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：US20190300906A1

公开(公告)日：2019-10-03

申请号：US16302341

申请日：2017-05-18

Applicant: MODERNATX, INC.

Inventor： Paolo MARTINI ,  Stephen HOGE ,  Kerry BENENATO ,  Vladimir PRESNYAK ,  Iain McFADYEN ,  Ellalahewage Sathyajith KUMARASINGHE ,  Ding AN ,  Staci SABNIS

IPC: C12N15/88 ,  C07K14/14 ,  C12N9/12 ,  B82Y5/00 ,  G01N33/68 ,  A61K9/51

Abstract: The invention relates to mRNA therapy for the treatment of galactosemia type 1 (Gal-1). mRNAs for use in the invention, when administered in vivo, encode human galactose-1-phosphate uridylyltransferase (GALT), isoforms thereof, functional fragments thereof, and fusion proteins comprising GALT. 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 GALT expression and/or activity in subjects. mRNA therapies of the invention further decrease levels of toxic metabolites associated with deficient GALT activity in subjects, namely galactose-1-phosphate (Gal-1-P).

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：US20190298657A1

公开(公告)日：2019-10-03

申请号：US16302298

申请日：2017-05-18

Applicant: MODERNATX, INC.

Inventor： Paolo MARTINI ,  Stephen HOGE ,  Kerry BENENATO ,  Vladimir PRESNYAK ,  Iain McFADYEN ,  Ellalahewage Sathyajith KUMARASINGHE ,  Evan Lockwood RACHLIN ,  Staci SABNIS

IPC: A61K9/51 ,  C12N9/02 ,  A61K48/00

Abstract: The invention relates to mRNA therapy for the treatment of VLCADD. mRNAs for use in the invention, when administered in vivo, encode human acyl-CoA dehydrogenase, very longchain (ACADVL), isoforms thereof, functional fragments thereof, and fusion proteins comprising ACADVL. 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 ACADVL expression and/or activity in subjects. mRNA therapies of the invention further decrease levels of toxic metabolites associated with deficient ACADVL activity in subjects, namely acylcarnitine and acylcarnitine metabolites.

公开(公告)号：US20230112986A1

公开(公告)日：2023-04-13

申请号：US17813984

申请日：2022-07-21

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

Inventor： Paolo MARTINI ,  Stephen HOGE ,  Kerry BENENATO ,  Vladimir PRESNVAK ,  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 ,  A61K48/00 ,  A61P7/08 ,  C12N15/88

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：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：US20190298658A1

公开(公告)日：2019-10-03

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: A61K9/51 ,  A61K9/00 ,  A61P11/00 ,  A61K38/17

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：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.