公开(公告)号：US20220265857A1

公开(公告)日：2022-08-25

申请号：US17583674

申请日：2022-01-25

Applicant: ModernaTX,Inc.

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

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

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：US11311602B2

公开(公告)日：2022-04-26

申请号：US17308686

申请日：2021-05-05

Applicant: ModernaTX, Inc.

Inventor： Joshua Frederick ,  Susannah Hewitt ,  Ailin Bai ,  Stephen Hoge ,  Vladimir Presnyak ,  Iain 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.

公开(公告)号：US11000573B2

公开(公告)日：2021-05-11

申请号：US16842300

申请日：2020-04-07

Applicant: ModernaTX, Inc.

Inventor： Joshua Frederick ,  Susannah Hewitt ,  Ailin Bai ,  Stephen Hoge ,  Vladimir Presnyak ,  Iain 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.

公开(公告)号：US10646549B2

公开(公告)日：2020-05-12

申请号：US16192274

申请日：2018-11-15

Applicant: ModernaTX, Inc.

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

IPC: A61K48/00 ,  A61K38/20 ,  A61K9/51 ,  C07K14/54 ,  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.

公开(公告)号：US20190247417A1

公开(公告)日：2019-08-15

申请号：US16333330

申请日：2017-09-14

Applicant: ModernaTX, Inc.

Inventor： Stephen Hoge ,  William Issa ,  Edward J. Miracco ,  Jennifer Nelson ,  Amy E. Rabideau ,  Gabor Butora

IPC: A61K31/7105 ,  G01N33/68 ,  C12N15/10 ,  A61P33/00 ,  A61P31/04 ,  A61P35/00 ,  A61P31/12

CPC classification number: A61K31/7105 ,  A61P31/04 ,  A61P31/12 ,  A61P33/00 ,  A61P35/00 ,  C07K14/505 ,  C12N15/10 ,  C12P19/34 ,  G01N33/6851

Abstract: The invention relates to improved RNA compositions for use in therapeutic applications. The RNA compositions are particularly suited for use in human therapeutic application (e.g., in RNA therapeutics). The RNA compositions are made by improved processes, in particular, improved in vitro-transcription (IVT) processes. The invention also relates to methods for producing and purifying RNA (e.g, therapeutic RNAs), as well as methods for using the RNA compositions and therapeutic applications thereof.

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：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：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：US20230233475A1

公开(公告)日：2023-07-27

申请号：US17928684

申请日：2021-06-01

Applicant: ModernaTX, Inc.

Inventor： Kerry Benenato ,  Kristine Burke ,  Jingsong Cao ,  Paloma Hoban Giangrande ,  Edward J. Hennessy ,  Stephen Hoge ,  Jaclyn Milton ,  Staci Sabnis ,  Timothy Salerno ,  Matthew Theisen

IPC: A61K9/51 ,  A61K31/7088 ,  A61K9/00 ,  C12N9/16 ,  A61P35/00

CPC classification number: A61K9/5123 ,  A61K31/7088 ,  A61K9/0019 ,  C12Y301/03009 ,  C12N9/16 ,  A61P35/00

Abstract: This disclosure relates to ionizable 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 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：US11285222B2

公开(公告)日：2022-03-29

申请号：US16599661

申请日：2019-10-11

Applicant: ModernaTX, Inc.

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

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