公开(公告)号：WO2023076605A1

公开(公告)日：2023-05-04

申请号：PCT/US2022/048231

申请日：2022-10-28

Applicant: MODERNATX, INC.

Inventor： SEEPERSAUD, Mohindra ,  CRAWFORD, Matthew ,  TATE, Daniel ,  CORNEBISE, Mark ,  MCLAUGHLIN, Christopher Karl ,  CADETE PIRES, Ana

IPC: C07J41/00 ,  A61K48/00 ,  C07J43/00

Abstract: The present invention relates to lipid amine compounds of formula (A1) which are useful in the preparation of lipid nanoparticle compositions for delivery of therapeutic or prophylactic payload into cells.

公开(公告)号：WO2022204390A1

公开(公告)日：2022-09-29

申请号：PCT/US2022/021726

申请日：2022-03-24

Applicant: MODERNATX, INC.

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

IPC: A61K9/51 ,  A61K48/00 ,  C12N9/16 ,  A61K47/50 ,  A61K48/0041 ,  A61K9/5123 ,  C12N15/88 ,  C12N9/0071 ,  C12Y114/16001

Abstract: This disclosure relates to ionizable amino lipid-based lipid nanoparticles for delivery of mRNA encoding phenylalanine hydroxylase. Lipid nanoparticle/mRNA therapies of the invention increase and/or restore deficient levels of phenylalanine hydroxylase expression and/or activity in subjects and are useful for the treatment of phenylketonuria

公开(公告)号：WO2021055833A1

公开(公告)日：2021-03-25

申请号：PCT/US2020/051609

申请日：2020-09-18

Applicant: MODERNATX, INC.

Inventor： BENENATO, Kerry E. ,  CORNEBISE, Mark ,  HENNESSY, Edward ,  KUMARASINGHE, Ellalahewage S.

IPC: C07C229/12 ,  C07C229/16 ,  A61K9/127 ,  A61K9/51 ,  A61K47/06 ,  A61K47/24

Abstract: The application relates to lipids of Formula (A), Formula (B) and Formula (1-1), and to lipid nanoparticles (empty or loaded LNPs) including such a lipid as well as additional lipids such as phospholipids, structural lipids, and PEG lipids. Lipid nanoparticles 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.

公开(公告)号：WO2022204288A1

公开(公告)日：2022-09-29

申请号：PCT/US2022/021560

申请日：2022-03-23

Applicant: MODERNATX, INC.

Inventor： BENENATO, Kerry E. ,  CORNEBISE, Mark ,  HENNESSY, Edward ,  SEEPERSAUD, Mohindra ,  DANNEMAN, Michael W.

IPC: C07C233/36 ,  C07C233/38 ,  C07C233/40 ,  C07C233/58 ,  C07C233/78 ,  C07C235/10 ,  C07C235/40 ,  C07C237/22 ,  C07C311/05 ,  C07C311/24 ,  C07D205/04 ,  C07D213/74 ,  C07D261/08 ,  C07D271/04 ,  C07D285/10 ,  C07D307/24 ,  C07D309/08 ,  A61K9/00

Abstract: The disclosure features novel lipids and compositions involving the same. Lipid nanoparticles (e.g., empty LNPs or loaded LNPs) include a novel lipid as well as additional lipids such as phospholipids, structural lipids, and PEG lipids. Lipid nanoparticles (e.g., empty LNPs or loaded LNPs) 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.

公开(公告)号：WO2022032154A2

公开(公告)日：2022-02-10

申请号：PCT/US2021/045038

申请日：2021-08-06

Applicant: MODERNATX, INC.

Inventor： CADETE PIRES, Ana ,  SUNG, Jean C. ,  CORNEBISE, Mark ,  HRKACH, Jeffrey

IPC: A61K9/127 ,  A61K47/18 ,  A61K47/60 ,  A61K9/51 ,  A61K31/7088 ,  A61K38/00 ,  A61K9/1273 ,  A61K9/5123 ,  C07J9/00

Abstract: The present disclosure provides LNPs comprising payload molecules, e.g., mRNA therapeutics, for the treatment of diseases or disorders, which would benefit from delivery of payload molecules to airway cells.

公开(公告)号：WO2020061367A1

公开(公告)日：2020-03-26

申请号：PCT/US2019/052009

申请日：2019-09-19

Applicant: MODERNATX, INC.

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

IPC: C07C229/16 ,  C07C233/36 ,  C07C251/38 ,  C07C255/24 ,  C07C259/06 ,  C07C279/28 ,  C07C279/36 ,  C07C311/05 ,  C07C311/51 ,  C07C311/64 ,  C07C335/08 ,  A61K47/28 ,  A61K47/14 ,  A61K47/24 ,  C07C323/25 ,  C07C275/14

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.

公开(公告)号：WO2020061332A1

公开(公告)日：2020-03-26

申请号：PCT/US2019/051959

申请日：2019-09-19

Applicant: MODERNATX, INC.

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

IPC: A61K31/575 ,  C07J9/00

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：WO2022204380A1

公开(公告)日：2022-09-29

申请号：PCT/US2022/021713

申请日：2022-03-24

Applicant: MODERNATX, INC.

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

IPC: C12N15/52

Abstract: This disclosure relates to ionizable amino lipid-based lipid nanoparticles for delivery of mRNA encoding propionyl-CoA carboxylase alpha (PCCA) and propionyl-CoA carboxylase beta (PCCB). Lipid nanoparticle/mRNA therapies of the invention increase and/or restore deficient levels of PCCA and/or PCCB expression and/or activity in subjects and are useful for the treatment of propionic academia.

公开(公告)号：WO2022204369A1

公开(公告)日：2022-09-29

申请号：PCT/US2022/021689

申请日：2022-03-24

Applicant: MODERNATX, INC.

Inventor： BENENATO, Kerry ,  CORNEBISE, Mark ,  HENNESSY, Edward ,  JAIN, Ruchi ,  PRESNYAK, Vladimir

IPC: C12N15/52 ,  A61K9/127 ,  A61K38/52 ,  A61P3/00 ,  A61P43/00 ,  C12N9/90 ,  A61K9/0019 ,  A61K9/5123 ,  C12Y504/99002

Abstract: This disclosure relates to messenger RNA (mRNA) therapy for the treatment of methylmalonic acidemia. mRNAs for use in the invention, when administered in vivo, encode methylmalonyl-CoA mutase. mRNA therapies of the disclosure increase and/or restore deficient levels of MUT expression and/or activity in subjects.

公开(公告)号：WO2019152557A1

公开(公告)日：2019-08-08

申请号：PCT/US2019/015913

申请日：2019-01-30

Applicant: MODERNATX, INC.

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

IPC: A61K9/127 ,  C12N15/88 ,  A61K47/24 ,  A61K47/28

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.