ZA201803962B - Gene therapy for recessive dystrophic epidermolysis bullosa using genetically corrected autologous keratinocytes - Google Patents

Gene therapy for recessive dystrophic epidermolysis bullosa using genetically corrected autologous keratinocytes

Info

Publication number
ZA201803962B
ZA201803962B ZA2018/03962A ZA201803962A ZA201803962B ZA 201803962 B ZA201803962 B ZA 201803962B ZA 2018/03962 A ZA2018/03962 A ZA 2018/03962A ZA 201803962 A ZA201803962 A ZA 201803962A ZA 201803962 B ZA201803962 B ZA 201803962B
Authority
ZA
South Africa
Prior art keywords
gene therapy
epidermolysis bullosa
dystrophic epidermolysis
genetically corrected
recessive dystrophic
Prior art date
Application number
ZA2018/03962A
Other languages
English (en)
Inventor
Siprashvili Zurab
T Nguyen Ngon
Peter Marinkovich M
Tang Jean
T Lane Alfred
A Khavari Paul
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 ZA201803962B publication Critical patent/ZA201803962B/en

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)
ZA2018/03962A 2016-01-04 2018-06-13 Gene therapy for recessive dystrophic epidermolysis bullosa using genetically corrected autologous keratinocytes ZA201803962B (en)

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
ZA201803962B true ZA201803962B (en) 2023-01-25

Family

ID=58010360

Family Applications (1)

Application Number Title Priority Date Filing Date
ZA2018/03962A ZA201803962B (en) 2016-01-04 2018-06-13 Gene therapy for recessive dystrophic epidermolysis bullosa using genetically corrected autologous keratinocytes

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)

Families Citing this family (10)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
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

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
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
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
EP3995158A1 (en) * 2012-11-06 2022-05-11 Imbed Biosciences, Inc. Methods and compositions for wound healing
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
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
BR112018013576A2 (pt) 2018-12-11
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

Similar Documents

Publication Publication Date Title
IL293465A (en) Gene therapy for dystrophic blistering of the skin using genetically modified autologous keratinocytes
IL287033A (en) Regulation of nuclease-mediated gene expression
IL260796B1 (en) Factor VIII genes are effective
ZA201705331B (en) Oligonucleotide therapy for leber congenital amaurosis
ZA201604874B (en) Rna-guided gene drives
LT3155049T (lt) Kietėjančios dviejų dalių dangos, skirtos laidininkams
SG11202002130WA (en) Artificial genome manipulation for gene expression regulation
IL257831B2 (en) Diplosporic garden
EP3643316A4 (en) TREATMENT PRODUCTS FOR EPIDERMOLYSIS BULLOSA
EP3164796A4 (en) Firmware update
ZA201804397B (en) Biosynthetic gene cluster of carrimycin
IL249254B (en) Gene expression system
EP3284445A4 (en) Hybrid stent
IL280640A (en) Nanoparticles for transfection
EP3286237A4 (en) Two-part polyurethane erosion resistant coatings
EP3295959A4 (en) Cmlck gene introduction
EP3271462A4 (en) System for stable gene expression
GB201508425D0 (en) Artificial chromosome for retroviral production
GB201608944D0 (en) Gene Tharapy
GB201717695D0 (en) Methods for conditional gene knock-out
GB201620091D0 (en) Methods for conditional gene knock-out
HK1259782A1 (en) Cmv vectors comprising microrna recognition elements
AU2016901739A0 (en) Gene expression system
EP3323892A4 (en) Antisense oligonucleotide inhibition of 2GPI expression
GB201507125D0 (en) Gene delivery