公开(公告)号：US12116603B2

公开(公告)日：2024-10-15

申请号：US17091735

申请日：2020-11-06

Applicant: University of Florida Research Foundation, Inc.

Inventor： Yunwei Charles Cao ,  Tian Jiang

IPC: C12N9/22 ,  C12N15/01 ,  C12N15/10

CPC classification number: C12N9/22 ,  C12N15/01 ,  C12N15/102 ,  C12N2310/52 ,  C12Y301/27005

Abstract: Disclosed herein are improved nanozymes for targeting RNA. The disclosed nanozymes are synthesized using recombinant RNase A with site-specific cysteine-substituted mutations that can be covalently functionalized with a length-tunable multi-thiol tether and then loaded onto gold particles through multiple gold-sulfur bonds. This new RNase A loading mechanism is site specific, and it allows high-density loading of alkylthiol modified DNA oligonucleotides. The disclosed nanozymes can also include additional capturer strands and/or involve DNA-recombinant-RNase-A unibodies to further increase the nanozyme's enzymatic activity and target selectivity. Also disclosed are functional on-off switchable nanozymes to control nanozyme activity. In some embodiments, the disclosed nanozyme are core-free hollow forms. The removal of the inorganic nanoparticle cores from nanozymes can effectively eliminate the potential long-term toxicity induced by the core, and also creates a cavity for loading and delivery of small molecule drugs.

公开(公告)号：US11987904B2

公开(公告)日：2024-05-21

申请号：US16081635

申请日：2017-03-02

Applicant: Aptamer Group Limited

Inventor： Shuji Ikeda ,  Shozo Fujita ,  Tsuyoshi Fujihara

IPC: C40B40/06 ,  C12N15/10 ,  C12N15/115 ,  C40B40/08

CPC classification number: C40B40/08 ,  C12N15/1093 ,  C12N15/115 ,  C12N2310/16 ,  C12N2310/52 ,  C12N2320/11 ,  C12N2330/31

Abstract: Oligonucleotide constructs are described, each comprising a functional element and a coding element, wherein the functional element comprises a functional sequence, the functional sequence comprising a sequence of nucleotides in which one or more, or each, nucleotide is modified and the coding element comprises a coding sequence, the coding sequence comprising a sequence of nucleotides which do not contain the modifications of the functional sequence, wherein the coding sequence encodes the sequence structure of the functional sequence.

公开(公告)号：US20190106452A1

公开(公告)日：2019-04-11

申请号：US15554360

申请日：2016-03-09

Applicant: UNIVERSITY OF KENTUCKY RESEARCH FOUNDATION

Inventor： Peixuan Guo ,  Carlo M. Croce ,  Tae Jin Lee ,  Farzin Haque ,  Hui Li

IPC: C07H21/02 ,  A61P35/00 ,  A61K47/55 ,  C12N15/113 ,  C12N15/115

CPC classification number: C07H21/02 ,  A61K47/551 ,  A61K47/6929 ,  A61P35/00 ,  B82Y5/00 ,  C12N15/111 ,  C12N15/113 ,  C12N15/115 ,  C12N2310/113 ,  C12N2310/14 ,  C12N2310/3231 ,  C12N2310/52 ,  C12N2320/32

Abstract: The presently-disclosed subject matter relates to an artificial RNA nanostructure molecule and method to treat brain tumor in a subject. More particularly, the presently disclosed subject matter relates to a RNA nanostructure containing a multiple branched RNA nanoparticle, a brain tumor targeting module, and an effective amount of a therapeutic agent. Further, the presently disclosed subject matter relates to a method of using the RNA nanostructure composition to treat brain tumor in a subject having or at risk of having brain tumor.

公开(公告)号：US20180030444A1

公开(公告)日：2018-02-01

申请号：US15698754

申请日：2017-09-08

Applicant: Translate Bio MA, Inc.

Inventor： Eugen Uhlmann ,  Romesh Subramanian ,  Arthur M. Krieg

IPC: C12N15/113 ,  C12N15/11

CPC classification number: C12N15/113 ,  C12N15/111 ,  C12N2310/11 ,  C12N2310/141 ,  C12N2310/315 ,  C12N2310/3231 ,  C12N2310/3341 ,  C12N2310/341 ,  C12N2310/3519 ,  C12N2310/51 ,  C12N2310/52 ,  C12N2320/30 ,  C12N2330/30

Abstract: The disclosure provides multimeric oligonucleotide compounds, comprising two or more target-specific oligonucleotides (e.g., antisense oligonucleotides (ASOs)), each being resistant to cleavage, and linked together by a cleavable linker. In particular, two or more linked target-specific oligonucleotides, each to a different target, allows concomitant inhibition of multiple genes' expression levels, while exhibiting favorable pharmacokinetic and pharmacodynamic properties. Methods of making and uses of the described compounds are also provided.

公开(公告)号：US20170312367A1

公开(公告)日：2017-11-02

申请号：US15419593

申请日：2017-01-30

Applicant: UNIVERSITY OF MASSACHUSETTS

Inventor： Anastasia Khvorova ,  Matthew Hassler ,  Julia Alterman

IPC: A61K47/26 ,  C07H21/04 ,  C07H3/08 ,  C07H99/00 ,  A61K31/7084

CPC classification number: A61K47/26 ,  A61K31/7084 ,  A61K47/549 ,  A61K47/55 ,  A61K47/551 ,  C07H3/08 ,  C07H21/04 ,  C07H99/00 ,  C12N15/111 ,  C12N15/113 ,  C12N2310/11 ,  C12N2310/14 ,  C12N2310/3513 ,  C12N2310/3515 ,  C12N2310/52

Abstract: Provided herein are branched oligonucleotides exhibiting efficient and specific tissue distribution, cellular uptake, minimum immune response and off-target effects, without formulation.

