BR112018013576A2 - terapia gênica para epidermólise bolhosa distrófica recessiva usando queratinócitos autólogos geneti-camente corrigidos - Google Patents

terapia gênica para epidermólise bolhosa distrófica recessiva usando queratinócitos autólogos geneti-camente corrigidos

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Publication number
BR112018013576A2
BR112018013576A2 BR112018013576A BR112018013576A BR112018013576A2 BR 112018013576 A2 BR112018013576 A2 BR 112018013576A2 BR 112018013576 A BR112018013576 A BR 112018013576A BR 112018013576 A BR112018013576 A BR 112018013576A BR 112018013576 A2 BR112018013576 A2 BR 112018013576A2
Authority
BR
Brazil
Prior art keywords
gene therapy
bullous epidermolysis
genetically corrected
recessive dystrophic
autologous keratinocytes
Prior art date
Application number
BR112018013576A
Other languages
English (en)
Portuguese (pt)
Inventor
T Lane Alfred
Tang Jean
Peter Marinkovich M
T Nguyen Ngon
A Khavari Paul
Siprashvili Zurab
Original Assignee
Univ Leland Stanford Junior
U S Government As Represented By The Dept 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 Univ Leland Stanford Junior, U S Government As Represented By The Dept Of Veterans Affairs filed Critical Univ Leland Stanford Junior
Publication of BR112018013576A2 publication Critical patent/BR112018013576A2/pt

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    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N5/00Undifferentiated human, animal or plant cells, e.g. cell lines; Tissues; Cultivation or maintenance thereof; Culture media therefor
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00Medicinal preparations containing peptides
    • A61K38/16Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/17Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • A61K38/39Connective tissue peptides, e.g. collagen, elastin, laminin, fibronectin, vitronectin, cold insoluble globulin [CIG]
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/005Medicinal 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
    • A61K48/0058Nucleic acids adapted for tissue specific expression, e.g. having tissue specific promoters as part of a contruct
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/0075Medicinal 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
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/0091Purification or manufacturing processes for gene therapy compositions
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61LMETHODS OR APPARATUS FOR STERILISING MATERIALS OR OBJECTS IN GENERAL; DISINFECTION, STERILISATION OR DEODORISATION OF AIR; CHEMICAL ASPECTS OF BANDAGES, DRESSINGS, ABSORBENT PADS OR SURGICAL ARTICLES; MATERIALS FOR BANDAGES, DRESSINGS, ABSORBENT PADS OR SURGICAL ARTICLES
    • A61L27/00Materials for grafts or prostheses or for coating grafts or prostheses
    • A61L27/36Materials for grafts or prostheses or for coating grafts or prostheses containing ingredients of undetermined constitution or reaction products thereof, e.g. transplant tissue, natural bone, extracellular matrix
    • A61L27/3604Materials for grafts or prostheses or for coating grafts or prostheses containing ingredients of undetermined constitution or reaction products thereof, e.g. transplant tissue, natural bone, extracellular matrix characterised by the human or animal origin of the biological material, e.g. hair, fascia, fish scales, silk, shellac, pericardium, pleura, renal tissue, amniotic membrane, parenchymal tissue, fetal tissue, muscle tissue, fat tissue, enamel
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61LMETHODS OR APPARATUS FOR STERILISING MATERIALS OR OBJECTS IN GENERAL; DISINFECTION, STERILISATION OR DEODORISATION OF AIR; CHEMICAL ASPECTS OF BANDAGES, DRESSINGS, ABSORBENT PADS OR SURGICAL ARTICLES; MATERIALS FOR BANDAGES, DRESSINGS, ABSORBENT PADS OR SURGICAL ARTICLES
    • A61L27/00Materials for grafts or prostheses or for coating grafts or prostheses
    • A61L27/36Materials for grafts or prostheses or for coating grafts or prostheses containing ingredients of undetermined constitution or reaction products thereof, e.g. transplant tissue, natural bone, extracellular matrix
