公开(公告)号：US20230250439A1

公开(公告)日：2023-08-10

申请号：US18093119

申请日：2023-01-04

Applicant: ModernaTX, Inc.

Inventor： David Mauger ,  Iain Mcfadyen ,  Vladimir Presnyak

IPC: C12N15/67 ,  A61K48/00

CPC classification number: C12N15/67 ,  A61K48/0066

Abstract: The disclosure relates to synthetic thermostable polynucleotides, as well as methods of synthesizing and delivering the polynucleotides.

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：US20230009009A1

公开(公告)日：2023-01-12

申请号：US17275053

申请日：2019-09-12

Applicant: ModernaTX, Inc.

Inventor： Paolo G. V. Martini ,  Athanasios Dousis ,  Vladimir Presnyak ,  Jingsong Cao

IPC: A61K9/51 ,  A61K38/46 ,  A61P3/06 ,  A61K9/00 ,  C12N9/16

Abstract: This disclosure relates to mRNA therapy for the treatment of glycogen storage disease type 1a, (GSD-Ia), and related symptoms such as hypoglycemia. mRNAs for use in the invention, when administered in vivo, encode human glucose-6-phosphatase (G6Pase or G6PC), and functional fragments and variants thereof. 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 G6PC expression and/or activity in subjects. mRNA therapies of the invention further increase the glucose production, and reduce the abnormal accumulation of glycogen and/or glucose-6-phosphate associated with GSD-Ia.

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：US20220110966A1

公开(公告)日：2022-04-14

申请号：US17271401

申请日：2019-08-29

Applicant: ModernaTX, Inc.

Inventor： Vladimir Presnyak ,  Paolo Martini

IPC: A61K31/7115 ,  A61K9/00 ,  A61K9/51 ,  A61K38/44

Abstract: This disclosure relates to mRNA therapy for the treatment of very long-chain specific acyl-CoA dehydrogenase deficiency (VLCADD). mRNAs for use in the invention, when administered in vivo, encode human very long-chain specific acyl-CoA dehydrogenase (VLCAD). mRNA therapies of the disclosure increase and/or restore deficient levels of VLCAD expression and/or activity in subjects. mRNA therapies of the disclosure further decrease abnormal accumulation of acylcarnitine associated with deficient VLCAD activity in subjects.

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).

公开(公告)号：US10730924B2

公开(公告)日：2020-08-04

申请号：US16025302

申请日：2018-07-02

Applicant: ModernaTX, Inc.

Inventor： Barry Ticho ,  Nadege Briancon-Eris ,  Zhinan Xia ,  Athanasios Dousis ,  Seymour de Picciotto ,  Vladimir Presnyak ,  Stephen Hoge ,  Iain Mcfadyen ,  Kerry Benenato ,  Ellalahewage Sathyajith Kumarasinghe

IPC: C12N15/62 ,  A61K47/14 ,  C07K14/64 ,  A61K31/7105 ,  A61K47/69 ,  A61P9/00 ,  A61K38/17 ,  C07K16/26 ,  C12N15/52 ,  A61K9/127 ,  A61K38/00 ,  A61K9/00

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

公开(公告)号：US20200030432A1

公开(公告)日：2020-01-30

申请号：US16494988

申请日：2018-03-16

Applicant: ModernaTX, Inc.

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

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

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：US10406113B2

公开(公告)日：2019-09-10

申请号：US16222155

申请日：2018-12-17

Applicant: ModernaTX, Inc.

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

IPC: A61K48/00 ,  A61K9/51 ,  A61K38/17 ,  A61K38/20 ,  A61K9/00 ,  A61K39/39 ,  A61K45/06 ,  A61K9/127 ,  C07K14/54 ,  C07K14/545 ,  C07K14/705 ,  A61P35/00 ,  A61K39/395 ,  A61K39/00 ,  B82Y5/00 ,  C12N15/88

Abstract: The disclosure relates to compositions and methods for the preparation, manufacture and therapeutic use of combinations of immunomodulatory polynucleotides (e.g., mRNAs) encoding an immune response primer polypeptide (e.g., an interleukin 23 (IL-23) polypeptide or an interleukin 36γ (IL-36-gamma) polypeptide), and an immune response co-stimulatory signal polypeptide (e.g., an OX40L polypeptide).