MX2018007986A - Terapia genica para epidermolisis ampollosa distrofica recesiva con queratinocitos autologos geneticamente corregidos. - Google Patents

Terapia genica para epidermolisis ampollosa distrofica recesiva con queratinocitos autologos geneticamente corregidos.

Info

Publication number
MX2018007986A
MX2018007986A MX2018007986A MX2018007986A MX2018007986A MX 2018007986 A MX2018007986 A MX 2018007986A MX 2018007986 A MX2018007986 A MX 2018007986A MX 2018007986 A MX2018007986 A MX 2018007986A MX 2018007986 A MX2018007986 A MX 2018007986A
Authority
MX
Mexico
Prior art keywords
epidermolysis bullosa
gene therapy
dystrophic epidermolysis
genetically corrected
recessive dystrophic
Prior art date
Application number
MX2018007986A
Other languages
English (en)
Spanish (es)
Inventor
Siprashvili Zurab
T Nguyen Ngon
Peter Marinkovich M
Tang Jean
T Lane Alfred
A Khavari Paul
Original Assignee
Univ Leland Stanford Junior
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 filed Critical Univ Leland Stanford Junior
Publication of MX2018007986A publication Critical patent/MX2018007986A/es

Links

Classifications

    • 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
    • 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
    • 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
    • A61L27/3604—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
    • 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)
MX2018007986A 2016-01-04 2017-01-03 Terapia genica para epidermolisis ampollosa distrofica recesiva con queratinocitos autologos geneticamente corregidos. MX2018007986A (es)

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
MX2018007986A true MX2018007986A (es) 2018-11-09

Family

ID=58010360

Family Applications (1)

Application Number Title Priority Date Filing Date
MX2018007986A MX2018007986A (es) 2016-01-04 2017-01-03 Terapia genica para epidermolisis ampollosa distrofica recesiva con queratinocitos autologos geneticamente corregidos.

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
DK3400287T3 (da) 2021-03-01
JP2023002634A (ja) 2023-01-10
AU2020239779A1 (en) 2020-10-22
CN114699558B (zh) 2023-07-28
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
MX2018007986A (es) Terapia genica para epidermolisis ampollosa distrofica recesiva con queratinocitos autologos geneticamente corregidos.
ZA202310779B (en) Compositions and methods for enhanced gene expression
EP3593809A4 (en) PHARMACEUTICAL COMPOSITION CONTAINING MMR1 POSITIVE MESENCHYMATIC STEM CELLS IN THE PROPHYLACTIC OR THERAPEUTIC TREATMENT OF A FIBROSIS-ASSOCIATED DISEASE, ITS PREPARATION PROCESS, AND PROCESS FOR THE PROPHYLACTIC TREATMENT OF FIBROSIS-ASSOCIATED DISEASE OR A FIBROSIS-ASSOCIATED THERAPEUTIC TREATMENT, AND PROCESS FOR THE PROPHYLACTIC TREATMENT OF FIBROSIS ASSOCIATED OR THERAPEUTIC TREATMENT MMR-POSITIVE MESENCHYMATIC STEM CELLS
AU2018372763A1 (en) Materials and methods for treatment of autosomal dominant Retinitis Pigmentosa
AU2018338608A1 (en) Methods, compositions, and implantable elements comprising active cells
PH12020550450A1 (en) Certain chemical entities, compositions and methods
MX2017004890A (es) Metodos y composiciones para la generación o el mantenimiento de células pluripotentes.
MX2024001378A (es) Oligonucleotidos multi-conjugados definidos.
BR112016029781A2 (pt) composições e métodos para inibir a expressão de gene de alfa-1 antitripsina
MX2020004541A (es) Edicion de genes de celulas primarias.
MX372651B (es) ARN SINTÉTICO PARA SU Uso EN EL TRATAMIENTO DE LA EPIDERMÓLISIS AMPOLLOSA DISTRÓFICA.
JOP20210043A1 (ar) تراكيب وأساليب لتثبيط تعبير جيني للـ lpa
EP3284816A4 (en) Culture medium additive, culture medium composition, and method for culturing cells or tissue using same
EP4484443A3 (en) Materials and methods for treatment of friedreich ataxia and other related disorders
MY197519A (en) Notch pathway signaling inhibitor compounds
MX347112B (es) Composiciones y metodos para regular osmolaridad celular.
WO2019118518A3 (en) Inducible cell receptors for cell-based therapeutics
EP3269402A4 (en) Anti-adhesion material and artificial biological membrane each comprising decellularized tissue
EP3984596A4 (en) POPULATION OF CELLS COMPRISING MESENCHYMATOUS CELLS, PHARMACEUTICAL COMPOSITION COMPRISING THEM AND METHOD FOR PRODUCTION THEREOF
IL246525B (en) Pomegranate derived cell culture and methods for preparing and using the same
MX2020011470A (es) Metodos de terapia genica.
WO2019213128A8 (en) Compositions and therapeutic methods of microrna gene delivery
EP3733838A4 (en) CELL POPULATION WITH ADHESIVE STEM CELLS, METHOD FOR MANUFACTURING SUCH CELL POPULATION AND PHARMACEUTICAL COMPOSITION
EP3861098A4 (en) CELL CULTURE BIOREACTOR
EP3873538A4 (en) ORAL DELIVERY OF THERAPEUTIC MAMMALIAN CELLS