JP2022539238A - ウイルスベクター療法 - Google Patents

ウイルスベクター療法 Download PDF

Info

Publication number
JP2022539238A
JP2022539238A JP2022500019A JP2022500019A JP2022539238A JP 2022539238 A JP2022539238 A JP 2022539238A JP 2022500019 A JP2022500019 A JP 2022500019A JP 2022500019 A JP2022500019 A JP 2022500019A JP 2022539238 A JP2022539238 A JP 2022539238A
Authority
JP
Japan
Prior art keywords
gene therapy
compstatin
subject
moieties
complement
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
JP2022500019A
Other languages
English (en)
Japanese (ja)
Other versions
JP2022539238A5 (https=
Inventor
フランソワ，セドリック
シャイブラー，ルーカス
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.)
Apellis Pharmaceuticals Inc
Original Assignee
Apellis Pharmaceuticals Inc
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 Apellis Pharmaceuticals Inc filed Critical Apellis Pharmaceuticals Inc
Publication of JP2022539238A publication Critical patent/JP2022539238A/ja
Publication of JP2022539238A5 publication Critical patent/JP2022539238A5/ja
Pending legal-status Critical Current

Links

Images

Classifications

    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/0083—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the administration regime
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K39/00—Medicinal preparations containing antigens or antibodies
    • A61K39/395—Antibodies; Immunoglobulins; Immune serum, e.g. antilymphocytic serum
    • A61K39/39533—Antibodies; Immunoglobulins; Immune serum, e.g. antilymphocytic serum against materials from animals
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P21/00—Drugs for disorders of the muscular or neuromuscular system
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P33/00—Antiparasitic agents
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P7/00—Drugs for disorders of the blood or the extracellular fluid
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K16/00—Immunoglobulins [IG], e.g. monoclonal or polyclonal antibodies
    • C07K16/18—Immunoglobulins [IG], e.g. monoclonal or polyclonal antibodies against material from animals or humans
    • 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
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2317/00—Immunoglobulins specific features
    • C07K2317/70—Immunoglobulins specific features characterized by effect upon binding to a cell or to an antigen
    • C07K2317/76—Antagonist effect on antigen, e.g. neutralization or inhibition of binding
    • 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

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
  • General Health & Medical Sciences (AREA)
  • Medicinal Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Molecular Biology (AREA)
  • Biotechnology (AREA)
  • Biophysics (AREA)
  • General Engineering & Computer Science (AREA)
  • Zoology (AREA)
  • Biochemistry (AREA)
  • Biomedical Technology (AREA)
  • Wood Science & Technology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Microbiology (AREA)
  • Immunology (AREA)
  • Epidemiology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Virology (AREA)
  • Diabetes (AREA)
  • Tropical Medicine & Parasitology (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Neurology (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Hematology (AREA)
  • Mycology (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Peptides Or Proteins (AREA)
JP2022500019A 2019-07-05 2020-07-02 ウイルスベクター療法 Pending JP2022539238A (ja)

Applications Claiming Priority (9)

Application Number Priority Date Filing Date Title
US201962871058P 2019-07-05 2019-07-05
US62/871,058 2019-07-05
US201962871700P 2019-07-08 2019-07-08
US62/871,700 2019-07-08
US201962875925P 2019-07-18 2019-07-18
US62/875,925 2019-07-18
US201962935569P 2019-11-14 2019-11-14
US62/935,569 2019-11-14
PCT/US2020/040741 WO2021007111A1 (en) 2019-07-05 2020-07-02 Viral vector therapy

Publications (2)

Publication Number Publication Date
JP2022539238A true JP2022539238A (ja) 2022-09-07
JP2022539238A5 JP2022539238A5 (https=) 2024-03-25

Family

ID=74114222

Family Applications (1)

Application Number Title Priority Date Filing Date
JP2022500019A Pending JP2022539238A (ja) 2019-07-05 2020-07-02 ウイルスベクター療法

Country Status (6)

Country Link
US (1) US20220249706A1 (https=)
EP (1) EP3996744A4 (https=)
JP (1) JP2022539238A (https=)
AU (1) AU2020311330A1 (https=)
CA (1) CA3143257A1 (https=)
WO (1) WO2021007111A1 (https=)

Families Citing this family (7)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US11903994B2 (en) 2015-10-07 2024-02-20 Apellis Pharmaceuticals, Inc. Dosing regimens
IL269844B2 (en) * 2017-04-07 2025-01-01 Apellis Pharmaceuticals Inc Dosage regimens and related compositions and methods
WO2019118938A1 (en) 2017-12-15 2019-06-20 Apellis Pharmaceuticals, Inc. Dosing regimens and related compositions and methods
CN119264211A (zh) 2018-08-27 2025-01-07 瑞泽恩制药公司 拉曼光谱在下游纯化中的应用
EP4346843A4 (en) * 2021-05-27 2025-05-21 Apellis Pharmaceuticals, Inc. GENOME EDITING OF COMPLEMENT
CA3245921A1 (en) * 2022-03-17 2023-09-21 Adverum Biotechnologies, Inc. Antibody Cassettes of Constituent 3 Anti-Complement, Vectorization and Therapeutic Application
CN120359233A (zh) * 2022-11-24 2025-07-22 阿麦达斯制药美国有限责任公司 用于基于载体的疗法的坎普他汀类似物

Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2005070459A1 (ja) * 2004-01-22 2005-08-04 Dnavec Research Inc. 繰返し投与を伴うベクターの発現を継続させる方法
JP2016505527A (ja) * 2012-11-15 2016-02-25 アペリス・ファーマシューティカルズ・インコーポレイテッドＡｐｅｌｌｉｓ Ｐｈａｒｍａｃｅｕｔｉｃａｌｓ，Ｉｎｃ． 細胞反応性、長時間作用型または標的化コンプスタチン類似体ならびに関係する組成物および方法

Family Cites Families (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2000029033A2 (en) * 1998-11-17 2000-05-25 The General Hospital Corporation A method of genetic vector delivery
US10238700B2 (en) * 2014-01-02 2019-03-26 Genelux Corporation Oncolytic virus adjunct therapy with agents that increase virus infectivity

Patent Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2005070459A1 (ja) * 2004-01-22 2005-08-04 Dnavec Research Inc. 繰返し投与を伴うベクターの発現を継続させる方法
JP2016505527A (ja) * 2012-11-15 2016-02-25 アペリス・ファーマシューティカルズ・インコーポレイテッドＡｐｅｌｌｉｓ Ｐｈａｒｍａｃｅｕｔｉｃａｌｓ，Ｉｎｃ． 細胞反応性、長時間作用型または標的化コンプスタチン類似体ならびに関係する組成物および方法

Non-Patent Citations (1)

* Cited by examiner, † Cited by third party
Title
MOL IMMUNOL, vol. 46, JPN6024026844, 2009, pages 2911 - 2917, ISSN: 0005735443 *

Also Published As

Publication number Publication date
AU2020311330A1 (en) 2022-02-17
EP3996744A1 (en) 2022-05-18
US20220249706A1 (en) 2022-08-11
WO2021007111A1 (en) 2021-01-14
CA3143257A1 (en) 2021-01-14
EP3996744A4 (en) 2023-07-12

Similar Documents

Publication Publication Date Title
JP2022539238A (ja) ウイルスベクター療法
US20230009757A1 (en) Combination therapy for c3 inhibition
JP7077404B2 (ja) Ｒｎａ治療薬を含むエクソソーム
JP7065772B2 (ja) 低反復性ヌクレオチド配列を有する、プロリンおよびアラニン残基が豊富な反復性アミノ酸配列をコードする核酸
US20250002910A1 (en) Treatment of complement-mediated disorders
JP6621409B2 (ja) Ｃ／ＥＢＰα小分子活性化ＲＮＡ組成物
JP2021502058A (ja) Ｒｎａを編集するための組成物および方法
KR20170116055A (ko) 최적화된 인간 응고 인자 viii 유전자 발현 카세트 및 그의 용도
JP2023514570A (ja) 補体阻害のためのｒｎａ
KR102549520B1 (ko) 비염증성 식세포작용 유도 활성을 갖는 융합분자
JP2022514465A (ja) 第ｉｘ因子を発現するレンチウイルスベクターの使用
US20250075209A1 (en) Rnas for complement inhibition
US20220280598A1 (en) Complement inhibitor dosing regimens
US12251395B2 (en) RNAS for complement inhibition
US20250163454A1 (en) In vitro and in vivo protein translation via in situ circularized rnas
CN109337928B (zh) 通过过表达腺相关病毒受体提高基因治疗效率的方法
Vicidomini Development of AAV-based liver gene therapies for stable correction of Ornithine Transcarbamylase Deficiency
KR20240012480A (ko) Kcnv2 변이체 및 그것들의 사용
HK40112215A (zh) 用於c3抑制的组合疗法
Enhancer-Blocking RNA ViRus VectoRs
HK40014102A (en) Combination therapy for c3 inhibition

Legal Events

Date Code Title Description
A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20230629

A621 Written request for application examination

Free format text: JAPANESE INTERMEDIATE CODE: A621

Effective date: 20230629

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20240311

A131 Notification of reasons for refusal

Free format text: JAPANESE INTERMEDIATE CODE: A131

Effective date: 20240709

A601 Written request for extension of time

Free format text: JAPANESE INTERMEDIATE CODE: A601

Effective date: 20241007

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20241206

A02 Decision of refusal

Free format text: JAPANESE INTERMEDIATE CODE: A02

Effective date: 20250318

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20250717