公开(公告)号：US20250136953A1

公开(公告)日：2025-05-01

申请号：US18909701

申请日：2024-10-08

Applicant: Arcturus Therapeutics, Inc.

Inventor： Padmanabh Chivukula ,  Priya Prakash Karmali ,  Kiyoshi Tachikawa ,  Suezanne E. Parker ,  Marciano Rodriguez Sablad ,  Pattraranee Limphong ,  Yanjie Bao ,  Jerel Boyd Lee Vega

IPC: C12N9/10 ,  A61K9/1271 ,  A61K9/1272 ,  A61K31/221 ,  A61K39/00 ,  A61K48/00

Abstract: The present disclosure describes compositions and methods for treating ornithine transcarbamylase (OTC) deficiency. The compositions include a lipid formulation and messenger RNA (mRNA) encoding an OTC enzyme. The lipid formulations can comprise an ionizable cationic lipid in a lipid nanoparticle encapsulating the mRNA.

公开(公告)号：US12220485B2

公开(公告)日：2025-02-11

申请号：US18457090

申请日：2023-08-28

Applicant: Arcturus Therapeutics, Inc.

Inventor： Priya Karmali ,  Padmanabh Chivukula ,  Joseph E. Payne ,  Yanjie Bao ,  Michael Figa ,  Scott A. Roberts ,  Andreas Wagner

IPC: A61K47/69 ,  A61K9/14 ,  A61K47/60 ,  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.

公开(公告)号：US11685921B2

公开(公告)日：2023-06-27

申请号：US16724122

申请日：2019-12-20

Applicant: Arcturus Therapeutics, Inc.

Inventor： Pattraranee Limphong ,  Kiyoshi Tachikawa ,  Christine Esau ,  Padmanabh Chivukula

IPC: C12N15/11 ,  C12N15/113 ,  A61K9/127 ,  A61K47/69

CPC classification number: C12N15/1131 ,  A61K9/1271 ,  A61K47/6911 ,  C12N2310/14 ,  C12N2310/315 ,  C12N2310/321 ,  C12N2310/322 ,  C12N2310/323 ,  C12N2310/344 ,  C12N2310/3515 ,  C12N2320/32

Abstract: This invention encompasses compounds and compositions useful in methods for medical therapy, in general, for inhibiting Hepatitis B virus in a subject. The compounds have a first strand and a second strand, each of the strands being 19-29 monomers in length, the monomers comprising UNA monomers and nucleic acid monomers, and the compounds are targeted to a sequence of an HBV genome.

公开(公告)号：US20220370624A1

公开(公告)日：2022-11-24

申请号：US17737884

申请日：2022-05-05

Applicant: Arcturus Therapeutics, Inc.

Inventor： Kumar Rajappan ,  Steven Tanis ,  Rajesh Mukthavaram ,  Amit Sagi ,  Priya Prakash Karmali ,  Padmanabh Chivukula

IPC: A61K47/54 ,  A61K47/26 ,  C07K7/04 ,  C07K14/00 ,  C07K19/00 ,  A61P35/00 ,  A61K9/00

Abstract: A lipid composition containing a nucleic acid, wherein the lipid composition comprises a peptide-lipid conjugate, is provided. The peptide of the peptide-lipid conjugates can be from 4 to 52 amino acids in length. Methods of using the lipid composition in the in vivo delivery of nucleic acids are further provided.

公开(公告)号：US11015204B2

公开(公告)日：2021-05-25

申请号：US15994683

申请日：2018-05-31

Applicant: Arcturus Therapeutics, Inc.

Inventor： Pattraranee Limphong ,  Carlos G. Perez-Garcia ,  Kiyoshi Tachikawa ,  Padmanabh Chivukula ,  Arisa Cale ,  Angel I-Jou Leu ,  Jared Davis

IPC: C12N15/52 ,  C12N15/82 ,  C07K14/805 ,  C07K14/775 ,  C07K14/75 ,  C07K14/47 ,  C07K14/415 ,  C07K14/505 ,  A61K38/01 ,  A61K38/17 ,  A61K38/42 ,  A61K38/36 ,  A61K38/18

Abstract: This invention provides expressible polynucleotides, which can express a target protein or polypeptide. Synthetic mRNA constructs for producing a protein or polypeptide can contain one or more 5′ UTRs, where a 5′ UTR may be expressed by a gene of a plant. In some embodiments, a 5′ UTR may be expressed by a gene of a member of Arabidopsis genus. The synthetic mRNA constructs can be used as pharmaceutical agents for expressing a target protein or polypeptide in vivo.

