公开(公告)号：US20210145982A1

公开(公告)日：2021-05-20

申请号：US16623069

申请日：2018-06-15

Applicant: ModernaTX, Inc.

Inventor： Stephen Hoge ,  Joseph Schariter ,  Charles Bowerman ,  Michael H. Smith ,  Yan Xia

IPC: A61K47/69 ,  A61K47/54 ,  A61K31/7105

Abstract: This disclosure provides improved lipid-based compositions, including lipid nanoparticle compositions, and methods of use thereof for delivering agents in vivo including nucleic acids and proteins.

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：US10849920B2

公开(公告)日：2020-12-01

申请号：US15761220

申请日：2016-10-05

Applicant: ModernaTX, Inc.

Inventor： Stephen Hoge ,  Tirtha Chakraborty ,  Gilles Besin ,  Ruchi Jain

IPC: A61K48/00 ,  C07H21/04 ,  A61K31/7115 ,  C12N15/67 ,  A61K9/00 ,  A61K9/127 ,  C12N15/113

Abstract: The disclosure features methods of reducing or inhibiting an anti-drug antibody response in a subject, as well as methods of reducing or inhibiting unwanted immune cell activation in a subject to be treated with a messenger RNA (mRNA), comprising administering to the subject a mRNA, e.g., a chemically modified messenger RNA (mmRNA), encoding a polypeptide of interest, wherein the mRNA comprises at least one microRNA (miR) binding site for a miR expressed in immune cells, such as miR-126 binding site and/or miR-142 binding site, such that an anti-drug antibody response to the polypeptide or interest, or unwanted immune cell activation (e.g., B cell activation, cytokine secretion), is reduced or inhibited in the subject. The disclosure further provides therapeutic treatment regimens designed to reduce or inhibit AD A or unwanted immune cell activation (e.g., B cell activation, cytokine secretion) in a subject being treated with mRNA-based therapeutics.

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：US10556018B2

公开(公告)日：2020-02-11

申请号：US15674107

申请日：2017-08-10

Applicant: ModernaTX, Inc.

Inventor： Gilles Besin ,  Stephen Hoge ,  Joseph Senn ,  Kerry Benenato ,  Staci Sabnis

IPC: A61K48/00 ,  A61K9/127 ,  C12N15/88 ,  A61K47/54 ,  A61K47/69 ,  A61K47/60 ,  A61K31/713 ,  A61K39/39 ,  A61K47/10 ,  C08G65/332 ,  C08G65/333 ,  C08G65/334 ,  C08G65/335 ,  C12N15/117 ,  A61K31/7115 ,  A61K47/14 ,  A61K47/18 ,  A61K39/00

Abstract: This disclosure provides improved lipid-based compositions, including lipid nanoparticle compositions, and methods of use thereof for delivering agents in vivo including nucleic acids and proteins. These compositions are not subject to accelerated blood clearance and they have an improved toxicity profile in vivo.

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).

公开(公告)号：US10172808B2

公开(公告)日：2019-01-08

申请号：US15995889

申请日：2018-06-01

Applicant: ModernaTX, Inc.

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

IPC: A61K48/00 ,  A61K31/7115 ,  A61K9/51 ,  A61K38/20 ,  A61K38/17 ,  C07K14/54 ,  C07K14/705 ,  A61K39/395 ,  A61K9/00 ,  A61P35/00 ,  A61K39/00

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).

公开(公告)号：US20180371047A1

公开(公告)日：2018-12-27

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: C07K14/64 ,  A61P9/00 ,  C12N15/62 ,  C12N15/52 ,  A61K47/69 ,  C07K16/26 ,  A61K38/17

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：US12252704B2

公开(公告)日：2025-03-18

申请号：US17154309

申请日：2021-01-21

Applicant: ModernaTX, Inc.

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

IPC: A61K9/51 ,  A61K48/00 ,  B82Y5/00 ,  C07K14/14 ,  C12N9/10 ,  C12N9/12 ,  C12N15/88 ,  G01N33/68

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：US12103955B2

公开(公告)日：2024-10-01

申请号：US16944014

申请日：2020-07-30

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 ,  A61K31/7105 ,  A61K38/17 ,  A61K47/14 ,  A61K47/69 ,  A61K48/00 ,  A61P9/00 ,  C07K14/64 ,  C07K16/26 ,  C12N15/52 ,  A61K9/00 ,  A61K9/127 ,  A61K38/00

CPC classification number: C07K14/64 ,  A61K31/7105 ,  A61K38/1796 ,  A61K47/6929 ,  A61P9/00 ,  C07K16/26 ,  C12N15/52 ,  C12N15/62 ,  A61K9/0019 ,  A61K9/127 ,  A61K38/00 ,  C07K2319/00 ,  C07K2319/31

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.