10

申请号：US17154325

申请日：2021-01-21

Applicant: ModernaTX, Inc.

Inventor： Paolo Martini ,  Stephen Hoge ,  Kerry Benenato ,  Vladimir Presnyak ,  Iain McFadyen ,  Ellalahewage Sathyajith Kumarasinghe ,  Jingsong Cao ,  Lin Tung Guey ,  Staci Sabnis

IPC: A61K9/51 ,  C07K14/47 ,  A61P13/00 ,  A61K9/00

Abstract: The invention relates to mRNA therapy for the treatment of Citrullinemia Type 2 (“CTLN2”). mRNAs for use in the invention, when administered in vivo, encode human Citrin, isoforms thereof, functional fragments thereof, and fusion proteins comprising Citrin. 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 Citrin expression and/or activity in subjects. mRNA therapies of the invention further decrease levels of biomarkers associated with deficient Citrin activity in subjects, namely ammonia and/or triglycerides.

公开(公告)号：US10993918B2

公开(公告)日：2021-05-04

申请号：US16302360

申请日：2017-05-18

Applicant: ModernaTX, Inc.

Inventor： Paolo Martini ,  Stephen Hoge ,  Kerry Benenato ,  Vladimir Presnyak ,  Iain McFadyen ,  Ellalahewage Sathyajith Kumarasinghe ,  Jingsong Cao ,  Lin Tung Guey ,  Staci Sabnis

IPC: A61K9/51 ,  C07K14/47 ,  A61P13/00 ,  A61K9/00 ,  A61K38/00

Abstract: The invention relates to mRNA therapy for the treatment of Citrullinemia Type 2 (“CTLN2”). mRNAs for use in the invention, when administered in vivo, encode human Citrin, isoforms thereof, functional fragments thereof, and fusion proteins comprising Citrin. 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 Citrin expression and/or activity in subjects. mRNA therapies of the invention further decrease levels of biomarkers associated with deficient Citrin activity in subjects, namely ammonia and/or triglycerides.

公开(公告)号：US20200368162A1

公开(公告)日：2020-11-26

申请号：US16487734

申请日：2018-02-24

Applicant: ModernaTX, Inc.

Inventor： Paolo Martini

IPC: A61K9/127 ,  C07K14/47 ,  A61K9/51 ,  A61K31/221

Abstract: The invention related to polynucleotides comprising an open reading frame of linked nucleosides encoding therapeutic proteins or variant therapeutic proteins, isoforms thereof, functional fragments thereof, and fusion proteins comprising therapeutic proteins. In some embodiments, the open reading frame is sequence-optimized. The invention also relates to methods of treating muscular dystrophies.

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：US10426738B2

公开(公告)日：2019-10-01

申请号：US16002472

申请日：2018-06-07

Applicant: ModernaTX, Inc.

Inventor： Paolo Martini ,  Vladimir Presnyak

IPC: C12N15/11 ,  A61K9/51 ,  A61K9/127 ,  C12N9/90 ,  A61P3/00 ,  A61P43/00 ,  A61K38/52 ,  A61K48/00 ,  C12N15/52 ,  A61K38/00

Abstract: The disclosure relates to polynucleotides comprising an open reading frame of linked nucleosides encoding human methylmalonyl-CoA mutase precursor, human methylmalonyl-CoA mutase (MCM) mature form, or functional fragments thereof. In some embodiments, the disclosure includes methods of treating methylmalonic acidemia in a subject in need thereof comprising administering an mRNA encoding an MCM polypeptide.

公开(公告)号：US20180271795A1

公开(公告)日：2018-09-27

申请号：US16002376

申请日：2018-06-07

Applicant: ModernaTX, Inc.

Inventor： Paolo Martini ,  Vladimir Presnyak

IPC: A61K9/51 ,  A61P3/00 ,  C12N9/90

CPC classification number: A61K9/5123 ,  A61K9/1272 ,  A61K38/00 ,  A61K38/52 ,  A61K48/005 ,  A61P3/00 ,  A61P43/00 ,  C12N9/90 ,  C12N15/52 ,  C12Y504/99002

Abstract: The disclosure relates to polynucleotides comprising an open reading frame of linked nucleosides encoding human methylmalonyl-CoA mutase precursor, human methylmalonyl-CoA mutase (MCM) mature form, or functional fragments thereof. In some embodiments, the disclosure includes methods of treating methylmalonic acidemia in a subject in need thereof comprising administering an mRNA encoding an MCM polypeptide.

公开(公告)号：US12123030B2

公开(公告)日：2024-10-22

申请号：US16302368

申请日：2017-05-18

Applicant: ModernaTX, Inc.

Inventor： Kerry Benenato ,  Stephen Hoge ,  Paolo Martini ,  Iain McFadyen ,  Vladimir Presnyak ,  Ellalahewage Sathyajith Kumarasinghe

IPC: A61K48/00 ,  A61K9/127 ,  A61K9/51 ,  A61K47/14 ,  C12N9/20 ,  B82Y5/00 ,  B82Y30/00

CPC classification number: C12N9/20 ,  A61K9/1274 ,  A61K9/5123 ,  A61K47/14 ,  B82Y5/00 ,  B82Y30/00 ,  C12Y301/01034

Abstract: The invention relates to mRNA therapy for the treatment of hyperlipidemia. mRNAs for use in the invention, when administered in vivo, encode human lipoprotein lipase (LPL), isoforms thereof, functional fragments thereof, and fusion proteins comprising LPL. 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 LPL expression and/or activity in subjects. mRNA therapies of the invention further decrease levels of triglycerides associated with deficient LPL activity in subjects.

公开(公告)号：US20220054653A1

公开(公告)日：2022-02-24

申请号：US17276112

申请日：2019-09-13

Applicant: ModernaTX, Inc.

Inventor： Paolo Martini ,  Vladimir Presnyak ,  Jingsong CAO

IPC: A61K48/00 ,  A61K9/51 ,  C07K14/705 ,  A61P1/16

Abstract: The present disclosure provides compositions of nucleic acids relating to biliary epithelial transporters. For example, the present disclosure relates to nucleic acids capable of regulating the biliary secretion of phospholipids, including phosphatidylcholine, e.g., those encoded by ATP binding cassette subfamily B member 4 (ABCB4) or a biologically active fragment thereof, in a target cell. In a preferred embodiment, the present disclosure provides compositions comprising modified mRNA encoding ABCB4 formulated in a lipid nanoparticle (LNP) carrier and derivative constructs, which are useful for treating or preventing progressive familial intrahepatic cholestasis type 3 (PFIC3).