DK3400287T3 - Genterapi for recessiv dystrofisk epidermolysis bullosa under anvendelse af genetisk korrigerede autologe keratinocytter - Google Patents

Genterapi for recessiv dystrofisk epidermolysis bullosa under anvendelse af genetisk korrigerede autologe keratinocytter Download PDF

Info

Publication number
DK3400287T3
DK3400287T3 DK17704327.0T DK17704327T DK3400287T3 DK 3400287 T3 DK3400287 T3 DK 3400287T3 DK 17704327 T DK17704327 T DK 17704327T DK 3400287 T3 DK3400287 T3 DK 3400287T3
Authority
DK
Denmark
Prior art keywords
gene therapy
epidermolysis bullosa
dystrophic epidermolysis
genetically corrected
recessive dystrophic
Prior art date
Application number
DK17704327.0T
Other languages
Danish (da)
English (en)
Inventor
M Peter Marinkovich
Jean Tang
Alfred T Lane
Paul A Khavari
Zurab Siprashvili
Ngon T Nguyen
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
Application granted granted Critical
Publication of DK3400287T3 publication Critical patent/DK3400287T3/da

Links

Classifications

    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61L—METHODS 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
    • A61L27/00—Materials for grafts or prostheses or for coating grafts or prostheses
    • A61L27/36—Materials 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
    • A61L27/3604—Materials 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
    • 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
    • C12N5/00—Undifferentiated human, animal or plant cells, e.g. cell lines; Tissues; Cultivation or maintenance thereof; Culture media therefor
    • C12N5/06—Animal cells or tissues; Human cells or tissues
    • C12N5/0602—Vertebrate cells
    • C12N5/0625—Epidermal cells, skin cells; Cells of the oral mucosa
    • C12N5/0629—Keratinocytes; Whole skin
    • 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
    • C12N5/00—Undifferentiated human, animal or plant cells, e.g. cell lines; Tissues; Cultivation or maintenance thereof; Culture media therefor
    • 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
    • A61K38/39—Connective tissue peptides, e.g. collagen, elastin, laminin, fibronectin, vitronectin, cold insoluble globulin [CIG]
    • 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
    • A61K48/0058—Nucleic acids adapted for tissue specific expression, e.g. having tissue specific promoters as part of a contruct
    • 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
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/0091—Purification or manufacturing processes for gene therapy compositions
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61L—METHODS 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
    • A61L27/00—Materials for grafts or prostheses or for coating grafts or prostheses
    • A61L27/36—Materials 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
    • A61L27/3683—Materials 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
    • A61L27/3695—Materials 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P17/00—Drugs for dermatological disorders
    • 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61L—METHODS 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
    • A61L2430/00—Materials or treatment for tissue regeneration
    • A61L2430/16—Materials or treatment for tissue regeneration for reconstruction of eye parts, e.g. intraocular lens, cornea
    • 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
    • C12N2510/00—Genetically modified cells
    • 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
    • C12N2760/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssRNA viruses negative-sense
    • C12N2760/00011—Details
    • C12N2760/16011—Orthomyxoviridae
    • C12N2760/16041—Use of virus, viral particle or viral elements as a vector
    • C12N2760/16043—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)
  • 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)
DK17704327.0T 2016-01-04 2017-01-03 Genterapi for recessiv dystrofisk epidermolysis bullosa under anvendelse af genetisk korrigerede autologe keratinocytter DK3400287T3 (da)

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
DK3400287T3 true DK3400287T3 (da) 2021-03-01

Family

ID=58010360

Family Applications (1)

Application Number Title Priority Date Filing Date
DK17704327.0T DK3400287T3 (da) 2016-01-04 2017-01-03 Genterapi for recessiv dystrofisk epidermolysis bullosa under anvendelse af genetisk korrigerede autologe keratinocytter

Country Status (17)

Country Link
US (4) US12173314B2 (enExample)
EP (2) EP3842520A1 (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)

Families Citing this family (10)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP3842520A1 (en) * 2016-01-04 2021-06-30 The Board of Trustees of the Leland Stanford Junior University Gene therapy for recessive dystrophic epidermolysis bullosa using genetically corrected autologous keratinocytes
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
SG11202005934WA (en) * 2017-12-29 2020-07-29 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
KR20210116531A (ko) 2019-01-18 2021-09-27 고꾸리쯔 다이가꾸 호우징 오사까 다이가꾸 영양 장애형 표피 수포증 치료약
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