    • A61L27/3683Materials for grafts or prostheses or for coating grafts or prostheses containing ingredients of undetermined constitution or reaction products thereof, e.g. transplant tissue, natural bone, extracellular matrix subjected to a specific treatment prior to implantation, e.g. decellularising, demineralising, grinding, cellular disruption/non-collagenous protein removal, anti-calcification, crosslinking, supercritical fluid extraction, enzyme treatment
    • A61L27/3695Materials for grafts or prostheses or for coating grafts or prostheses containing ingredients of undetermined constitution or reaction products thereof, e.g. transplant tissue, natural bone, extracellular matrix subjected to a specific treatment prior to implantation, e.g. decellularising, demineralising, grinding, cellular disruption/non-collagenous protein removal, anti-calcification, crosslinking, supercritical fluid extraction, enzyme treatment characterised by the function or physical properties of the final product, where no specific conditions are defined to achieve this
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P17/00Drugs for dermatological disorders
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P27/00Drugs for disorders of the senses
    • A61P27/02Ophthalmic agents
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    • C12N5/00Undifferentiated human, animal or plant cells, e.g. cell lines; Tissues; Cultivation or maintenance thereof; Culture media therefor
    • C12N5/06Animal cells or tissues; Human cells or tissues
    • C12N5/0602Vertebrate cells
    • C12N5/0625Epidermal cells, skin cells; Cells of the oral mucosa
    • C12N5/0629Keratinocytes; Whole skin
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61LMETHODS OR APPARATUS FOR STERILISING MATERIALS OR OBJECTS IN GENERAL; DISINFECTION, STERILISATION OR DEODORISATION OF AIR; CHEMICAL ASPECTS OF BANDAGES, DRESSINGS, ABSORBENT PADS OR SURGICAL ARTICLES; MATERIALS FOR BANDAGES, DRESSINGS, ABSORBENT PADS OR SURGICAL ARTICLES
    • A61L2430/00Materials or treatment for tissue regeneration
    • A61L2430/16Materials or treatment for tissue regeneration for reconstruction of eye parts, e.g. intraocular lens, cornea
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    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2510/00Genetically modified cells
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    • C12N2760/00MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssRNA viruses negative-sense
    • C12N2760/00011Details
    • C12N2760/16011Orthomyxoviridae
    • C12N2760/16041Use of virus, viral particle or viral elements as a vector
    • C12N2760/16043Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • General Health & Medical Sciences (AREA)
  • Biomedical Technology (AREA)
  • Genetics & Genomics (AREA)
  • Medicinal Chemistry (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Biotechnology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Epidemiology (AREA)
  • Organic Chemistry (AREA)
  • Molecular Biology (AREA)
  • Zoology (AREA)
  • Dermatology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Wood Science & Technology (AREA)
  • Biochemistry (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Microbiology (AREA)
  • General Engineering & Computer Science (AREA)
  • Cell Biology (AREA)
  • Botany (AREA)
  • Oral & Maxillofacial Surgery (AREA)
  • Transplantation (AREA)
  • Manufacturing & Machinery (AREA)
  • Urology & Nephrology (AREA)
  • Ophthalmology & Optometry (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Immunology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Materials For Medical Uses (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
BR112018013576A 2016-01-04 2017-01-03 terapia gênica para epidermólise bolhosa distrófica recessiva usando queratinócitos autólogos geneti-camente corrigidos BR112018013576A2 (pt)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201662274700P 2016-01-04 2016-01-04
US201662414533P 2016-10-28 2016-10-28
PCT/US2017/012061 WO2017120147A1 (en) 2016-01-04 2017-01-03 Gene therapy for recessive dystrophic epidermolysis bullosa using genetically corrected autologous keratinocytes

