公开(公告)号：US20240024349A1

公开(公告)日：2024-01-25

申请号：US18374023

申请日：2023-09-28

Applicant: DEUTSCHES KREBSFORSCHUNGSZENTRUM STIFTUNG DES ÖFFENTLICHEN RECHTS

Inventor： MATHIAS HEIKENWÄLDER ,  LARS ZENDER ,  ACHIM WEBER

IPC: A61K31/7105 ,  A61P1/16 ,  A61K9/00 ,  A61K31/192 ,  A61K31/405 ,  A61K31/616 ,  C12N15/113 ,  A61K31/713 ,  A61K31/00

CPC classification number: A61K31/7105 ,  A61P1/16 ,  A61K9/0014 ,  A61K9/0019 ,  A61K9/0053 ,  A61K31/192 ,  A61K31/405 ,  A61K31/616 ,  C12N15/113 ,  A61K31/713 ,  A61K31/00 ,  C12N2310/20 ,  C12N2310/11

Abstract: The present invention provides novel compounds that target thrombocyte activity or aggregation capacity through cellular components for the treatment of diseases associated with Non-Alcoholic Fatty Liver Disease (NAFLD). The invention provides these compounds for treating non-alcoholic steatohepatitis (NASH), a progressed stage of NAFL (non-alcoholic fatty liver), in order to avoid the development of liver cirrhosis and Hepatocellular Carcinoma (HCC). Further provided are pharmaceutical compositions, comprising the compounds of the invention, and methods for screening new NASH therapeuticals.

公开(公告)号：US20240024239A1

公开(公告)日：2024-01-25

申请号：US18222839

申请日：2023-07-17

Applicant: NEW JERSEY INSTITUTE OF TECHNOLOGY

Inventor： Xiaoyang Xu ,  Zhongyu Li

IPC: A61K9/127 ,  A61K48/00 ,  A61K47/22 ,  A61K31/711 ,  A61K31/7105 ,  A61K31/713 ,  C12P7/6436 ,  C12P17/12

CPC classification number: A61K9/1272 ,  A61K48/0033 ,  A61K48/0091 ,  A61K9/1277 ,  A61K47/22 ,  A61K31/711 ,  A61K31/7105 ,  A61K31/713 ,  C12P7/6436 ,  C12P17/12

Abstract: Ionizable cationic lipid compounds have an amine moiety from amino alcohols and a lipid moiety from a lipid synthesized via esterification. The ionizable cationic lipid compounds which comprise an amino alcohol mediated ionizable cationic lipid compound are useful for in vivo or in vitro delivery of one or more nucleic acid agents including DNA, siRNA, a microRNA, an mRNA, a RNAi, and a plasmid.

公开(公告)号：US20240016827A1

公开(公告)日：2024-01-18

申请号：US18205229

申请日：2023-06-02

Applicant: ModernaTX, Inc.

Inventor： Meredith Packer ,  Dipendra Gyawali ,  Serenus Hua ,  Gabor Butora ,  Gregory John Mercer

IPC: A61K31/7105 ,  A61K9/16

CPC classification number: A61K31/7105 ,  A61K9/1617 ,  A61K9/1641

Abstract: Provided herein are compositions and methods of reducing adduct formation.

公开(公告)号：US11874284B2

公开(公告)日：2024-01-16

申请号：US16884768

申请日：2020-05-27

Applicant: THE TRUSTEES OF COLUMBIA UNIVERSITY IN THE CITY OF NEW YORK ,  UNIVERSITÀ DEGLI STUDI DI MILANO-BICOCCA

Inventor： Francesca Bartolini ,  Maria Elena Pero ,  Guido Cavaletti

IPC: G01N33/68 ,  A61P25/02 ,  A61K45/06 ,  G01N33/574 ,  A61K31/7105 ,  A61K31/69

CPC classification number: G01N33/6896 ,  A61K31/69 ,  A61K31/7105 ,  A61K45/06 ,  A61P25/02 ,  G01N33/574 ,  G01N2800/2842 ,  G01N2800/52

Abstract: The present disclosure relates to delta-2-tubulin and its use as a biomarker for determining if a subject is at risk of developing peripheral neuropathy or if a subject has developed peripheral neuropathy. The present disclosure further relates to methods for the treatment of peripheral neuropathy in a subject and assays for identifying compounds that can be used to treat and/or prevent peripheral neuropathy.

公开(公告)号：US11865164B2

公开(公告)日：2024-01-09

申请号：US17041670

申请日：2019-03-27

Applicant: G+FLAS LIFE SCIENCES

Inventor： Sunghwa Choe ,  Mi Jin Park ,  Aiden Yeonghoon Park ,  Jung Hak Lim ,  Dong Wook Kim ,  Sungyong In ,  Jongjin Park

IPC: A61K48/00 ,  A61K38/46 ,  A61K47/64 ,  A61P35/00 ,  A61K31/7105

CPC classification number: A61K38/465 ,  A61K31/7105 ,  A61K47/64 ,  A61P35/00

Abstract: A pharmaceutical composition for treating cancer, containing a crRNA and an endonuclease as active ingredients is disclosed. The composition can be customized according to the needs of patients or cell types by specifically treating cancer cells on the basis of specific binding properties of DNA and RNA. The nuclease activity of a CRISPR PLUS system, containing both an endonuclease and an exonuclease can be activated by means of the binding between crRNA and a gene specifically found in cancer cells. Therefore, the cancer treatment effect of the composition is more specific than that of other anti-cancer agents that have been developed up till now.