公开(公告)号：US09771600B2

公开(公告)日：2017-09-26

申请号：US15368570

申请日：2016-12-02

Applicant: Caribou Biosciences, Inc.

Inventor： Paul Daniel Donohoue ,  Andrew Paul May

IPC: C12N15/113 ,  C12N15/90 ,  C12N9/22

CPC classification number: C12N15/902 ,  C12N9/22 ,  C12N15/10 ,  C12N15/113 ,  C12N2310/20 ,  C12N2310/51 ,  C12N2310/52

Abstract: The present disclosure provides engineered polynucleotide sequences that form scaffolds and nucleoprotein complexes comprising such engineered polynucleotide sequences that form scaffolds and nucleic acid binding proteins. Nucleic acid sequences encoding the engineered polynucleotide sequences that form scaffolds, as well as expression cassettes, vectors and cells comprising such polynucleotide sequences, are described. A variety of methods for making and using the engineered polynucleotide sequences that form scaffolds are also disclosed.

公开(公告)号：US20170233741A1

公开(公告)日：2017-08-17

申请号：US15366509

申请日：2016-12-01

Applicant: Idera Pharmaceuticals, Inc.

Inventor： Ekambar R. Kandimalla ,  Daqing Wang ,  Ireneusz Nowak ,  Sudhir Agrawal

IPC: C12N15/117 ,  A61K45/06 ,  A61K31/7125

CPC classification number: C12N15/117 ,  A61K31/7115 ,  A61K31/7125 ,  A61K45/06 ,  C12N2310/17 ,  C12N2310/31 ,  C12N2310/317 ,  C12N2310/321 ,  C12N2310/3341 ,  C12N2310/336 ,  C12N2310/51 ,  C12N2310/52 ,  Y02A50/401 ,  Y02A50/411 ,  Y02A50/414 ,  A61K2300/00

Abstract: The invention provides novel immune regulatory oligonucleotides (IRO) as antagonist of TLRs and methods of use thereof. These IROs have unique sequences that inhibit or suppress TLR-mediated signaling in response to a TLR ligand or TLR agonist. The methods may have use in the prevention and treatment of cancer, an autoimmune disorder, airway inflammation, inflammatory disorders, infectious disease, skin disorders, allergy, asthma or a disease caused by a pathogen.

公开(公告)号：US09732340B2

公开(公告)日：2017-08-15

申请号：US14698239

申请日：2015-04-28

Applicant: Translate Bio MA, Inc.

Inventor： Eugen Uhlmann ,  Markus Weber ,  Romesh Subramanian ,  Thomas Dino Rockel ,  Arthur M. Krieg

IPC: C12N15/113

CPC classification number: C12N15/113 ,  C12N2310/11 ,  C12N2310/113 ,  C12N2310/315 ,  C12N2310/3231 ,  C12N2310/341 ,  C12N2310/346 ,  C12N2310/51 ,  C12N2310/52

Abstract: The disclosure provides multimeric oligonucleotide compounds, comprising two or more target-specific oligonucleotides (e.g., antisense oligonucleotides (ASOs)), each being resistant to cleavage, and linked together by a cleavable linker. In particular, two or more linked target-specific oligonucleotides, each to a different target, allows concomitant inhibition of multiple genes' expression levels, while exhibiting favorable pharmacokinetic and pharmacodynamic properties. Methods of making and uses of the described compounds are also provided.

公开(公告)号：US20170159073A1

公开(公告)日：2017-06-08

申请号：US15368570

申请日：2016-12-02

Applicant: Caribou Biosciences, Inc.

Inventor： Paul Daniel Donohoue ,  Andrew Paul May

IPC: C12N15/90 ,  C12N9/22

CPC classification number: C12N15/902 ,  C12N9/22 ,  C12N15/10 ,  C12N15/113 ,  C12N2310/20 ,  C12N2310/51 ,  C12N2310/52

Abstract: The present disclosure provides engineered polynucleotide sequences that form scaffolds and nucleoprotein complexes comprising such engineered polynucleotide sequences that form scaffolds and nucleic acid binding proteins. Nucleic acid sequences encoding the engineered polynucleotide sequences that form scaffolds, as well as expression cassettes, vectors and cells comprising such polynucleotide sequences, are described. A variety of methods for making and using the engineered polynucleotide sequences that form scaffolds are also disclosed.

公开(公告)号：US20170121708A1

公开(公告)日：2017-05-04

申请号：US15022530

申请日：2014-09-17

Applicant: THE USA, AS REPRESENTED BY THE SECRETARY, DEPARTMENT OF HEALTH & HUMAN SERVICES

Inventor： Bruce A. Shapiro ,  Kirill A. Afonin ,  Mathias D. Viard ,  Angelica N. Martins

IPC: C12N15/113 ,  A61K31/713

CPC classification number: C12N15/113 ,  A61K31/713 ,  A61K47/549 ,  A61K47/6929 ,  B82Y5/00 ,  C12N15/111 ,  C12N15/1132 ,  C12N15/87 ,  C12N2310/14 ,  C12N2310/351 ,  C12N2310/3519 ,  C12N2310/51 ,  C12N2310/52 ,  C12N2320/32 ,  C12N2330/30

Abstract: The instant invention provides RNA nanoparticles and R/DNA chimeric nanoparticles comprising one or more functionalities. The multifunctional RNA nanoparticles are suitable for therapeutic or diagnostic use in a number of diseases or disorders.