公开(公告)号：US20220071915A1

公开(公告)日：2022-03-10

申请号：US17154325

申请日：2021-01-21

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：US20210252129A1

公开(公告)日：2021-08-19

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

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：US10993918B2

公开(公告)日：2021-05-04

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 ,  A61K38/00

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：US20200338004A1

公开(公告)日：2020-10-29

申请号：US16760406

申请日：2018-10-31

Applicant: MODERNATX, INC.

Inventor： Kenny Mikael Hansson ,  Kerry Benenato ,  Maria Wågberg ,  Annika Pålsson ,  Regina Fritsche-Danielson

IPC: A61K9/127 ,  A61P43/00 ,  A61K9/00 ,  A61K31/7088 ,  A61K38/18

Abstract: The disclosure relates to nanoparticles comprising a lipid component and a modified RNA encoding a VEGF-A polypeptide. Aspects of the disclosure further relate to uses of nanoparticles comprising a lipid component and a modified RNA encoding a VEGF-A polypeptide, for improving wound healing in a subject.

公开(公告)号：US10519455B2

公开(公告)日：2019-12-31

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: C12N15/11 ,  C12N15/67 ,  A61K9/51 ,  C12N9/40 ,  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：US10335486B2

公开(公告)日：2019-07-02

申请号：US15996146

申请日：2018-06-01

Applicant: ModernaTX, Inc.

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

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

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：US20180028664A1

公开(公告)日：2018-02-01

申请号：US15674872

申请日：2017-08-11

Applicant: ModernaTX, Inc.

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

IPC: A61K47/10 ,  C12N15/117 ,  A61K39/39 ,  C08G65/333 ,  C08G65/332 ,  C08G65/335 ,  A61K31/713 ,  C08G65/334

CPC classification number: A61K48/0033 ,  A61K9/127 ,  A61K31/7115 ,  A61K31/713 ,  A61K39/39 ,  A61K47/10 ,  A61K47/543 ,  A61K47/544 ,  A61K47/60 ,  A61K47/6929 ,  A61K48/0066 ,  A61K2039/55555 ,  C08G65/3322 ,  C08G65/3326 ,  C08G65/33306 ,  C08G65/3331 ,  C08G65/33317 ,  C08G65/33324 ,  C08G65/3348 ,  C08G65/3356 ,  C08G2650/04 ,  C12N15/117 ,  C12N15/88 ,  C12N2310/141 ,  C12N2310/17 ,  C12N2320/32

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：US12123030B2

公开(公告)日：2024-10-22

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: A61K48/00 ,  A61K9/127 ,  A61K9/51 ,  A61K47/14 ,  C12N9/20 ,  B82Y5/00 ,  B82Y30/00

CPC classification number: C12N9/20 ,  A61K9/1274 ,  A61K9/5123 ,  A61K47/14 ,  B82Y5/00 ,  B82Y30/00 ,  C12Y301/01034

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：US20240207374A1

公开(公告)日：2024-06-27

申请号：US18282643

申请日：2022-03-24

Applicant: ModernaTX, Inc.

Inventor： Kerry Benenato ,  Mark Cornebise ,  Edward Hennessy

IPC: A61K38/46 ,  A61K9/51 ,  A61K47/18 ,  A61K48/00

CPC classification number: A61K38/465 ,  A61K9/5123 ,  A61K47/183 ,  A61K48/0033

Abstract: This disclosure relates to ionizable amino lipid-based lipid nanoparticles for delivery of mRNA encoding glucose-6-phosphatase. Lipid nanoparticle/mRNA therapies of the invention increase and/or restore deficient levels of glucose-6-phosphatase expression and/or activity in subjects and are useful for the treatment of glycogen storage disease type 1a (GSD-Ia). Lipid nanoparticle/mRNA therapies of the invention increase glucose production and reduce the abnormal accumulation of glycogen and glucose-6-phosphate associated with GSD-Ia.

公开(公告)号：US11571463B2

公开(公告)日：2023-02-07

申请号：US17721583

申请日：2022-04-15

Applicant: ModernaTX, Inc.

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

IPC: A61K48/00 ,  A61K38/20 ,  A61K9/51 ,  C07K14/54 ,  A61K31/7088 ,  A61K31/7115 ,  C12N15/62 ,  A61P35/00 ,  A61K9/00

Abstract: The present disclosure relates to polynucleotides comprising an open reading frame of linked nucleosides encoding human interleukin-12 (IL12), functional fragments thereof, and fusion proteins comprising IL12. In some embodiments, the open reading frame is sequence-optimized. In particular embodiments, the disclosure provides sequence-optimized polynucleotides comprising nucleotides encoding the polypeptide sequence of human IL12, or sequences having high sequence identity with those sequence optimized polynucleotides.