JP2022553307A - プログラニュリン関連神経変性疾患または障害の治療のためのアデノ随伴ウイルス（ａａｖ）システム - Google Patents

プログラニュリン関連神経変性疾患または障害の治療のためのアデノ随伴ウイルス（ａａｖ）システム Download PDF

Info

Publication number
JP2022553307A
JP2022553307A JP2022523408A JP2022523408A JP2022553307A JP 2022553307 A JP2022553307 A JP 2022553307A JP 2022523408 A JP2022523408 A JP 2022523408A JP 2022523408 A JP2022523408 A JP 2022523408A JP 2022553307 A JP2022553307 A JP 2022553307A
Authority
JP
Japan
Prior art keywords
aav
nucleic acid
seq
polynucleotide
promoter
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
JP2022523408A
Other languages
English (en)
Japanese (ja)
Other versions
JPWO2021081201A5 (https=
JP2022553307A5 (https=
Inventor
リー，ジュアン
ファリバー，タナズ
マンダパディ，サヴィトリ
ペノック，スティーヴン
シャーマン，マーク
ニューマーク，ジュディス
ティマース，エイドリアン
Original Assignee
アプライド ジェネティック テクノロジーズ コーポレイション
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 アプライド ジェネティック テクノロジーズ コーポレイション filed Critical アプライド ジェネティック テクノロジーズ コーポレイション
Publication of JP2022553307A publication Critical patent/JP2022553307A/ja
Publication of JPWO2021081201A5 publication Critical patent/JPWO2021081201A5/ja
Publication of JP2022553307A5 publication Critical patent/JP2022553307A5/ja
Pending legal-status Critical Current

Links

Images

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
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86—Viral vectors
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • A61P25/28—Drugs for disorders of the nervous system for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/46—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • C07K14/47—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/475—Growth factors; Growth regulators
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/575—Hormones
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C07K2319/40—Fusion polypeptide containing a tag for immunodetection, or an epitope for immunisation
    • C07K2319/42—Fusion polypeptide containing a tag for immunodetection, or an epitope for immunisation containing a HA(hemagglutinin)-tag
    • 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
    • C12N2710/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA dsDNA viruses
    • C12N2710/00011—Details
    • C12N2710/16011—Herpesviridae
    • C12N2710/16051—Methods of production or purification of viral material
    • C12N2710/16052—Methods of production or purification of viral material relating to complementing cells and packaging systems for producing virus or viral particles
    • 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
    • C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141—Use of virus, viral particle or viral elements as a vector
    • 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
    • C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141—Use of virus, viral particle or viral elements as a vector
    • C12N2750/14143—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
    • 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
    • C12N2800/00—Nucleic acids vectors
    • C12N2800/22—Vectors comprising a coding region that has been codon optimised for expression in a respective host
    • 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
    • C12N2830/00—Vector systems having a special element relevant for transcription
    • C12N2830/48—Vector systems having a special element relevant for transcription regulating transport or export of RNA, e.g. RRE, PRE, WPRE, CTE
    • 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
    • C12N2830/00—Vector systems having a special element relevant for transcription
    • C12N2830/50—Vector systems having a special element relevant for transcription regulating RNA stability, not being an intron, e.g. poly A signal

Landscapes

  • Health & Medical Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Organic Chemistry (AREA)
  • Genetics & Genomics (AREA)
  • General Health & Medical Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Zoology (AREA)
  • Medicinal Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biochemistry (AREA)
  • Biophysics (AREA)
  • Molecular Biology (AREA)
  • Biomedical Technology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Toxicology (AREA)
  • Wood Science & Technology (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Neurology (AREA)
  • Neurosurgery (AREA)
  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Plant Pathology (AREA)
  • Microbiology (AREA)
  • Virology (AREA)
  • Physics & Mathematics (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Hospice & Palliative Care (AREA)
  • Psychiatry (AREA)
  • Endocrinology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Epidemiology (AREA)
  • Immunology (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
JP2022523408A 2019-10-22 2020-10-22 プログラニュリン関連神経変性疾患または障害の治療のためのアデノ随伴ウイルス（ａａｖ）システム Pending JP2022553307A (ja)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201962924340P 2019-10-22 2019-10-22
US62/924,340 2019-10-22
PCT/US2020/056860 WO2021081201A1 (en) 2019-10-22 2020-10-22 Adeno-associated virus (aav) systems for treatment of progranulin associated neurodegeneative diseases or disorders

