公开(公告)号：US20190175517A1

公开(公告)日：2019-06-13

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

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：US20170233332A1

公开(公告)日：2017-08-17

申请号：US15498698

申请日：2017-04-27

Applicant: MODERNATX, Inc.

Inventor： Kerry E. Benenato ,  Ellalahewage Sathyajith Kumarasinghe ,  Mark Cornebise

IPC: C07C229/12 ,  C07C227/16 ,  A61K38/18 ,  A61K9/16

CPC classification number: C07C229/12 ,  A61K9/0019 ,  A61K9/0043 ,  A61K9/0073 ,  A61K9/127 ,  A61K9/1271 ,  A61K9/1272 ,  A61K9/1617 ,  A61K9/1641 ,  A61K9/5123 ,  A61K9/5146 ,  A61K31/7105 ,  A61K38/1725 ,  A61K38/1816 ,  A61K47/543 ,  A61K47/6911 ,  A61K48/00 ,  A61K48/0033 ,  A61K48/005 ,  C07C227/16 ,  C07C227/18 ,  C07C229/16 ,  C07C233/36 ,  C07C235/10 ,  C07C255/24 ,  C07C271/20 ,  C07C275/14 ,  C07C279/12 ,  C07C279/24 ,  C07C279/28 ,  C07C279/32 ,  C07C311/05 ,  C07C335/08 ,  C07C2601/02 ,  C07C2601/04 ,  C07C2601/14 ,  C07C2601/18 ,  C07D207/27 ,  C07D233/72 ,  C07D249/04 ,  C07D263/20 ,  C07D265/33 ,  C07D271/06 ,  C07D271/10 ,  C07D277/38 ,  C07F9/091 ,  C07K14/505

Abstract: The disclosure features novel lipids and compositions involving the same. Nanoparticle compositions include a novel lipid as well as additional lipids such as phospholipids, structural lipids, and PEG lipids. Nanoparticle compositions further including therapeutic and/or prophylactics such as RNA are useful in the delivery of therapeutic and/or prophylactics to mammalian cells or organs to, for example, regulate polypeptide, protein, or gene expression.

公开(公告)号：US20170224844A1

公开(公告)日：2017-08-10

申请号：US15493281

申请日：2017-04-21

Applicant: MODERNATX, Inc.

Inventor： Kerry E. Benenato ,  Ellalahewage Sathyajith Kumarasinghe ,  Mark Cornebise

IPC: A61K48/00 ,  C07C227/18 ,  A61K38/18 ,  A61K31/7105 ,  A61K38/17 ,  C07C229/16 ,  A61K9/51

CPC classification number: C07C229/12 ,  A61K9/0019 ,  A61K9/0043 ,  A61K9/0073 ,  A61K9/127 ,  A61K9/1271 ,  A61K9/1272 ,  A61K9/1617 ,  A61K9/1641 ,  A61K9/5123 ,  A61K9/5146 ,  A61K31/7105 ,  A61K38/1725 ,  A61K38/1816 ,  A61K47/543 ,  A61K47/6911 ,  A61K48/00 ,  A61K48/0033 ,  A61K48/005 ,  C07C227/16 ,  C07C227/18 ,  C07C229/16 ,  C07C233/36 ,  C07C235/10 ,  C07C255/24 ,  C07C271/20 ,  C07C275/14 ,  C07C279/12 ,  C07C279/24 ,  C07C279/28 ,  C07C279/32 ,  C07C311/05 ,  C07C335/08 ,  C07C2601/02 ,  C07C2601/04 ,  C07C2601/14 ,  C07C2601/18 ,  C07D207/27 ,  C07D233/72 ,  C07D249/04 ,  C07D263/20 ,  C07D265/33 ,  C07D271/06 ,  C07D271/10 ,  C07D277/38 ,  C07F9/091 ,  C07K14/505

Abstract: The disclosure features novel lipids and compositions involving the same. Nanoparticle compositions include a novel lipid as well as additional lipids such as phospholipids, structural lipids, and PEG lipids. Nanoparticle compositions further including therapeutic and/or prophylactics such as RNA are useful in the delivery of therapeutic and/or prophylactics to mammalian cells or organs to, for example, regulate polypeptide, protein, or gene expression.

公开(公告)号：US12128113B2

公开(公告)日：2024-10-29

申请号：US16302607

申请日：2017-05-18

Applicant: ModernaTX, Inc.

Inventor： Kerry Benenato ,  Stephen Hoge ,  Paolo Martini ,  Iain Mcfadyen ,  Vladimir Presnyak ,  Ding An ,  Ellalahewage Sathyajith Kumarasinghe

IPC: A61K48/00 ,  A61K9/00 ,  A61K9/51 ,  A61K47/00 ,  A61K47/14 ,  A61K47/16 ,  A61K47/22 ,  A61K47/24 ,  C12N15/88 ,  A61K9/127 ,  A61K47/10 ,  A61K47/28 ,  C07K14/705

CPC classification number: A61K48/0066 ,  A61K9/51 ,  A61K47/14 ,  A61K47/16 ,  A61K47/22 ,  A61K47/24 ,  A61K48/0041 ,  C12N15/88 ,  A61K9/1271 ,  A61K9/5123 ,  A61K47/10 ,  A61K47/28 ,  C07K14/705

Abstract: The invention relates to mRNA therapy for the treatment of Alagille syndrome (ALGS), mRNAs for use in the invention, when administered in vivo, encode JAGGED 1 (JAG1), isoforms thereof functional fragments thereof, and fusion proteins comprising JAG1, 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 JAG1 expression and/or activity in subjects. mRNA therapies of the invention further decrease levels of toxic metabolites associated with deficient JAG1 activity in subjects.

