JP2023551223A - Ｍｓｕｔ２を抑制するための組成物及び方法 - Google Patents

Ｍｓｕｔ２を抑制するための組成物及び方法 Download PDF

Info

Publication number
JP2023551223A
JP2023551223A JP2023531075A JP2023531075A JP2023551223A JP 2023551223 A JP2023551223 A JP 2023551223A JP 2023531075 A JP2023531075 A JP 2023531075A JP 2023531075 A JP2023531075 A JP 2023531075A JP 2023551223 A JP2023551223 A JP 2023551223A
Authority
JP
Japan
Prior art keywords
seq
sirna molecule
sirna
group
molecule
Prior art date
Legal status (The legal status is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the status listed.)
Pending
Application number
JP2023531075A
Other languages
English (en)
Japanese (ja)
Other versions
JP2023551223A5 (https=
JPWO2022109376A5 (https=
Inventor
ブライアン クレーマー
ジャンナ エム ウィーラー
パメラ マクミラン
ティモシー ジェイ ストゥロヴァス
ジェレミー ベイカー
Current Assignee (The listed assignees may be inaccurate. Google has not performed a legal analysis and makes no representation or warranty as to the accuracy of the list.)
US Department of Veterans Affairs
Original Assignee
US Department of Veterans Affairs
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Application filed by US Department of Veterans Affairs filed Critical US Department of Veterans Affairs
Publication of JP2023551223A publication Critical patent/JP2023551223A/ja
Publication of JP2023551223A5 publication Critical patent/JP2023551223A5/ja
Publication of JPWO2022109376A5 publication Critical patent/JPWO2022109376A5/ja
Pending legal-status Critical Current

Links

Classifications

    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/21—Esters, e.g. nitroglycerine, selenocyanates
    • A61K31/27—Esters, e.g. nitroglycerine, selenocyanates of carbamic or thiocarbamic acids, meprobamate, carbachol, neostigmine
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/33—Heterocyclic compounds
    • A61K31/395—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins
    • A61K31/435—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having six-membered rings with one nitrogen as the only ring hetero atom
    • A61K31/44—Non condensed pyridines; Hydrogenated derivatives thereof
    • A61K31/445—Non condensed piperidines, e.g. piperocaine
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/33—Heterocyclic compounds
    • A61K31/395—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins
    • A61K31/55—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having seven-membered rings, e.g. azelastine, pentylenetetrazole
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K45/00—Medicinal preparations containing active ingredients not provided for in groups A61K31/00 - A61K41/00
    • A61K45/06—Mixtures of active ingredients without chemical characterisation, e.g. antiphlogistics and cardiaca
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/14—Type of nucleic acid interfering nucleic acids [NA]
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2320/00—Applications; Uses
    • C12N2320/10—Applications; Uses in screening processes
    • C12N2320/11—Applications; Uses in screening processes for the determination of target sites, i.e. of active nucleic acids

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • General Health & Medical Sciences (AREA)
  • Biomedical Technology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Epidemiology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Medicinal Chemistry (AREA)
  • Molecular Biology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Organic Chemistry (AREA)
  • Zoology (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Wood Science & Technology (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • Biochemistry (AREA)
  • Physics & Mathematics (AREA)
  • Emergency Medicine (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
JP2023531075A 2020-11-23 2021-11-22 Ｍｓｕｔ２を抑制するための組成物及び方法 Pending JP2023551223A (ja)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US202063117213P 2020-11-23 2020-11-23
US63/117,213 2020-11-23
PCT/US2021/060279 WO2022109376A2 (en) 2020-11-23 2021-11-22 Compositions and methods for suppressing msut2

Publications (3)

Publication Number Publication Date
JP2023551223A true JP2023551223A (ja) 2023-12-07
JP2023551223A5 JP2023551223A5 (https=) 2024-11-29
JPWO2022109376A5 JPWO2022109376A5 (https=) 2024-11-29

Family

ID=81709733

Family Applications (1)

Application Number Title Priority Date Filing Date
JP2023531075A Pending JP2023551223A (ja) 2020-11-23 2021-11-22 Ｍｓｕｔ２を抑制するための組成物及び方法

Country Status (6)

Country Link
US (1) US20240002848A1 (https=)
EP (1) EP4247394A4 (https=)
JP (1) JP2023551223A (https=)
AU (1) AU2021383834A1 (https=)
CA (1) CA3199887A1 (https=)
WO (1) WO2022109376A2 (https=)

Citations (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20190328766A1 (en) * 2018-04-12 2019-10-31 United States Government As Represented By The Department Of Veterans Affairs Compositions and methods for suppressing msut2