Publications (3)

Publication Number Publication Date
JP2022553307A true JP2022553307A (ja) 2022-12-22
JPWO2021081201A5 JPWO2021081201A5 (https=) 2023-10-30
JP2022553307A5 JP2022553307A5 (https=) 2023-10-30

Family

ID=75620215

Family Applications (1)

Application Number Title Priority Date Filing Date
JP2022523408A Pending JP2022553307A (ja) 2019-10-22 2020-10-22 プログラニュリン関連神経変性疾患または障害の治療のためのアデノ随伴ウイルス（ａａｖ）システム

Country Status (10)

Country Link
US (2) US20210147872A1 (https=)
EP (1) EP4048799A4 (https=)
JP (1) JP2022553307A (https=)
KR (1) KR20230019402A (https=)
CN (1) CN115715327A (https=)
AU (1) AU2020371662A1 (https=)
CA (1) CA3158516A1 (https=)
IL (1) IL292382A (https=)
MX (1) MX2022004812A (https=)
WO (1) WO2021081201A1 (https=)

Families Citing this family (6)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2021533126A (ja) * 2018-07-31 2021-12-02 コーネル・ユニバーシティーCornell University 臓器機能を制御するための遺伝子治療方法
KR20230128001A (ko) * 2020-12-01 2023-09-01 더 트러스티스 오브 더 유니버시티 오브 펜실베니아 엔젤만 증후군의 치료를 위한 조성물 및 이의 용도
US20240033332A1 (en) * 2020-12-17 2024-02-01 Kyoto University Agent for activating a neuron
WO2024035649A1 (en) * 2022-08-08 2024-02-15 Shape Therapeutics Inc. Compositions for progranulin expression and methods of use thereof
KR20250131772A (ko) * 2022-11-13 2025-09-03 알렉시온 파마 인터내셔널 오퍼레이션즈 리미티드 전두측두엽 치매에 대한 유전자 요법
WO2025251041A1 (en) * 2024-05-31 2025-12-04 Bluerock Therapeutics Lp METHODS AND COMPOSITIONS FOR TREATING PROGRANULIN DEFICIENCIES USING iPSC-DERIVED CELLS

Citations (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2019070894A1 (en) * 2017-10-03 2019-04-11 Prevail Therapeutics, Inc. GENE THERAPIES FOR LYSOSOMIAL DISORDERS

Family Cites Families (9)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2009089635A1 (en) * 2008-01-16 2009-07-23 Neurodyn, Inc. Treating neurodegenerative diseases with progranulin (pgrn)
JP5879256B2 (ja) * 2009-05-02 2016-03-08 ジェンザイム・コーポレーション 神経変性障害のための遺伝子治療
AU2013243948A1 (en) * 2012-04-02 2014-10-30 Moderna Therapeutics, Inc. Modified polynucleotides for the production of proteins associated with human disease
WO2016125330A1 (ja) * 2015-02-02 2016-08-11 原　英彰 網膜再生促進薬
WO2017151884A1 (en) * 2016-03-02 2017-09-08 The Children's Hospital Of Philadelphia Therapy for frontotemporal dementia
WO2018136680A1 (en) * 2017-01-19 2018-07-26 New York University Progranulin and progranulin derivatives in treating impaired fracture healing
EP3692075A4 (en) * 2017-10-03 2022-02-09 Prevail Therapeutics, Inc. GENE THERAPIES FOR LYSOSOMAL DISEASES
US20220111005A1 (en) * 2019-02-01 2022-04-14 Avrobio, Inc. Compositions and methods for treating neurocognitive disorders
BR112021015817A2 (pt) * 2019-02-22 2021-10-13 The Trustees Of The University Of Pennsylvania Vírus adeno-associado recombinante para tratamento de neurodegeneração com início na idade adulta associado a grn

