公开(公告)号：US20210038529A1

公开(公告)日：2021-02-11

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

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：US20190298658A1

公开(公告)日：2019-10-03

申请号：US16302370

申请日：2017-05-18

Applicant: MODERNATX, INC.

Inventor： Kerry BENENATO ,  Stephen HOGE ,  Iain McFADYEN ,  Vladimir PRESNYAK ,  Paolo MARTINI ,  Ellalahewage Sathyajith KUMARASINGHE

IPC: A61K9/51 ,  A61K9/00 ,  A61P11/00 ,  A61K38/17

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

公开(公告)号：US20190000932A1

公开(公告)日：2019-01-03

申请号：US16110788

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: A61K38/47 ,  A61K9/51 ,  A61P3/00

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：US20230026381A1

公开(公告)日：2023-01-26

申请号：US17930369

申请日：2022-09-07

Applicant: ModernaTX, Inc.

Inventor： Joshua FREDERICK ,  Ailin BAI ,  Vladimir PRESNYAK ,  Stephen HOGE ,  Kerry BENENATO ,  Iain MCFADYEN ,  Ellalahewage Sathyajith KUMARASINGHE ,  Susannah HEWITT

IPC: A61K9/51 ,  A61K48/00 ,  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

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：US20220402965A1

公开(公告)日：2022-12-22

申请号：US17277829

申请日：2019-09-19

Applicant: ModernaTX, Inc.

Inventor： Kerry BENENATO ,  Mark CORNEBISE ,  Edward J. HENNESSY

IPC: C07J7/00 ,  A61K9/51 ,  C07J17/00 ,  C07J43/00 ,  C07J33/00 ,  C07J41/00 ,  C07J9/00 ,  C07J21/00 ,  C07J71/00 ,  C07J31/00 ,  C07J51/00 ,  A61K31/7115 ,  A61K38/46 ,  A61K31/7105 ,  A61K47/10 ,  A61K47/24 ,  A61K47/28 ,  A61K48/00

Abstract: The invention relates to compositions and methods for the preparation, manufacture, and therapeutic use of compositions comprising mRNA and a lipid nanoparticle comprising a compound of the invention and an ionizable lipid.

公开(公告)号：US20220265774A1

公开(公告)日：2022-08-25

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: A61K38/20 ,  A61K9/51 ,  C07K14/54 ,  A61K31/7088 ,  A61K31/7115 ,  A61K48/00 ,  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：US20190382774A1

公开(公告)日：2019-12-19

申请号：US16300223

申请日：2017-05-18

Applicant: ModernaTX, Inc.

Inventor： Stephen G. HOGE ,  Kerry BENENATO ,  Vladimir PRESNYAK ,  Iain MCFADYEN ,  Ellalahewage Sathyajith KUMARASINGHE ,  Staci SABNIS ,  William BUTCHER

IPC: C12N15/67 ,  A61K31/7115 ,  A61K48/00

Abstract: The invention related to polyribonucleotides comprising an open reading frame of linked nucleosides encoding a polypeptide of interest (e.g., a therapeutic polypeptide), isoforms thereof, functional fragments thereof, and fusion proteins comprising the polypeptide. In some embodiments, the open reading frame is sequence-optimized. In particular embodiments, the invention provides sequence-optimized polyribonucleotides comprising nucleotides encoding the sequence of the polypeptide of interest, or sequence having high sequence identity with those sequence optimized polyribonucleotides.

公开(公告)号：US20190314291A1

公开(公告)日：2019-10-17

申请号：US16262758

申请日：2019-01-30

Applicant: ModernaTX, Inc.

Inventor： Gilles BESIN ,  Luis BRITO ,  Stephen G. HOGE ,  Edward HENNESSY ,  Mark CORNEBISE ,  Kerry BENENATO ,  Staci SABNIS ,  Michael W. DANNEMAN

IPC: A61K9/51 ,  C07K14/525 ,  A61P35/00 ,  C07K14/705 ,  A61K39/245

Abstract: The disclosure features immune cell delivery lipid nanoparticle (LNP) compositions that allow for enhanced delivery of agents, e.g., nucleic acids, such as therapeutic and/or prophylactic RNAs, to immune cells, in particular T cells, as well as B cells, dendritic cells and monocytes. The LNPs comprise an effective amount of an immune cell delivery potentiating lipid such that delivery of an agent by an immune cell delivery LNP is enhanced as compared to an LNP lacking the immune cell delivery potentiating agent. Methods of using the immune cell delivery LNPs for delivery of agents, e.g., nucleic acid delivery, for protein expression, for modulating immune cell activity and modulating immune responses are also disclosed.

公开(公告)号：US20190298657A1

公开(公告)日：2019-10-03

申请号：US16302298

申请日：2017-05-18

Applicant: MODERNATX, INC.

Inventor： Paolo MARTINI ,  Stephen HOGE ,  Kerry BENENATO ,  Vladimir PRESNYAK ,  Iain McFADYEN ,  Ellalahewage Sathyajith KUMARASINGHE ,  Evan Lockwood RACHLIN ,  Staci SABNIS

IPC: A61K9/51 ,  C12N9/02 ,  A61K48/00

Abstract: The invention relates to mRNA therapy for the treatment of VLCADD. mRNAs for use in the invention, when administered in vivo, encode human acyl-CoA dehydrogenase, very longchain (ACADVL), isoforms thereof, functional fragments thereof, and fusion proteins comprising ACADVL. 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 ACADVL expression and/or activity in subjects. mRNA therapies of the invention further decrease levels of toxic metabolites associated with deficient ACADVL activity in subjects, namely acylcarnitine and acylcarnitine metabolites.

公开(公告)号：US20190105280A1

公开(公告)日：2019-04-11

申请号：US16219418

申请日：2018-12-13

Applicant: ModernaTX, Inc.

Inventor： Joshua FREDERICK ,  Ailin BAI ,  Vladimir PRESNYAK ,  Stephen HOGE ,  Kerry BENENATO ,  Iain MCFADYEN ,  Ellalahewage Sathyajith KUMARASINGHE ,  Susannah HEWITT

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

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).