Family Cites Families (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2001053312A1 (en) * 1999-12-23 2001-07-26 Hyseq, Inc. Novel nucleic acids and polypeptides
US20070009899A1 (en) * 2003-10-02 2007-01-11 Mounts William M Nucleic acid arrays for detecting gene expression in animal models of inflammatory diseases
US8598333B2 (en) * 2006-05-26 2013-12-03 Alnylam Pharmaceuticals, Inc. SiRNA silencing of genes expressed in cancer
KR101704988B1 (ko) * 2009-05-28 2017-02-08 큐알엔에이, 인크. 항바이러스 유전자에 대한 천연 안티센스 전사체의 억제에 의한 항바이러스 유전자 관련된 질환의 치료
WO2012040784A1 (en) * 2010-09-30 2012-04-05 Chipdx Llc Gene marker sets and methods for classification of cancer patients

Patent Citations (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20190328766A1 (en) * 2018-04-12 2019-10-31 United States Government As Represented By The Department Of Veterans Affairs Compositions and methods for suppressing msut2

Non-Patent Citations (1)

* Cited by examiner, † Cited by third party
Title
CHRIS R. GUTHRIE ET AL.: ""MSUT2 is a determinant of susceptibility to tau neurotoxicity"", HUMAN MOLECULAR GENETICS，, vol. 20, no. 10, JPN6025040283, 25 February 2011 (2011-02-25), pages 1989 - 1999, ISSN: 0005700340 *

Also Published As

Publication number Publication date
AU2021383834A1 (en) 2023-07-06
EP4247394A4 (en) 2025-10-29
US20240002848A1 (en) 2024-01-04
CA3199887A1 (en) 2022-05-27
AU2021383834A9 (en) 2024-06-20
WO2022109376A3 (en) 2022-07-14
EP4247394A2 (en) 2023-09-27
WO2022109376A2 (en) 2022-05-27

Similar Documents

Publication Publication Date Title
JP6236498B2 (ja) 非対称性干渉ｒｎａの組成物およびその使用
US8901097B2 (en) Methods for delivery of siRNA to the spinal cord and therapies arising therefrom
RU2426544C2 (ru) Лечение нарушений цнс
KR102825946B1 (ko) IL4Rα, TRPA1, 또는 F2RL1을 표적화하는 RNA 복합체를 사용한 아토피 피부염 및 천식의 치료
JP2014097072A5 (https=)
WO2019043027A1 (en) ANTISENSE OLIGONUCLEOTIDES FOR THE TREATMENT OF HUNTINGTON'S DISEASE
JP2023538284A (ja) Ｐｌｐ１発現を阻害するための組成物及び方法
KR102136539B1 (ko) 눈 증상의 치료 및/또는 예방을 위한 방법 및 조성물에서 sirna 및 이들의 용도
CN120752341A (zh) 靶向sod1的寡核苷酸
US9932585B2 (en) Manipulating microRNA for the management of neurological diseases or conditions and compositions related thereto
KR20200014320A (ko) Apcs의 발현을 억제하는 핵산
JP2023551223A (ja) Ｍｓｕｔ２を抑制するための組成物及び方法
US12378558B2 (en) RNAi agents for inhibiting expression of complement factor B (CFB), pharmaceutical compositions thereof, and methods of use
WO2024077262A2 (en) Methods and compositions for silencing elavl2 expression for the treatment of disease
TW202546228A (zh) 用於抑制胰島素樣生長因子１受體表達的ｒｎａ抑制劑
TW202535430A (zh) 用於抑制類活化素受體激酶－７（ＡＬＫ７）表現之ＲＮＡｉ藥劑、其組合物及使用方法
TW202430635A (zh) 用於抑制補體成分C3(C3)表現之RNAi藥劑、其醫藥組合物及使用方法
EA049851B1 (ru) РНКи-АГЕНТЫ ДЛЯ ИНГИБИРОВАНИЯ ЭКСПРЕССИИ PNPLA3, ИХ ФАРМАЦЕВТИЧЕСКИЕ КОМПОЗИЦИИ И СПОСОБЫ ПРИМЕНЕНИЯ

Legal Events

Date Code Title Description
A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20241121

A621 Written request for application examination

Free format text: JAPANESE INTERMEDIATE CODE: A621

Effective date: 20241121

A131 Notification of reasons for refusal

Free format text: JAPANESE INTERMEDIATE CODE: A131

Effective date: 20251002

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20251222

A131 Notification of reasons for refusal

Free format text: JAPANESE INTERMEDIATE CODE: A131

Effective date: 20260106