Publications (1)

Publication Number Publication Date
BR112018013576A2 true BR112018013576A2 (pt) 2018-12-11

Family

ID=58010360

Family Applications (1)

Application Number Title Priority Date Filing Date
BR112018013576A BR112018013576A2 (pt) 2016-01-04 2017-01-03 terapia gênica para epidermólise bolhosa distrófica recessiva usando queratinócitos autólogos geneti-camente corrigidos

Country Status (17)

Country Link
US (4) US12173314B2 (enExample)
EP (2) EP3400287B1 (enExample)
JP (3) JP7159048B2 (enExample)
KR (1) KR20180093967A (enExample)
CN (2) CN114699558B (enExample)
AU (2) AU2017205925B2 (enExample)
BR (1) BR112018013576A2 (enExample)
CA (1) CA3008676A1 (enExample)
DK (1) DK3400287T3 (enExample)
ES (1) ES2861453T3 (enExample)
IL (2) IL260038B (enExample)
MX (1) MX2018007986A (enExample)
NZ (1) NZ743682A (enExample)
PT (1) PT3400287T (enExample)
RU (2) RU2021121824A (enExample)
WO (1) WO2017120147A1 (enExample)
ZA (1) ZA201803962B (enExample)

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* Cited by examiner, † Cited by third party
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DK3400287T3 (da) * 2016-01-04 2021-03-01 Univ Leland Stanford Junior Genterapi for recessiv dystrofisk epidermolysis bullosa under anvendelse af genetisk korrigerede autologe keratinocytter
IT201700094210A1 (it) * 2017-08-17 2019-02-17 Holostem Terapie Avanzate S R L Metodo per produrre lembi cellulari
IT201700104587A1 (it) * 2017-09-19 2019-03-19 Holostem Terapie Avanzate S R L Usi terapeutici di lembi di cellule geneticamente modificate
EP3733210A4 (en) * 2017-12-29 2021-10-06 Genemedicine Co., Ltd. CELL SHEET FOR GENE DELIVERY
RU2730661C2 (ru) * 2018-12-27 2020-08-24 Селл энд Джин Терапи Лтд Генотерапевтический ДНК-вектор на основе генотерапевтического ДНК-вектора VTvaf17, несущий целевой ген, выбранный из группы генов COL1A1, COL1A2, P4HA1, P4HA2, COL7A1, CLCA2, ELN, PLOD1 для повышения уровня экспрессии этих целевых генов, способ его получения и применения, штамм Escherichia coli SCS110-AF/VTvaf17-COL1A1, или Escherichia coli SCS110-AF/VTvaf17-COL1A2, или Escherichia coli SCS110-AF/VTvaf17-P4HA1, или Escherichia coli SCS110-AF/VTvaf17-P4HA2, или Escherichia coli SCS110-AF/VTvaf17-COL7A1, или Escherichia coli SCS110-AF/VTvaf17-CLCA2, или Escherichia coli SCS110-AF/VTvaf17-ELN, или Escherichia coli SCS110-AF/VTvaf17- PLOD1, несущий генотерапевтический ДНК-вектор, способ его получения, способ производства в промышленных масштабах генотерапевтического ДНК-вектора
WO2020149395A1 (ja) 2019-01-18 2020-07-23 国立大学法人大阪大学 栄養障害型表皮水疱症治療薬
CN116529361B (zh) * 2019-10-18 2024-01-26 北赛泓升(北京)生物科技有限公司 使用多顺反子sox2、klf4和任选的c-myc产生诱导多能干细胞
KR20230043906A (ko) * 2020-07-22 2023-03-31 고꾸리쯔 다이가꾸 호우징 오사까 다이가꾸 영양 장애형 표피 수포증의 치료약
MX2024004434A (es) 2021-10-12 2024-07-09 Phoenix Tissue Repair Inc Terapia de reemplazo de proteínas con colágeno 7.
JP2025094280A (ja) * 2022-01-21 2025-06-25 国立大学法人大阪大学 細胞シート

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US5830725A (en) * 1995-04-28 1998-11-03 The Board Of Trustees For The Leland Stanford Junior University Rapid, stable high-titre production of recombing retrovirus
US20020090725A1 (en) 2000-11-17 2002-07-11 Simpson David G. Electroprocessed collagen
NZ516897A (en) * 1999-07-02 2004-01-30 Human Genome Sciences Inc Polynucleotides and associated polypeptides for KGF-2 (also known as Fibroblast Growth Factor 12) to promote and accelerate internal and external wound healing
FR2857671B1 (fr) * 2003-07-18 2007-10-05 Centre Nat Rech Scient Nouveau procede de culture de keratinocytes et ses applications
CN1916166B (zh) * 2006-09-08 2010-08-11 北京赛尔泰和生物医药科技有限公司 自体角膜上皮的制备方法
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GB201202561D0 (en) * 2012-02-15 2012-03-28 Univ Dundee Treatment of skin disorders
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US8697359B1 (en) 2012-12-12 2014-04-15 The Broad Institute, Inc. CRISPR-Cas systems and methods for altering expression of gene products
CN105087565A (zh) * 2015-08-21 2015-11-25 内蒙古自治区人民医院 一种营养不良型大疱性表皮松解症核苷酸及其应用
DK3400287T3 (da) * 2016-01-04 2021-03-01 Univ Leland Stanford Junior Genterapi for recessiv dystrofisk epidermolysis bullosa under anvendelse af genetisk korrigerede autologe keratinocytter
CA3017757A1 (en) * 2016-03-18 2017-09-21 Intrexon Corporation Compositions and methods for treatment of type vii collagen deficiencies

Also Published As

Publication number Publication date
US12110504B2 (en) 2024-10-08
ZA201803962B (en) 2023-01-25
ES2861453T3 (es) 2021-10-06
US12385010B2 (en) 2025-08-12
CN114699558A (zh) 2022-07-05
IL293465B1 (en) 2023-04-01
AU2020239779A1 (en) 2020-10-22
US20240067926A1 (en) 2024-02-29
US12173314B2 (en) 2024-12-24
IL260038A (enExample) 2018-07-31
DK3400287T3 (da) 2021-03-01
WO2017120147A1 (en) 2017-07-13
JP2024167419A (ja) 2024-12-03
EP3400287B1 (en) 2020-12-30
JP2019506925A (ja) 2019-03-14
KR20180093967A (ko) 2018-08-22
NZ743682A (en) 2020-01-31
RU2753248C2 (ru) 2021-08-12
JP7159048B2 (ja) 2022-10-24
RU2018127524A (ru) 2020-01-27
CA3008676A1 (en) 2017-07-13
CN108473952B (zh) 2022-04-08
RU2018127524A3 (enExample) 2020-01-27
AU2017205925A1 (en) 2018-07-12
US20190382724A1 (en) 2019-12-19
JP7555380B2 (ja) 2024-09-24
CN108473952A (zh) 2018-08-31
EP3842520A1 (en) 2021-06-30
US20250075178A1 (en) 2025-03-06
IL293465B2 (en) 2023-08-01
AU2017205925B2 (en) 2020-10-15
MX2018007986A (es) 2018-11-09
IL260038B (en) 2022-07-01
EP3400287A1 (en) 2018-11-14
PT3400287T (pt) 2021-03-17
RU2021121824A (ru) 2021-08-18
CN114699558B (zh) 2023-07-28
US20250257319A1 (en) 2025-08-14
IL293465A (en) 2022-08-01
JP2023002634A (ja) 2023-01-10

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