Patent Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2019070894A1 (en) * 2017-10-03 2019-04-11 Prevail Therapeutics, Inc. GENE THERAPIES FOR LYSOSOMIAL DISORDERS
JP2020537544A (ja) * 2017-10-03 2020-12-24 プリベイル セラピューティクス, インコーポレーテッドPrevail Therapeutics, Inc. ライソゾーム病の遺伝子治療

Non-Patent Citations (1)

* Cited by examiner, † Cited by third party
Title
NEUROSCIENCE LETTERS, vol. 576, JPN6025025266, 2014, pages 73 - 78, ISSN: 0005781075 *

Also Published As

Publication number Publication date
WO2021081201A1 (en) 2021-04-29
MX2022004812A (es) 2023-02-23
AU2020371662A1 (en) 2022-05-12
CA3158516A1 (en) 2021-04-29
EP4048799A4 (en) 2023-11-15
CN115715327A (zh) 2023-02-24
WO2021081201A8 (en) 2022-04-14
US20240318199A1 (en) 2024-09-26
US20210147872A1 (en) 2021-05-20
IL292382A (en) 2022-06-01
EP4048799A1 (en) 2022-08-31
KR20230019402A (ko) 2023-02-08

Similar Documents

Publication Publication Date Title
US20250236887A1 (en) Adeno-associated virus (aav) systems for treatment of genetic hearing loss
US20260027235A1 (en) Adeno-associated virus vector delivery of micro-dystrophin to treat muscular dystrophy
US20240318199A1 (en) Adeno-associated virus (aav) systems for treatment of progranulin associated neurodegenerative diseases or disorders
AU2014337783B2 (en) AAV-5 pseudotyped vector for gene therapy for neurological diseases
EP2661494B1 (en) Promoters, expression cassettes and vectors for use treating achromatopsia and other diseases
EP3517135B1 (en) Promoters, expression cassettes, vectors, kits, and methods for the treatment of achromatopsia and other diseases
MX2013009604A (es) Vectores de virus asociados con adeno (aav) para uso en terapia de genes de coroideremia.
JP2025539750A (ja) 前頭側頭型認知症の遺伝子治療
KR20240095165A (ko) 근이영양증의 치료
JP2023523573A (ja) 酸性セラミダーゼ欠乏症を処置するためのベクター
US20250136651A1 (en) Recombinant optimized mecp2 cassettes and methods for treating rett syndrome and related disorders
JP2025534666A (ja) 操作された核酸調節エレメントならびにその使用方法
JP2022552014A (ja) Ｉｒｆ２ｂｐｌ遺伝子の変異に関連する障害の治療のための材料および方法
WO2026017965A1 (en) Recombinant therapeutic fxn constructs and methods of treating friedreich ataxia and related conditions
WO2025160429A1 (en) Artificial micrornas targeting snca
AU2023227586A1 (en) Materials and methods for the treatment of eif2b5 mutations and diseases resulting therefrom
EA045824B1 (ru) Выделенный модифицированный белок vp1 капсида aav5
EA052000B1 (ru) Доставка микродистрофина вектором на основе адено-ассоциированного вируса для лечения мышечной дистрофии

Legal Events

Date Code Title Description
A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20231020

A621 Written request for application examination

Free format text: JAPANESE INTERMEDIATE CODE: A621

Effective date: 20231020

A131 Notification of reasons for refusal

Free format text: JAPANESE INTERMEDIATE CODE: A131

Effective date: 20240924

A601 Written request for extension of time

Free format text: JAPANESE INTERMEDIATE CODE: A601

Effective date: 20241218

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20250324

A131 Notification of reasons for refusal

Free format text: JAPANESE INTERMEDIATE CODE: A131

Effective date: 20250624

A601 Written request for extension of time

Free format text: JAPANESE INTERMEDIATE CODE: A601

Effective date: 20250918

A02 Decision of refusal

Free format text: JAPANESE INTERMEDIATE CODE: A02

Effective date: 20260127