公开(公告)号：US20240002854A1

公开(公告)日：2024-01-04

申请号：US18316556

申请日：2023-05-12

Applicant: MiNA Therapeutics Limited

Inventor： Pål Sætrom

IPC: C12N15/113 ,  A61K31/713 ,  A61K31/7105 ,  C12N15/11 ,  A61P35/00 ,  A61P1/16

CPC classification number: C12N15/113 ,  A61K31/713 ,  A61K31/7105 ,  C12N15/111 ,  A61P35/00 ,  A61P1/16 ,  C12N2320/30 ,  Y02A90/10 ,  C12N2310/113

Abstract: The present invention provides short activating RNA molecules which up-regulate albumin production. The present invention also provides methods of up-regulating albumin production, such methods involving the use of short activating RNA molecules capable of increasing the expression of albumin. The present invention also provides the use of the short activating RNA molecules in therapy, such as treating or preventing a hyperproliferative disorder and/or a disorder characterised by hypoalbuminemia.

公开(公告)号：US11858884B2

公开(公告)日：2024-01-02

申请号：US17566666

申请日：2021-12-30

Applicant: The Board of Regents of The University of Texas System

Inventor： Daniel J. Siegwart ,  Kejin Zhou

IPC: C07C321/14 ,  A61K9/51 ,  A61K31/7105 ,  A61K31/7125 ,  A61K47/59 ,  A61K47/60 ,  A61K47/69 ,  A61K48/00 ,  C07C323/52 ,  C07D295/15 ,  A61K47/20 ,  A61K47/22

CPC classification number: C07C321/14 ,  A61K9/513 ,  A61K9/5146 ,  A61K31/7105 ,  A61K31/7125 ,  A61K47/20 ,  A61K47/22 ,  A61K47/59 ,  A61K47/60 ,  A61K47/6931 ,  A61K48/0041 ,  C07C323/52 ,  C07D295/15

Abstract: Disclosed herein are methods for modulating an amount or activity of a gene or a gene product in a cell. The methods herein may comprise contacting a cell with a therapeutic agent assembled with a lipid composition, which lipid composition may comprise a dendrimer or dendron which may comprise one or more degradable diacyl group, in which may result in modulating the amount or activity of the gene or the gene product in the cell. The therapeutic agent modulating a gene or gene product in a cell may be sufficient to treat a disease or disorder in a subject. Further disclosed herein are pharmaceutical compositions, kits, and lipid compositions for modulating an amount or activity of a gene or a gene product in a cell.

公开(公告)号：US20230414516A1

公开(公告)日：2023-12-28

申请号：US18036676

申请日：2021-11-15

Applicant: BioNTech SE

Inventor： Bakul Subodh Bhatnagar ,  Ramin Darvari ,  Sumit Luthra ,  Serguei A. Tchessalov ,  Steffen Panzner ,  Christian Reinsch ,  Kaushik Thanki ,  Sukrut Somani

IPC: A61K9/19 ,  A61K9/16 ,  A61K9/51 ,  A61K31/7105

CPC classification number: A61K9/19 ,  A61K9/1694 ,  A61K9/1623 ,  A61K9/5123 ,  A61K31/7105

Abstract: This invention relates to enhanced stabilization and improved lyophilization methods of pharmaceutical substances.

公开(公告)号：US20230390201A1

公开(公告)日：2023-12-07

申请号：US18448254

申请日：2023-08-11

Applicant: BOARD OF REGENTS, THE UNIVERSITY OF TEXAS SYSTEM

Inventor： Elizabeth SHPALL ,  Raghu KALLURI ,  Katy REZVANI ,  Mayela MENDT ,  Valerie LEBLEU ,  Sushrut KAMERKAR

IPC: A61K9/127 ,  A61K9/00 ,  A61K31/7105 ,  A61K31/713 ,  A61K35/28 ,  A61K45/06 ,  C12N5/0775 ,  C12N15/113

CPC classification number: A61K9/1271 ,  A61K9/0019 ,  A61K31/7105 ,  A61K31/713 ,  A61K35/28 ,  A61K45/06 ,  C12N5/0663 ,  C12N5/0667 ,  C12N15/1137 ,  C12N2310/14 ,  C12N2310/141 ,  C12N2320/31 ,  C12N2320/32 ,  C12N2500/02 ,  C12N2500/90

Abstract: Provided herein are methods of manufacturing clinical grade exosomes derived from mesenchymal stem cells (MSCs). Further provided are methods of loading the exosomes with therapeutic agents, such as siRNA. Also provided herein are methods of treating diseases by administering the clinical grade exosome.

公开(公告)号：US20230381301A1

公开(公告)日：2023-11-30

申请号：US18189512

申请日：2023-03-24

Applicant: ModernaTX, Inc.

Inventor： Giuseppe Ciaramella ,  Sunny Himansu

IPC: A61K39/295 ,  A61K31/7105 ,  A61K31/7115 ,  A61K38/16 ,  C12N15/86 ,  A61K9/51 ,  A61K47/26 ,  A61K9/00

CPC classification number: A61K39/295 ,  A61K31/7105 ,  A61K31/7115 ,  A61K38/164 ,  C12N15/86 ,  A61K9/5146 ,  A61K47/26 ,  A61K9/0051 ,  A61K9/0019 ,  A61K2039/53

Abstract: Provided herein, in some embodiments, are vaccines (and vaccination methods) that include a ribonucleic acid (RNA) polynucleotide encoding a human metapneumovirus (hMPV) F protein and a RNA polynucleotide encoding a human parainfluenza virus 3 (hPIV3) F protein.