JP2024540470A - 可溶性ｃｄ５９を発現する遺伝子療法ベクターにより地図状萎縮を治療する方法 - Google Patents

可溶性ｃｄ５９を発現する遺伝子療法ベクターにより地図状萎縮を治療する方法 Download PDF

Info

Publication number
JP2024540470A
JP2024540470A JP2024529310A JP2024529310A JP2024540470A JP 2024540470 A JP2024540470 A JP 2024540470A JP 2024529310 A JP2024529310 A JP 2024529310A JP 2024529310 A JP2024529310 A JP 2024529310A JP 2024540470 A JP2024540470 A JP 2024540470A
Authority
JP
Japan
Prior art keywords
scd59
protein
amd
vector
drp
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
JP2024529310A
Other languages
English (en)
Japanese (ja)
Other versions
JPWO2023089564A5 (https=
JP2024540470A5 (https=
Inventor
ロジャース，アダム
Original Assignee
ヤンセン バイオテツク，インコーポレーテツド
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 ヤンセン バイオテツク，インコーポレーテツド filed Critical ヤンセン バイオテツク，インコーポレーテツド
Publication of JP2024540470A publication Critical patent/JP2024540470A/ja
Publication of JPWO2023089564A5 publication Critical patent/JPWO2023089564A5/ja
Publication of JP2024540470A5 publication Critical patent/JP2024540470A5/ja
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
    • A61K48/0075—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 delivery route, e.g. oral, subcutaneous
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/17—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • A61K38/177—Receptors; Cell surface antigens; Cell surface determinants
    • 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
    • A61K48/005—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 'active' part of the composition delivered, i.e. the nucleic acid delivered
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P27/00—Drugs for disorders of the senses
    • A61P27/02—Ophthalmic agents
    • 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
    • C07K14/4701—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals not used
    • C07K14/472—Complement proteins, e.g. anaphylatoxin, C3a, C5a
    • 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
    • C07K14/705—Receptors; Cell surface antigens; Cell surface determinants
    • C07K14/70596—Molecules with a "CD"-designation not provided for elsewhere
    • 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
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/0012—Galenical forms characterised by the site of application
    • A61K9/0019—Injectable compositions; Intramuscular, intravenous, arterial, subcutaneous administration; Compositions to be administered through the skin in an invasive manner
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/0012—Galenical forms characterised by the site of application
    • A61K9/0048—Eye, e.g. artificial tears
    • 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)
  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
  • General Health & Medical Sciences (AREA)
  • Medicinal Chemistry (AREA)
  • Organic Chemistry (AREA)
  • Zoology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Molecular Biology (AREA)
  • Biotechnology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Epidemiology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Biochemistry (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Biophysics (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biomedical Technology (AREA)
  • Cell Biology (AREA)
  • Immunology (AREA)
  • Toxicology (AREA)
  • Virology (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Microbiology (AREA)
  • Ophthalmology & Optometry (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Dermatology (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Peptides Or Proteins (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
JP2024529310A 2021-11-19 2022-11-18 可溶性ｃｄ５９を発現する遺伝子療法ベクターにより地図状萎縮を治療する方法 Pending JP2024540470A (ja)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US202163281190P 2021-11-19 2021-11-19
US63/281,190 2021-11-19
PCT/IB2022/061158 WO2023089564A1 (en) 2021-11-19 2022-11-18 Method of treating geographic atrophy with a gene therapy vector expressing soluble cd59

Publications (3)

Publication Number Publication Date
JP2024540470A true JP2024540470A (ja) 2024-10-31
JPWO2023089564A5 JPWO2023089564A5 (https=) 2025-11-27
JP2024540470A5 JP2024540470A5 (https=) 2025-11-27

Family

ID=84365536

Family Applications (1)

Application Number Title Priority Date Filing Date
JP2024529310A Pending JP2024540470A (ja) 2021-11-19 2022-11-18 可溶性ｃｄ５９を発現する遺伝子療法ベクターにより地図状萎縮を治療する方法

Country Status (11)

Country Link
US (1) US20250018063A1 (https=)
EP (1) EP4433496A1 (https=)
JP (1) JP2024540470A (https=)
KR (1) KR20240107156A (https=)
CN (1) CN118369333A (https=)
AU (1) AU2022394125A1 (https=)
CA (1) CA3237907A1 (https=)
IL (1) IL312903A (https=)
JO (1) JOP20240112A1 (https=)
MX (1) MX2024006087A (https=)
WO (1) WO2023089564A1 (https=)

Families Citing this family (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2025074268A1 (en) * 2023-10-02 2025-04-10 Janssen Biotech, Inc. Methods for treating macular degeneration
WO2025083169A1 (en) * 2023-10-17 2025-04-24 Tenpoint Therapeutics Limited Combination of a vegf inhibitor and a complement pathway inhibitor for treating ocular disorders

