公开(公告)号：US20180222863A1

公开(公告)日：2018-08-09

申请号：US15893362

申请日：2018-02-09

Applicant: Arcturus Therapeutics, Inc.

Inventor： Joseph E. Payne ,  Padmanabh Chivukula ,  Steven P. Tanis

IPC: C07D211/60 ,  C07D207/16 ,  A61K9/127 ,  A61K47/54 ,  A61K31/713

CPC classification number: C07D211/60 ,  A61K9/1272 ,  A61K31/713 ,  A61K47/545 ,  C07D207/16

Abstract: What is described is a compound wherein R1 and R2 are the same or different, each a linear or branched alkyl consisting of 1 to 20 carbons, or a linear or branched alkenyl or alkynyl consisting of 2 to 20 carbons; L1 and L2 are the same or different, each a bond, a linear alkylene of 1-18 carbons, or a linear alkenylene consisting of 2 to 18 carbons; L3 is a bond or a linear or branched alkylene consisting of 1 to 6 carbons; L4 is a bond or a methylene; X is S or O, R3 is a linear or branched alkylene consisting of 1 to 6 carbons, and R4 and R5 are the same or different, each a hydrogen or a linear or branched alkyl consisting of 1 to 6 carbons; or a pharmaceutically acceptable salt thereof.

公开(公告)号：US20180169268A1

公开(公告)日：2018-06-21

申请号：US15387067

申请日：2016-12-21

Applicant: Arcturus Therapeutics, Inc.

Inventor： Joseph E. Payne ,  Padmanabh Chivukula ,  Priya Karmali ,  Steven P. Tanis

IPC: A61K48/00 ,  C07C333/04 ,  A61K9/51 ,  A61K47/20

CPC classification number: A61K48/0025 ,  A61K9/5123 ,  A61K47/20 ,  C07C333/04

Abstract: What is described is a compound of formula I consisting of a compound in which R1 is a branched chain alkyl consisting of 10 to 31 carbons; R2 is a linear alkyl, alkenyl, or alkynyl consisting of 2 to 20 carbons; L1 and L2 are the same or different, each a linear alkylene of 1 to 20 carbons or a linear alkenylene of 2 to 20 carbons; X1 is S or O; R3 is a linear or branched alkylene consisting of 1 to 6 carbons; and R4 and R5 are the same or different, each a hydrogen or a linear or branched alkyl consisting of 1 to 6 carbons; or a pharmaceutically acceptable salt thereof.

公开(公告)号：US09856475B2

公开(公告)日：2018-01-02

申请号：US14807223

申请日：2015-07-23

Applicant: Arcturus Therapeutics, Inc.

Inventor： Kiyoshi Tachikawa ,  Joseph E. Payne ,  Padmanabh Chivukula

IPC: C12N15/113 ,  A61K9/127 ,  A61K31/685 ,  A61K31/713 ,  C12N15/11

CPC classification number: C12N15/113 ,  A61K9/127 ,  A61K31/685 ,  A61K31/713 ,  C12N15/111 ,  C12N2310/14 ,  C12N2310/32 ,  C12N2310/323 ,  C12N2320/32 ,  C12N2320/34

Abstract: This invention provides pharmaceutical compositions containing a UNA oligomer targeted to TTR and a pharmaceutically acceptable carrier. The compositions can be used in methods for treating or preventing TTR-related amyloidosis in a primate. The compositions, upon administering a single dose to the primate, can reduce TTR protein in the primate for a period of days to weeks.

公开(公告)号：US09670152B2

公开(公告)日：2017-06-06

申请号：US15347647

申请日：2016-11-09

Applicant: Arcturus Therapeutics, Inc.