公开(公告)号：US20230270836A1

公开(公告)日：2023-08-31

申请号：US17938823

申请日：2022-10-07

Applicant: ModernaTX, Inc.

Inventor： Giuseppe Ciaramella ,  Sunny Himansu ,  Vladimir Presnyak ,  Kerry Benenato ,  Ellalahewage Sathyajith Kumarasinghe

IPC: A61K39/12 ,  A61P31/14 ,  A61K31/7115 ,  C12N15/86

CPC classification number: A61K39/12 ,  A61P31/14 ,  A61K31/7115 ,  C12N15/86 ,  A61K2039/53

Abstract: The disclosure relates to Lassa virus, Nipah virus, and betacoronavirus ribonucleic acid vaccines as well as methods of using the vaccines and compositions comprising the vaccines.

公开(公告)号：US20230270685A1

公开(公告)日：2023-08-31

申请号：US17989941

申请日：2022-11-18

Applicant: ModernaTX, Inc.

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

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

CPC classification number: A61K9/5123 ,  A61K9/1272 ,  A61K38/52 ,  A61K48/005 ,  A61P3/00 ,  A61P43/00 ,  C12N9/90 ,  C12N15/52 ,  C12Y504/99002 ,  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：US11660341B2

公开(公告)日：2023-05-30

申请号：US17202829

申请日：2021-03-16

Applicant: ModernaTX, Inc.

Inventor： Joshua P. Frederick ,  Susannah Hewitt ,  Ailin Bai ,  Stephen G. Hoge ,  Vladimir Presnyak ,  Iain McFadyen ,  Kerry Benenato ,  Ellalahewage Sathyajith Kumarasinghe

IPC: A61K48/00 ,  A61K39/395 ,  C07K14/54 ,  C07K14/705 ,  C07K16/28 ,  A61K39/39 ,  A61K45/06 ,  A61K31/713 ,  A61K39/00 ,  A61P35/00

CPC classification number: A61K39/39558 ,  A61K31/713 ,  A61K39/0011 ,  A61K39/39 ,  A61K45/06 ,  A61K48/005 ,  C07K14/54 ,  C07K14/5434 ,  C07K14/5443 ,  C07K14/705 ,  C07K14/70503 ,  C07K14/70532 ,  C07K14/70575 ,  C07K14/70596 ,  C07K16/2818 ,  A61K2039/505 ,  A61K2039/51 ,  A61K2039/53 ,  A61K2039/585 ,  A61P35/00 ,  C07K2317/76 ,  C07K2319/30 ,  C07K2319/32 ,  A61K31/713 ,  A61K2300/00

Abstract: The present disclosure relates to the use of nucleic acid (e.g., mRNA) combination therapies for the treatment of cancer. The disclosure provides compositions, and methods for their preparation, manufacture, and therapeutic use, wherein those compositions comprise at least two polynucleotides (e.g., mRNAs) in combination wherein the at least two polynucleotides are selected from the group consisting of (i) a polynucleotide encoding an immune response primer (e.g., IL23), (ii) a polynucleotide encoding an immune response co-stimulatory signal (e.g., OX40L), (iii) a polynucleotide encoding a checkpoint inhibitor (e.g., an anti CTLA-4 antibody), and, (iv) a combination thereof. The therapeutic methods disclosed herein comprise, e.g., the administration of a combination therapy disclosed herein for the treatment of cancer, e.g., by reducing the size of a tumor or inhibiting the growth of a tumor, in a subject in need thereof. In some aspects, the combination therapies disclosed herein disclosed are administered intratumorally.

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.

公开(公告)号：US11497807B2

公开(公告)日：2022-11-15

申请号：US17155592

申请日：2021-01-22

Applicant: ModernaTX, Inc.

Inventor： Giuseppe Ciaramella ,  Sunny Himansu ,  Vladimir Presnyak ,  Kerry Benenato ,  Ellalahewage Sathyajith Kumarasinghe

IPC: A61K39/12 ,  A61P31/14 ,  A61K31/7115 ,  C12N15/86 ,  A61K39/00

Abstract: Provide herein are Lassa virus, Nipah virus, and betacoronavirus ribonucleic acid vaccines as well as methods of using the vaccines and compositions comprising the vaccines.

公开(公告)号：US11001861B2

公开(公告)日：2021-05-11

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 ,  A61K9/51 ,  B82Y5/00 ,  C07K14/14 ,  C12N9/12 ,  G01N33/68 ,  A61K48/00

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).