公开(公告)号：US20210269830A1

公开(公告)日：2021-09-02

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: 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：US11071716B2

公开(公告)日：2021-07-27

申请号：US17071685

申请日：2020-10-15

Applicant: ModernaTX, Inc.

Inventor： Joshua Frederick ,  Ailin Bai ,  Vladimir Presnyak ,  Stephen G. 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 ,  A61K31/7088 ,  A61K31/7115 ,  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：US20200085916A1

公开(公告)日：2020-03-19

申请号：US16302339

申请日：2017-05-18

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

Inventor： Paolo Martini ,  Stephen Hoge ,  Kerry Benenato ,  Vladimir Presnyak ,  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 ,  C12N15/88 ,  A61K48/00 ,  A61P7/08

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：US20200078314A1

公开(公告)日：2020-03-12

申请号：US16515256

申请日：2019-07-18

Applicant: ModernaTX, Inc.

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

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

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：US20190390181A1

公开(公告)日：2019-12-26

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: C12N9/20 ,  A61K47/14 ,  A61K9/127 ,  A61K9/51

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：US20190275170A1

公开(公告)日：2019-09-12

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 ,  A61K9/51 ,  A61K47/14 ,  A61K47/16 ,  A61K47/22 ,  A61K47/24 ,  C12N15/88

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 (JAG 1), 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：US10322090B2

公开(公告)日：2019-06-18

申请号：US16184282

申请日：2018-11-08

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：US10285950B2

公开(公告)日：2019-05-14

申请号：US16227810

申请日：2018-12-20

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 ,  C07K14/54 ,  A61K38/17 ,  A61K38/20 ,  C07K14/705 ,  A61P35/00 ,  A61K39/395 ,  A61K9/00 ,  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：US20190060246A1

公开(公告)日：2019-02-28

申请号：US16184282

申请日：2018-11-08

Applicant: ModernaTX, Inc.

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

IPC: A61K9/51 ,  C07K14/705 ,  A61P35/00 ,  C07K14/54 ,  C07K14/545 ,  A61K9/00 ,  A61K9/127 ,  A61K38/17 ,  A61K48/00 ,  A61K45/06 ,  A61K39/395 ,  A61K39/39 ,  A61K38/20 ,  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：US20170210698A1

公开(公告)日：2017-07-27

申请号：US15476263

申请日：2017-03-31

Applicant: MODERNATX, Inc.

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

IPC: C07C229/12 ,  A61K38/18 ,  A61K9/00 ,  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.