公开(公告)号：US20200140380A9

公开(公告)日：2020-05-07

申请号：US15925670

申请日：2018-03-19

Applicant: Arcturus Therapeutics, Inc.

Inventor： Joseph E. Payne ,  Padmanabh Chivukula

IPC: C07C323/52 ,  C12N15/113

Abstract: What is described is a method of synthesis of the compound of formula 1A, or a salt thereof, wherein R3 is a linear or branched alkene of 1, 2, 3, 4, 5 or 6 carbons; R4 and R5 are the same or different, each a hydrogen, or a linear or branched alkyl of 1, 2, 3, 4, 5 or 6 carbons; and L3 is a bond or an alkane of 1, 2, 3, 4, 5 or 6 carbons.

公开(公告)号：US09982259B2

公开(公告)日：2018-05-29

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 ,  A61K31/713 ,  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.

公开(公告)号：US09962449B2

公开(公告)日：2018-05-08

申请号：US15224474

申请日：2016-07-29

Applicant: Arcturus Therapeutics, Inc.

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

IPC: A61K31/713 ,  C12N15/113 ,  A61K47/48 ,  A61K9/00

CPC classification number: A61K47/554 ,  A61K9/0019 ,  A61K31/713 ,  A61K47/549 ,  C12N15/113 ,  C12N2310/14 ,  C12N2310/3515 ,  C12N2320/32

Abstract: Described herein is a compound having the structure of formula I, II, or III, wherein R comprises a double stranded RNA molecule, and L1, L2, and L3 independently for each occurrence comprise a ligand selected from the group consisting of a carbohydrate, a cholesteryl, or a peptide; a pharmaceutically accepted salt or pharmaceutical composition thereof; and a method of making the compound.

公开(公告)号：US09951002B2

公开(公告)日：2018-04-24

申请号：US15818424

申请日：2017-11-20

Applicant: Arcturus Therapeutics, Inc.

Inventor： Joseph E. Payne ,  Padmanabh Chivukula

IPC: C07C323/52 ,  C12N15/113

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 having the formula wherein R1 is branched alkyl of the structure (CH3(CH2)m)2CH—, wherein m is 2 or 3; R2 is a linear alkyl of 1, 2, 3, 4, 5, 6, 7, 8, or 9 carbons, a branched alkyl of 3, 4, 5, 6, 7, 8, or 9 carbons, or an alkenyl or alkynyl of 2, 3, 4, 5, 6, 7, 8, 9, 10 or 11 carbons; R3 is —(CH2)p—, wherein p is 2, 3, 4, 5 or 6; R4 and R5 are the same or different, each a hydrogen, or a linear or branched alkyl of 1, 2, 3, 4, 5 or 6 carbons; L1 and L2 are the same or different, of the structure —(CH2)n—, wherein n is 7, 8, 9, 10, 11, 12, 13, 14, 15, 16, 17, or 18; L3 is a bond; X1 is —CO—O— whereby -L2-CO—O—R2 is formed; X2 is S or O; and X3 is —CO—O— whereby -L1-CO—O—R1 is formed; and wherein m+n+p is 11, 12, 13, 14, 15, 16, 17, 18, 19, 20, 21, 22, 23, 24, 25, 26, or 27; or a pharmaceutically acceptable salt thereof.

公开(公告)号：US09834510B2

公开(公告)日：2017-12-05

申请号：US15393840

申请日：2016-12-29

Applicant: Arcturus Therapeutics, Inc.

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

IPC: C07C333/04 ,  C07C271/22

CPC classification number: C07C333/04 ,  C07C271/22

Abstract: What is described is a compound of formula I wherein X is an ethene, or an unsubstituted or substituted arene or heteroarene; Y is a bond, an ethene, or an unsubstituted or substituted arene or heteroarene; Z is S or O; L is a linear or branched alkylene of 1 to 6 carbons; R1 and R2 are independently a linear or branched alkyl or alkenyl of 1 to 18 carbons; R3 and R4 are independently a linear or branched alkyl of 1 to 6 carbons; n is 0 to 6; and m, p, q, and r are independently 1-18; or a pharmaceutically acceptable salt thereof.