Inventor： Joseph E. Payne ,  Padmanabh Chivukula

IPC: C07C323/25 ,  C07C333/04 ,  C07C271/22 ,  A61K47/20 ,  A61K47/18

CPC classification number: C07C323/52 ,  A61K47/18 ,  A61K47/20 ,  C07C235/12 ,  C07C237/12 ,  C07C271/22 ,  C07C323/25 ,  C07C323/60 ,  C07C333/04 ,  C07J41/0055 ,  C11C3/04 ,  C12N15/111 ,  C12N15/113 ,  C12N15/1137 ,  C12N2310/14 ,  C12N2320/32

Abstract: What is described is a compound of formula I wherein R is a linear alkyl of 1 to 12 carbons, or a linear alkenyl or alkynyl of 2 to 12 carbons; L is a linear alkylene or alkenylene of 5 to 18 carbons; X is —CO—O— or —O—CO—; Y is S or O; R1 is a linear or branched alkylene consisting of 1 to 6 carbons; and R2 and R3 are the same or different, consisting of a hydrogen or a linear or branched alkyl consisting of 1 to 6 carbons; and n is 1-6; or a salt, a solvate, or a pharmaceutical formulation thereof.

公开(公告)号：US20150322435A1

公开(公告)日：2015-11-12

申请号：US14807223

申请日：2015-07-23

Applicant: Arcturus Therapeutics, Inc.

Inventor： Kiyoshi Tachikawa ,  Joseph E. Payne ,  Padmanabh Chivukula

IPC: C12N15/113 ,  A61K9/127 ,  A61K31/713

CPC classification number: C12N15/113 ,  A61K9/127 ,  A61K31/685 ,  A61K31/713 ,  C12N15/111 ,  C12N2310/14 ,  C12N2310/32 ,  C12N2310/323 ,  C12N2320/32 ,  C12N2320/34

Abstract: This invention provides pharmaceutical compositions containing a UNA oligomer targeted to TTR and a pharmaceutically acceptable carrier. The compositions can be used in methods for treating or preventing TTR-related amyloidosis in a primate. The compositions, upon administering a single dose to the primate, can reduce TTR protein in the primate for a period of days to weeks.

Abstract translation: 本发明提供含有靶向TTR的UNA寡聚物和药学上可接受的载体的药物组合物。 组合物可用于治疗或预防灵长类动物中TTR相关淀粉样变性的方法。 组合物在向灵长类动物施用单一剂量时可以将灵长类动物中的TTR蛋白质减少数天至数周。

公开(公告)号：US20150307880A1

公开(公告)日：2015-10-29

申请号：US14667678

申请日：2015-03-25

Applicant: Arcturus Therapeutics, Inc.

Inventor： Kiyoshi Tachikawa ,  Joseph E. Payne ,  Padmanabh Chivukula

IPC: C12N15/113 ,  A61K9/127 ,  A61K31/685

CPC classification number: C12N15/113 ,  A61K9/127 ,  A61K31/685 ,  A61K31/713 ,  C12N2310/14 ,  C12N2310/32 ,  C12N2310/323 ,  C12N2310/344

Abstract: This invention provides UNA oligomers for selectively inhibiting V30M TTR expression, which can be used in treating amyloidosis. The UNA oligomers can have a first strand and a second strand, each of the strands being 19-29 monomers in length, the monomers being UNA monomers and nucleic acid monomers. Embodiments include pharmaceutical compositions and methods for treating or preventing TTR-related amyloidosis by administering a UNA oligomer to a subject.

Abstract translation: 本发明提供用于选择性抑制V30M TTR表达的UNA寡聚体，其可用于治疗淀粉样变性。 UNA低聚物可以具有第一链和第二链，每条链长度为19-29个单体，单体为UNA单体和核酸单体。 实施方案包括通过向受试者施用UNA寡聚体来治疗或预防TTR相关淀粉样变性的药物组合物和方法。

公开(公告)号：US12220455B2

公开(公告)日：2025-02-11

申请号：US18345893

申请日：2023-06-30

Applicant: Arcturus Therapeutics, Inc.

Inventor： Sean Michael Sullivan ,  Daiki Matsuda ,  Kiyoshi Tachikawa ,  Padmanabh Chivukula ,  Priya Prakash Karmali ,  Jared Henry Davis ,  Yanjie Bao ,  Amit Sagi

IPC: A61K39/215 ,  A61K9/51 ,  A61K38/00 ,  A61K39/00 ,  A61K39/12 ,  A61K47/10 ,  A61K47/20 ,  A61K47/26 ,  C07K14/005 ,  C07K14/18 ,  C12N7/00 ,  C12N15/86

Abstract: Provided herein are nucleic acid molecules encoding viral replication proteins and antigenic coronavirus proteins or fragments thereof. Also provided herein are compositions that include nucleic acid molecules encoding viral replication and antigenic proteins, and lipids. Nucleic acid molecules provided herein are useful for inducing immune responses.

