CA3143257A1 - Viral vector therapy - Google Patents

Viral vector therapy Download PDF

Info

Publication number
CA3143257A1
CA3143257A1 CA3143257A CA3143257A CA3143257A1 CA 3143257 A1 CA3143257 A1 CA 3143257A1 CA 3143257 A CA3143257 A CA 3143257A CA 3143257 A CA3143257 A CA 3143257A CA 3143257 A1 CA3143257 A1 CA 3143257A1
Authority
CA
Canada
Prior art keywords
gene therapy
moiety
compstatin analog
compstatin
subject
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
CA3143257A
Other languages
English (en)
French (fr)
Inventor
Cedric Francois
Lukas Scheibler
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 CA3143257A1 publication Critical patent/CA3143257A1/en
Pending legal-status Critical Current

Links

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)
CA3143257A 2019-07-05 2020-07-02 Viral vector therapy Pending CA3143257A1 (en)

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 (1)

Publication Number Publication Date
CA3143257A1 true CA3143257A1 (en) 2021-01-14

Family

ID=74114222

Family Applications (1)

Application Number Title Priority Date Filing Date
CA3143257A Pending CA3143257A1 (en) 2019-07-05 2020-07-02 Viral vector therapy

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

Family Cites Families (4)

* 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
JP2007112715A (ja) * 2004-01-22 2007-05-10 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
MX366404B (es) * 2012-11-15 2019-07-08 Apellis Pharmaceuticals Inc Analogos de compstatina de celula reactiva, de acción prolongada u objetivos y composiciones y metodos relacionados.
US10238700B2 (en) * 2014-01-02 2019-03-26 Genelux Corporation Oncolytic virus adjunct therapy with agents that increase virus infectivity

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
JP2022539238A (ja) 2022-09-07
EP3996744A4 (en) 2023-07-12

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