公开(公告)号：US20240327865A1

公开(公告)日：2024-10-03

申请号：US18577545

申请日：2022-07-08

Applicant: REPLICATE BIOSCIENCE, INC.

Inventor： Nathaniel Stephen WANG ,  Shigeki Joseph MIYAKE-STONER ,  Parinaz ALIAHMAD

IPC: C12N15/86 ,  A01K67/0275 ,  A61K9/00 ,  A61K9/51 ,  A61K38/00 ,  A61K39/00 ,  A61K48/00 ,  A61P31/00 ,  C07K14/005 ,  C12N7/00 ,  C12N15/88

CPC classification number: C12N15/86 ,  A01K67/0275 ,  A61K9/0019 ,  A61K9/5123 ,  A61K48/0041 ,  A61P31/00 ,  C07K14/005 ,  C12N7/00 ,  C12N15/88 ,  A01K2267/01 ,  A61K38/00 ,  A61K39/00 ,  A61K2039/53 ,  C12N2770/36122 ,  C12N2770/36134 ,  C12N2770/36143

Abstract: The present disclosure relates to the field of molecular virology, including nucleic acid molecules comprising modified viral genomes or replicons (e.g., self-replicating RNAs), pharmaceutical compositions containing the same, and the use of such nucleic acid molecules and compositions for production of desired products in cell cultures or in a living body. Also provided are methods for eliciting an immune response in a subject in need thereof, as well as methods for preventing and/or treating various health conditions.

公开(公告)号：US20240315970A1

公开(公告)日：2024-09-26

申请号：US18443959

申请日：2024-02-16

Applicant: FUJIFILM Corporation

Inventor： Shintaro TANABE ,  Masahiko YAMAMOTO ,  Kimihiko SATO ,  Motomasa TAKAHASHI ,  Kazuhiro TSUNA ,  Yasutaka TASAKI ,  Taisuke ENDO ,  Issei DOI ,  Hirofumi FUKUNAGA

IPC: A61K9/127 ,  C07C219/06 ,  C12N15/88

CPC classification number: A61K9/1272 ,  A61K9/1278 ,  C07C219/06 ,  C12N15/88

Abstract: An object of the present invention is to provide a compound or a salt thereof constituting lipid particles that can achieve a high nucleic acid encapsulation rate and excellent delivery of nucleic acids, and to provide lipid particles that can achieve a high nucleic acid encapsulation rate and excellent delivery of nucleic acids. According to an aspect of the present invention, a compound represented by Formula (1) or a salt thereof is provided.




In the formula, X represents —NR1— or —O—, R1 represents a hydrogen atom, a hydrocarbon group, or the like, R2 and R3 each independently represent a hydrogen atom, a hydrocarbon group, or the like, R4, R5, R6, R7, R8, R9, R10, R11, and R12 each independently represent a hydrogen atom or an alkyl group,



groups in any one or more pairs among R4 and R5, R10 and R5, R5 and R12, R4 and R6, R5 and R6, R6 and R7, R6 and R10, R12 and R7, and R7 and R8 may be linked to each other to form a 4- to 7-membered ring which may contain an O atom, a, b, c, and d are each independently represent an integer of 0 to 3, a+b is equal to or greater than 1, and c+d is equal to or greater than 1.

公开(公告)号：US20240294474A1

公开(公告)日：2024-09-05

申请号：US18644728

申请日：2024-04-24

Applicant: Generation Bio Co.

Inventor： Matthew G. Stanton ,  Birte Nolting

IPC: C07D211/22 ,  A61K9/51 ,  A61P3/00 ,  C12N15/88

CPC classification number: C07D211/22 ,  A61K9/5123 ,  A61P3/00 ,  C12N15/88

Abstract: Provided herein are lipids having the Formula (I):




and pharmaceutically acceptable salts thereof, wherein R1, R2, a, and b are as defined herein. Also provided herein are lipid nanoparticle (LNP) compositions comprising lipid having the Formula (I) and a capsid-free, non-viral vector (e.g., ceDNA). In one aspect of any of the aspects or embodiments herein, these LNPs can be used to deliver a capsid-free, non-viral DNA vector to a target site of interest (e.g., cell, tissue, organ, and the like).

公开(公告)号：US20240293574A1

公开(公告)日：2024-09-05

申请号：US18569623

申请日：2022-06-14

Applicant: Generation Bio Co.

Inventor： Matthew G. Stanton ,  Birte Nolting ,  Andrew Milstead

IPC: A61K47/69 ,  A61K9/51 ,  A61K31/713 ,  A61K47/54 ,  A61K48/00 ,  C07C229/12 ,  C12N15/88

CPC classification number: A61K47/6929 ,  A61K9/5123 ,  A61K31/713 ,  A61K47/542 ,  A61K47/544 ,  A61K48/0033 ,  C07C229/12 ,  C12N15/88

Abstract: Provided herein are cationic lipids having the Formula I or la: and pharmaceutically acceptable salts thereof, wherein R′, R1, R2, R3, R4, R5, R6a, R6b, X, and n are as defined herein. Also provided herein are lipid nanoparticle (LNP) compositions comprising a cationic lipid having the Formula I or la and a capsid-free, non-viral vector (e.g., ceDNA). In one aspect of any of the aspects or embodiments herein, these LNPs can be used to deliver a capsid-free, non-viral DNA vector to a target site of interest (e.g., cell, tissue, organ, and the like).

公开(公告)号：US20240279685A1

公开(公告)日：2024-08-22

申请号：US18004403

申请日：2021-07-06

Applicant: FLAGSHIP PIONEERING INNOVATIONS V, INC.

