公开(公告)号：US20240216288A1

公开(公告)日：2024-07-04

申请号：US18282640

申请日：2022-03-24

Applicant: ModernaTX, Inc.

Inventor： Kerry Benenato ,  Mark Cornebise ,  Edward Hennessy

IPC: A61K9/51 ,  A61K48/00 ,  C12N9/00 ,  C12N15/88

CPC classification number: A61K9/5123 ,  A61K48/0041 ,  C12N9/93 ,  C12N15/88 ,  C12Y604/01003

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：US10392341B2

公开(公告)日：2019-08-27

申请号：US16148822

申请日：2018-10-01

Applicant: ModernaTX, Inc.

Inventor： Kerry E. Benenato ,  Mark Cornebise

IPC: A61K9/51 ,  A61K9/00 ,  A61K48/00 ,  C12N15/63 ,  C07C229/12 ,  A61K9/127 ,  C07C271/20 ,  C07C279/12 ,  C07C279/28 ,  C07C279/32 ,  C07D233/72 ,  C07D249/04 ,  C07D263/20 ,  C07D265/33 ,  C07D271/06 ,  C07D271/10 ,  C07D207/27 ,  C07D277/38 ,  C07C275/14 ,  C07C279/24 ,  C07C311/05 ,  C07C229/16 ,  C07C335/08 ,  C07C233/36 ,  C07C235/10 ,  C07C255/24 ,  C07F9/09 ,  A61K9/16 ,  A61K38/18 ,  C07K14/505 ,  A61K47/54 ,  A61K47/69 ,  C07C227/16 ,  A61K31/7105 ,  A61K38/17 ,  C07C227/18

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：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.

公开(公告)号：US20250049727A1

公开(公告)日：2025-02-13

申请号：US18709314

申请日：2022-11-10

Applicant: ModernaTX, Inc.

Inventor： Ana Cadete Pires ,  Jean C. Sung ,  Mark Cornebise ,  Jeffrey Hrkach

IPC: A61K9/51 ,  A61K9/00 ,  A61K48/00 ,  C07C229/46 ,  C07C233/01 ,  C07C233/62 ,  C07J41/00 ,  C07J43/00

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：US20240226025A1

公开(公告)日：2024-07-11

申请号：US18282676

申请日：2022-03-24

Applicant: ModernaTX, Inc.

Inventor： Kerry Benenato ,  Mark Cornebise ,  Edward Hennessy ,  Ruchi Jain ,  Vladimir Presnyak

IPC: A61K9/51 ,  A61K38/52 ,  A61P3/00 ,  C12N9/90

CPC classification number: A61K9/5123 ,  A61K38/52 ,  A61P3/00 ,  C12N9/90 ,  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：US20240189449A1

公开(公告)日：2024-06-13

申请号：US18282647

申请日：2022-03-24

Applicant: ModernaTX, Inc.

Inventor： Kerry Benenato ,  Alicia Anne Bicknell ,  Mark Cornebise ,  Athanasios Dousis ,  Andrew Jacob Giessel ,  Edward Hennessy ,  Ruchi Jain ,  Caroline Köhrer ,  Stuart Spencer Licht ,  Kanchana Ravichandran ,  David Reid

IPC: A61K48/00 ,  A61K9/127 ,  A61K9/51 ,  A61K31/7105 ,  A61P7/00 ,  C12N9/10 ,  C12N15/67

CPC classification number: A61K48/005 ,  A61K9/1271 ,  A61K9/5123 ,  A61K31/7105 ,  A61K48/0041 ,  A61P7/00 ,  C12N9/1018 ,  C12N15/67 ,  C12Y201/03003

Abstract: This disclosure relates to mRNA therapy for the treatment of ornithine transcarbamylase deficiency (OTCD). mRNAs for use in the invention, when administered in vivo, encode human ornithine transcarbamylase (OTC). mRNA therapies of the disclosure increase and/or restore deficient levels of OTC expression and/or activity in subjects. mRNA therapies of the disclosure further decrease levels of toxic ammonia associated with deficient OTC activity in subjects.

公开(公告)号：US12151995B2

公开(公告)日：2024-11-26

申请号：US18467190

申请日：2023-09-14

Applicant: ModernaTX, Inc.

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

IPC: C07C229/12 ,  A61K9/00 ,  A61K9/127 ,  A61K9/16 ,  A61K9/51 ,  A61K31/7105 ,  A61K38/17 ,  A61K38/18 ,  A61K47/54 ,  A61K47/69 ,  A61K48/00 ,  C07C227/16 ,  C07C227/18 ,  C07C229/16 ,  C07C233/36 ,  C07C233/72 ,  C07C235/10 ,  C07C255/24 ,  C07C263/20 ,  C07C271/20 ,  C07C275/14 ,  C07C279/12 ,  C07C279/24 ,  C07C279/28 ,  C07C279/32 ,  C07C311/05 ,  C07C335/08 ,  C07D207/27 ,  C07D233/72 ,  C07D249/04 ,  C07D263/20 ,  C07D265/33 ,  C07D271/06 ,  C07D271/10 ,  C07D277/38 ,  C07F9/09 ,  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.

公开(公告)号：US11597698B2

公开(公告)日：2023-03-07

申请号：US17324426

申请日：2021-05-19

Applicant: ModernaTX, Inc.

Inventor： Kerry E. Benenato ,  Mark Cornebise ,  Edward Hennessy ,  Ellalahewage S. Kumarasinghe

IPC: C07C229/12 ,  A61K47/00 ,  A61K9/00 ,  C07C229/24 ,  A61K47/54 ,  A61K9/127 ,  A61K31/713 ,  A61K48/00

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：US10857105B2

公开(公告)日：2020-12-08

申请号：US16382423

申请日：2019-04-12

Applicant: ModernaTX, Inc.

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

IPC: A61K9/127 ,  A61K31/00 ,  A61K9/51 ,  A61K38/18 ,  A61K48/00 ,  C07C229/24

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：US10442756B2

公开(公告)日：2019-10-15

申请号：US15846084

申请日：2017-12-18

Applicant: ModernaTX, Inc.

Inventor： Kerry E. Benenato ,  Mark Cornebise

IPC: C07C229/12 ,  A61K9/127 ,  C07C271/20 ,  C07C279/12 ,  C07C279/28 ,  C07C279/32 ,  C07D233/72 ,  C07D249/04 ,  C07D263/20 ,  C07D265/33 ,  C07D271/06 ,  C07D271/10 ,  C07D207/27 ,  C07D277/38 ,  C07C275/14 ,  C07C279/24 ,  C07C311/05 ,  C07C229/16 ,  C07C335/08 ,  C07C233/36 ,  C07C233/72 ,  C07C263/20 ,  C07C235/10 ,  C07C255/24 ,  C07F9/09 ,  A61K9/16 ,  A61K38/18 ,  A61K48/00 ,  C07K14/505 ,  A61K9/00 ,  A61K47/54 ,  A61K47/69 ,  C07C227/16 ,  A61K9/51 ,  A61K31/7105 ,  A61K38/17 ,  C07C227/18

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.