Family Cites Families (12)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
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
MXPA02000152A (es) * 1999-07-02 2002-07-30 Human Genome Sciences Inc Factor 2 de crecimiento de queratinocitos.
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
US8865199B2 (en) * 2008-11-17 2014-10-21 Ingeneron, Inc. Biomatrix composition and methods of biomatrix seeding
GB201202561D0 (en) * 2012-02-15 2012-03-28 Univ Dundee Treatment of skin disorders
JP6334550B2 (ja) * 2012-11-06 2018-05-30 インベッド バイオサイエンシズ，インコーポレイテッド 創傷治癒の方法と組成物
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
EP3842520A1 (en) * 2016-01-04 2021-06-30 The Board of Trustees of the Leland Stanford Junior University Gene therapy for recessive dystrophic epidermolysis bullosa using genetically corrected autologous keratinocytes
US20190192636A1 (en) * 2016-03-18 2019-06-27 Intrexon Corporation Compositions and methods for treatment of type viii collagen deficiencies

Also Published As

Publication number Publication date
IL260038B (en) 2022-07-01
RU2021121824A (ru) 2021-08-18
CN108473952B (zh) 2022-04-08
US20250075178A1 (en) 2025-03-06
PT3400287T (pt) 2021-03-17
EP3842520A1 (en) 2021-06-30
CN114699558A (zh) 2022-07-05
WO2017120147A1 (en) 2017-07-13
JP2019506925A (ja) 2019-03-14
US20190382724A1 (en) 2019-12-19
IL293465A (en) 2022-08-01
US20240067926A1 (en) 2024-02-29
JP7555380B2 (ja) 2024-09-24
RU2018127524A3 (enExample) 2020-01-27
RU2018127524A (ru) 2020-01-27
US12173314B2 (en) 2024-12-24
IL293465B1 (en) 2023-04-01
JP2023002634A (ja) 2023-01-10
AU2020239779A1 (en) 2020-10-22
CN114699558B (zh) 2023-07-28
MX2018007986A (es) 2018-11-09
ZA201803962B (en) 2023-01-25
RU2753248C2 (ru) 2021-08-12
ES2861453T3 (es) 2021-10-06
CN108473952A (zh) 2018-08-31
JP2024167419A (ja) 2024-12-03
AU2017205925A1 (en) 2018-07-12
US12110504B2 (en) 2024-10-08
CA3008676A1 (en) 2017-07-13
IL293465B2 (en) 2023-08-01
US20250257319A1 (en) 2025-08-14
IL260038A (enExample) 2018-07-31
EP3400287A1 (en) 2018-11-14
EP3400287B1 (en) 2020-12-30
NZ743682A (en) 2020-01-31
AU2017205925B2 (en) 2020-10-15
US12385010B2 (en) 2025-08-12
KR20180093967A (ko) 2018-08-22
BR112018013576A2 (pt) 2018-12-11
JP7159048B2 (ja) 2022-10-24

Similar Documents

Publication Publication Date Title
DK3400287T3 (da) Genterapi for recessiv dystrofisk epidermolysis bullosa under anvendelse af genetisk korrigerede autologe keratinocytter
IL284796A (en) Control of gene expression through aptamer-mediated modulation of alternative splicing
FR22C1029I2 (fr) Nouveaux promédicaments hgh polymériques
EP3523847A4 (en) STABILIZATION COATINGS FOR MONOLITHIC BATTERIES
DK3390753T3 (da) Telekommunikationsfordelingselementer
DK3278159T3 (da) Telekommunikationsfordelingselementer
EP3398564A4 (en) LUMINOUS STENT
EP3369227A4 (en) TIME SYNCHRONIZATION FOR SMALL CELLS WITH LIMITED BACKHAUL
DK3191450T3 (da) Pyridonderivater som kinasehæmmere omarrangeret under transfektion (ret)
EP3247317C0 (en) Stent
IT201600117287A1 (it) Metodo di trasfezione transiente per produzione retrovirale.
EP3496660C0 (en) AORTIC IMPLANT
EP3359987A4 (en) METHOD FOR FORMING INTRAOCULAR LENSES
IL259776B (en) Resorbable crosslinked form stable membrane
MA41451A (fr) Constructions anti-tau
EP3632376C0 (en) SELF-CURVING STENT
EP3387076A4 (en) ELASTOMER COATINGS
DK3274465T3 (da) Biokatalytisk fremstilling af l-fucose
BR112017022921A2 (pt) novo método para síntese de metanol
EP3164796A4 (en) Firmware update
IL249254A0 (en) Gene expression system
EP3442544C0 (en) IMPROVED GENE DELIVERY METHODS
EP3438209A4 (en) PROCESS FOR STABILIZING SOLUBLE DYE
ITUB20152051A1 (it) Gruppo sottomarino
EP3991753A4 (en) Transfection method