Family Cites Families (29)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US4405712A (en) 1981-07-01 1983-09-20 The United States Of America As Represented By The Department Of Health And Human Services LTR-Vectors
US4650764A (en) 1983-04-12 1987-03-17 Wisconsin Alumni Research Foundation Helper cell
AU4404785A (en) 1984-05-25 1985-12-31 Dana-Farber Cancer Institute, Inc. Ltr vectors, methods of preparation and use
US4797368A (en) 1985-03-15 1989-01-10 The United States Of America As Represented By The Department Of Health And Human Services Adeno-associated virus as eukaryotic expression vector
US5139941A (en) 1985-10-31 1992-08-18 University Of Florida Research Foundation, Inc. AAV transduction vectors
US4861719A (en) 1986-04-25 1989-08-29 Fred Hutchinson Cancer Research Center DNA constructs for retrovirus packaging cell lines
US4980289A (en) 1987-04-27 1990-12-25 Wisconsin Alumni Research Foundation Promoter deficient retroviral vector
CA1315719C (en) 1988-02-05 1993-04-06 Arthur Bank Retroviral packaging cell lines and processes of using same
US5112767A (en) 1988-03-04 1992-05-12 University Of Southern California Vectors with enhancer domains
US6677311B1 (en) 1988-07-20 2004-01-13 The Salk Institute For Biological Studies Inducible HSV-TK in transformed cell populations
CA1339354C (en) 1988-09-01 1997-08-26 The Whitehead Institute For Biomedical Research Recombinant retroviruses with amphotropic and ecotropic host ranges
WO1990002797A1 (en) 1988-09-15 1990-03-22 North Carolina State University Self-inactivating, replication defective, retroviral vectors and helper cells for producing the same
US5124263A (en) 1989-01-12 1992-06-23 Wisconsin Alumni Research Foundation Recombination resistant retroviral helper cell and products produced thereby
WO1990013641A1 (en) 1989-05-10 1990-11-15 Sloan-Kettering Institute For Cancer Research Stably transformed eucaryotic cells comprising a foreign transcribable dna under the control of a pol iii promoter
EP1285967A3 (en) 1990-09-21 2005-03-02 Chiron Corporation Human retroviral packaging cell line
JP3249516B2 (ja) 1990-10-31 2002-01-21 ソマティクス セラピー コーポレイション 遺伝子治療のためのレトロウイルスのベクター
US5122767A (en) 1991-01-10 1992-06-16 Northern Telecom Limited Saw device tapped delay lines
DE69131908T3 (de) 1991-02-19 2008-04-03 Oxford Biomedica (Uk) Ltd. Viruspartikel mit veraendertem wirtspektrum
AU3434393A (en) 1992-01-17 1993-08-03 Regents Of The University Of Michigan, The Targeted virus
US5627264A (en) 1994-03-03 1997-05-06 Alexion Pharmaceuticals, Inc. Chimeric complement inhibitor proteins
US5998205A (en) 1994-11-28 1999-12-07 Genetic Therapy, Inc. Vectors for tissue-specific replication
US5851826A (en) 1995-07-26 1998-12-22 Children's Medical Center Corporation Helper virus-free herpesvirus vector packaging system
US6689600B1 (en) 1998-11-16 2004-02-10 Introgen Therapeutics, Inc. Formulation of adenovirus for gene therapy
WO2002068627A2 (en) 2001-02-23 2002-09-06 Novartis Ag Vector constucts
US20070196367A1 (en) 2006-02-22 2007-08-23 Valentin Dinu Methods of preventing and treating Alzheimer's disease, age related macular degeneration and other diseases involving extra-cellular debris through the inhibition of the complement system
AU2008295509A1 (en) 2007-08-29 2009-03-12 Tufts University Methods of making and using a cell penetrating peptide for enhanced delivery of nucleic acids, proteins, drugs, and adenovirus to tissues and cells, and compositions and kits
JP5697027B2 (ja) 2008-02-15 2015-04-08 タフツ ユニバーシティー マウス網膜上への細胞膜傷害複合体（ｍａｃ）生成のヒト化モデル、並びに、黄斑変性の処置のための組成物とキットと方法
CA2843684A1 (en) 2010-08-13 2012-02-16 Tufts University Compositions, kits and methods for treatment of macular degeneration using soluble membrane-independent cd59 protein
ES2992014T3 (es) * 2018-02-12 2024-12-05 Tufts College CD59 para inhibir la activación del inflamasoma

Also Published As

Publication number Publication date
CN118369333A (zh) 2024-07-19
KR20240107156A (ko) 2024-07-08
JOP20240112A1 (ar) 2024-05-16
WO2023089564A1 (en) 2023-05-25
MX2024006087A (es) 2024-05-30
AU2022394125A1 (en) 2024-07-04
EP4433496A1 (en) 2024-09-25
IL312903A (en) 2024-07-01
US20250018063A1 (en) 2025-01-16
CA3237907A1 (en) 2023-05-25

Similar Documents

Publication Publication Date Title
US20250243509A1 (en) Methods for treating eye disease
CA2873628C (en) Treatment of amd using aav sflt-1
US20190100582A1 (en) Compositions and methods for reducing ocular neovascularization
JP7275095B2 (ja) 補体関連障害を処置するための組成物、方法およびキット
US20240398898A1 (en) Compositions, kits and methods for treatment of complement-related disorders
CN117531025A (zh) 使用aav2变体和阿柏西普治疗amd
CN113286878A (zh) 补体因子i和补体因子i辅因子、编码它们的载体和治疗用途
JP2024540470A (ja) 可溶性ｃｄ５９を発現する遺伝子療法ベクターにより地図状萎縮を治療する方法
CN119585299A (zh) 编码抗vegf实体和补体负调节剂的核酸及其用于年龄相关性黄斑变性的治疗的用途
CA3106838A1 (en) Gene therapy for macular degeneration
US8877896B2 (en) Compositions, methods and kits for modeling, diagnosing, and treating complement disorders
CN112203677A (zh) 用于抑制炎症小体激活的cd59
WO2025074268A1 (en) Methods for treating macular degeneration
CN119256089A (zh) 用于表达治疗剂的组合物和方法
AU2024322589A1 (en) Compositions and methods for expressing therapeutics
WO2025015317A2 (en) Complement pathway inhibitor fusions and methods of using the same
CA2959376C (en) Compositions, methods and kits for treating complement related disorders

Legal Events

Date Code Title Description
A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20251118

A621 Written request for application examination

Free format text: JAPANESE INTERMEDIATE CODE: A621

Effective date: 20251118