公开(公告)号：US12070509B2

公开(公告)日：2024-08-27

申请号：US17246558

申请日：2021-04-30

Applicant: Arcturus Therapeutics, Inc.

Inventor： Carlos G. Perez-Garcia ,  Kiyoshi Tachikawa ,  Daiki Matsuda ,  Padmanabh Chivukula ,  Priya Prakash Karmali ,  Yanjie Bao ,  Jerel Boyd Lee Vega ,  Rajesh Mukthavaram ,  Amit Sagi

IPC: A61K48/00 ,  A61P11/00 ,  C07K14/47

CPC classification number: A61K48/0033 ,  A61K48/005 ,  A61P11/00 ,  C07K14/4702

Abstract: Nucleotides encoding a Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) protein are provided herein. Also describe are mRNA constructs that can be used to express CFTR protein in vitro or in vivo. The mRNA constructs can be formulated in a lipid formulation and administered via inhalation to treat cystic fibrosis.

公开(公告)号：US11939363B2

公开(公告)日：2024-03-26

申请号：US17812576

申请日：2022-07-14

Applicant: ARCTURUS THERAPEUTICS, INC.

Inventor： Pattraranee Limphong ,  Kiyoshi Tachikawa ,  Padmanabh Chivukula ,  Daiki Matsuda ,  Arisa Cale

IPC: C07K14/505 ,  A61K31/7115 ,  A61K38/17 ,  A61K38/18 ,  A61K38/48 ,  C07K14/575 ,  C07K14/745 ,  C07K14/81 ,  A61K38/00 ,  C12Q1/68

CPC classification number: C07K14/505 ,  A61K31/7115 ,  A61K38/1709 ,  A61K38/1816 ,  A61K38/482 ,  A61K38/4846 ,  C07K14/575 ,  C07K14/745 ,  C07K14/81 ,  A61K38/00

Abstract: A range of therapeutic mRNA molecules expressible to provide a target polypeptide or protein. The RNA molecules can contain one or more 5-methoxyuridines and 5-methylcytidines. Further provided are DNA templates, which can be transcribed to provide a target mRNA, and can have altered nucleotides, such as reduced deoxyadenosines. Also provided are processes for making the therapeutic mRNA molecules. The RNA molecules can be translated in vitro or in vivo to provide an active polypeptide or protein. The RNA molecules can be included in a composition used for preventing, treating, or ameliorating at least one symptom of a disease or condition in a subject in need thereof.

公开(公告)号：US11737979B2

公开(公告)日：2023-08-29

申请号：US16823212

申请日：2020-03-18

Applicant: Arcturus Therapeutics, Inc.

Inventor： Priya Karmali ,  Padmanabh Chivukula ,  Joseph E. Payne ,  Yanjie Bao

IPC: A61K47/69 ,  B82Y5/00 ,  A61K9/127 ,  A61K9/14 ,  A61K47/60

CPC classification number: A61K9/1277 ,  A61K9/145 ,  A61K47/60 ,  A61K47/69 ,  B82Y5/00

Abstract: A method of producing a lipid-encapsulated RNA nanoparticle, comprising the steps a) flowing an aqueous solution comprising an RNA through a 1st tube having an inner diameter (ID) of between about 0.1″ and 0.132″; b) flowing an ethanol solution comprising lipids through a 2nd tube having an ID of between about 0.005″ and 0.02″ at one third the flow rate of the aqueous solution through the 1st tube, wherein the lipids comprise a cationic lipid; and c) mixing the ethanol solution with the aqueous solution by flowing the ethanol solution and the aqueous solution into a mixing module consisting of the 2nd tube perpendicularly joined to the 1st tube; wherein the mixing produces an output solution flowing in the 1st tube comprising a turbulent flow of the RNA and the lipids in between about 10% to 75% ethanol v/v, and wherein the lipid-encapsulated RNA nanoparticles have a bilayer structure.