Inventor： Benhur Lee ,  Michael Travis Mee ,  Jagesh V. Shah ,  Kyle Marvin Trudeau

IPC: C12N15/88 ,  A61K9/51 ,  C07K14/005 ,  C12N5/071 ,  C12N7/00

CPC classification number: C12N15/88 ,  A61K9/5184 ,  C07K14/005 ,  C12N5/0602 ,  C12N7/00 ,  C12N2740/16043 ,  C12N2760/18222

Abstract: The present disclosure provides, at least in part, methods and compositions for producing fusosomes. In some embodiments, a producer cell for producing fusosomes comprises an exogenous or overexpresed cathepsin molecule. In some embodiments, these cells generate an increased level of active fusogen, leading to a higher proportion of fusogenically active fusosomes.

公开(公告)号：US12065664B2

公开(公告)日：2024-08-20

申请号：US17128736

申请日：2020-12-21

Applicant: RAMOT AT TEL-AVIV UNIVERSITY LTD.

Inventor： Dan Peer ,  Shiri Weinstein ,  Itai Antoine Toker ,  Srinivas Ramishetti

IPC: A61K39/395 ,  A61K9/00 ,  A61K9/51 ,  A61K31/105 ,  A61K31/7105 ,  A61K31/713 ,  A61K47/68 ,  A61K47/69 ,  A61K48/00 ,  A61P35/02 ,  C07K16/28 ,  C12N15/88

CPC classification number: C12N15/88 ,  A61K9/0019 ,  A61K9/513 ,  A61K31/105 ,  A61K31/7105 ,  A61K31/713 ,  A61K39/395 ,  A61K47/6849 ,  A61K47/6911 ,  A61K48/0066 ,  A61P35/02 ,  C07K16/2812 ,  C07K16/2896

Abstract: Disclosed are targeted lipid based particles for delivery of nucleic acid molecules (such as siRNA) to leukocytes (such as T-Cells and B-cells). Further disclosed are uses of the targeted lipid based particles for treating Leukocytes-associated diseases, such as, cancer.

公开(公告)号：US20240245764A1

公开(公告)日：2024-07-25

申请号：US18590312

申请日：2024-02-28

Applicant: CureVac SE

Inventor： Johannes LUTZ ,  Susanne RAUCH ,  Regina HEIDENREICH ,  Benjamin PETSCH

IPC: A61K39/155 ,  A61K39/00 ,  A61P31/14 ,  A61P37/04 ,  C07K14/005 ,  C12N7/00 ,  C12N15/88

CPC classification number: A61K39/155 ,  A61P31/14 ,  A61P37/04 ,  C07K14/005 ,  C12N7/00 ,  C12N15/88 ,  A61K2039/53 ,  A61K2039/6018 ,  C12N2760/18522 ,  C12N2760/18534

Abstract: The present invention is directed to an artificial nucleic acid, particularly to an artificial RNA suitable for use in treatment and/or prophylaxis of an infection with Respiratory syncytial virus (RSV) or a disorder related to such an infection. The invention further concerns a method of treating or preventing a disorder or a disease, first and second medical uses of the artificial RNA, compositions, and vaccines. Further, the invention is directed to a kit, particularly to a kit of parts, comprising the artificial RNA, compositions and vaccines.

公开(公告)号：US12043639B2

公开(公告)日：2024-07-23

申请号：US17222442

申请日：2021-04-05

Applicant: MOLECULAR TRANSFER, INC.

Inventor： Gulilat Gebeyehu ,  Joel Jessee

IPC: C07F9/10 ,  C07F9/6506 ,  C12N15/113 ,  C12N15/88 ,  C12P21/00

CPC classification number: C07F9/10 ,  C07F9/106 ,  C07F9/6506 ,  C12N15/113 ,  C12N15/88 ,  C12P21/00 ,  C12N2310/14

Abstract: New cationic lipids are provided that are useful for delivering macromolecules, such as nucleic acids, into eukaryotic cells. The lipids can be used alone, in combination with other lipids and/or in combination with other transfection enhancing reagents to prepare transfection complexes.

公开(公告)号：US20240238325A1

公开(公告)日：2024-07-18

申请号：US18288674

申请日：2022-04-28

Applicant: Japan Science and Technology Agency

Inventor： Noriko Miyamoto

IPC: A61K31/7088 ,  A61K47/10 ,  A61K47/69 ,  A61K48/00 ,  C12N15/88

CPC classification number: A61K31/7088 ,  A61K47/10 ,  A61K47/6931 ,  A61K48/0041 ,  C12N15/88

Abstract: A nucleic acid analog in which a cationic artificial nucleic acid and a hydrophilic polymer are bonded to each other. The cationic artificial nucleic acid has a backbone structure including a constituent unit in which a base is bonded to a ring structure selected from ribose and deoxyribose, and a linking structure linking two constituent units, the linking structure, in a cationic state, has a cationic group selected from the group consisting of the formulae (C1) to (C7), and the cationic artificial nucleic acid can associate by electrostatic interaction between a phosphate group of another nucleotide and the cationic group, wherein R1 to R3 represent hydrogen or an alkyl group, and may be the same or different, and Ring is a cyclic compound and may be a heterocyclic ring.

公开(公告)号：US20240218399A1

公开(公告)日：2024-07-04

申请号：US18556299

申请日：2022-10-07

Applicant: SUZHOU ABOGEN BIOSCIENCES CO., LTD.

Inventor： Dandan LING ,  Xiaobin PAN ,  Jerry ZHANG ,  Bo YING

IPC: C12N15/88

CPC classification number: C12N15/88

Abstract: Provided herein are sphingomyelin-containing compositions, and related nanoparticle compositions, lipid nanoparticles, including lipid raft nanoparticles. Also provided herein are related methods and uses of the nanoparticle compositions for delivering nucleic acid molecules to achieve enhanced nucleic acid expression.