HUE027142T2 - Eljárások és kompozíciók betegségek kezelésére - Google Patents

Eljárások és kompozíciók betegségek kezelésére Download PDF

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Publication number
HUE027142T2
HUE027142T2 HUE07870731A HUE07870731A HUE027142T2 HU E027142 T2 HUE027142 T2 HU E027142T2 HU E07870731 A HUE07870731 A HU E07870731A HU E07870731 A HUE07870731 A HU E07870731A HU E027142 T2 HUE027142 T2 HU E027142T2
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HU
Hungary
Prior art keywords
cells
gene
promoter
cell
lethal
Prior art date
Application number
HUE07870731A
Other languages
English (en)
Inventor
Thomas D Reed
Robert P Beech
Original Assignee
Intrexon Corp
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 Intrexon Corp filed Critical Intrexon Corp
Publication of HUE027142T2 publication Critical patent/HUE027142T2/hu

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Classifications

    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A61K31/711—Natural deoxyribonucleic acids, i.e. containing only 2'-deoxyriboses attached to adenine, guanine, cytosine or thymine and having 3'-5' phosphodiester links
    • 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
    • C12N5/0081—Purging biological preparations of unwanted cells
    • C12N5/0093—Purging against cancer cells
    • 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
    • A61K38/43—Enzymes; Proenzymes; Derivatives thereof
    • A61K38/45—Transferases (2)
    • 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P35/00—Antineoplastic agents
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P35/00—Antineoplastic agents
    • A61P35/02—Antineoplastic agents specific for leukemia
    • 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
    • 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
    • C12N5/0634—Cells from the blood or the immune system
    • C12N5/0647—Haematopoietic stem cells; Uncommitted or multipotent progenitors
    • 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
    • C12N2501/00—Active agents used in cell culture processes, e.g. differentation
    • C12N2501/70—Enzymes
    • C12N2501/72—Transferases (EC 2.)
    • C12N2501/724—Glycosyltransferases (EC 2.4.)
    • 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
    • C12Y—ENZYMES
    • C12Y204/00—Glycosyltransferases (2.4)
    • C12Y204/02—Pentosyltransferases (2.4.2)
    • C12Y204/02036—NAD(+)--diphthamide ADP-ribosyltransferase (2.4.2.36)

Claims (7)

  1. Szabadalmi igénypontok 1. hz m>o eljárás éld, nem megbeíegedeií sejtek nyerésére, she; a sejtek alkalmasak betegség kmW$m íteypm negbefepfe* sejtek megsemmisítése révén, ahol a sejtek egy eljárás áfM vámsak nyerve, amelyek tartalmazzák fs> legalább egy betegség starker gén aktivitásának meghatározását az alanyból származó sej tpopuláe lóban; ti·!} pohnukJe-otsd bevezetését a sejtekbe, amely kódol szelektálható marken és pobpeptkSct. amely iirnm0m letáks a sejt számára,; akik a letáiis polipeprid eyptesszíója kazveáenöi vagy kézvetve a lejpább egy betegség marker gén promoter« által vas vezérelve; Őri; kitesszük a sejteket: szelekciós feltételeknek, bogy sejteket nyerjünk, amelyek tartalmazzák a polimikieobdot; id) kezeljük a sejteket olyan feltételekkel, hogy indakáljnk a lerábs tx>iipepb<U\spress:dóját, amely megöli a sejteket, amelyek exprevszálják a legalább egy betegség market gént; és (e) elkülönítjük a megölt sejteket a maradék élő. nem megbetegedett sejtektől ahol az élő sejtek öem expeaszálják a letalis pokpepödel olya» méxtékbéö, bogy megöpk a nem megbetegedett sej leket,
  2. 2. Az I. igénypont szerint) eljárás, áttol a promoter mükridésiieg van kapcsolva a pedimskieotidhoz, amely kódolja a letális polípephdéri
  3. 3. Az: I, igénypont szerion eljárás, ahol a sejtek ki vannak választva a csoportból, amely áll a Irévetkezökbök heHtatojasletikas őssejtek, máj őasejtek, ernlö össej:íek, baspyál őssejtek, és tmmrmdhs; őssejtek. 4. A 3, igénypont szermi eljárás, ahol a sejtek hemätepotettte őssejtek.
  4. 5. Az 1, igénypont szerinti eprás, ahol a. pb|lríükieotid:; bevezetése tartalmazza a polmakleotid tranziens:írnnszlskeioíát a sejtekbe.
  5. 6. Az I, Igénypont: szerinti efárirs, ahol a pollpokleottd bevezetése tartalmazza a poiimskleotld stabil traaszíékeidíál a sejtekbe.
  6. 7. Az 1. igénypont szerinti; eljárás, also! a polmtikleotid tartalmaz: legalább keltő gén programot. I. Az I, igénypont szerinti eljárás, aboi a betegség marker gén pxnnotere ligáivá van első es második molekuláris inzereló íorgásposs között. 9, .A 7. Igénypont szerinti eljárás, aboi a poünokieotid. amely kódolja a letalis polipeptidet, amely ligáivá van: második és harmadik moleknlázA iztzerelo: tbrgáspohhlrézöő, M„ A. 81. vagy '9: .iféeypos* szerinti eljárás, afe#| az: moieMaris InzereiA lorgásponmk áhttak három vagy négy ritka vagy szokatlan restrikciós helyből iSjytatöiagos elrenslödezésírea, a ritka és szokatlan restrikciós telyek ki vasnak választva a csoportból, amely áll a restrikciós helyekből amelyek megfelelnek a; kővetkezőknek; AsiS K Pác I, Shfl. Fse I, Ase 1. Mlu L SnaB 1, Not I, Sal ), S\va .1,Jftsr il, BSíW 1, Sib l, Sgr AI, Ail III, Fml, Ngo Ml V Ase I, Ftp .1, Pom I, Sda 1, Sgi ï, Srf I és $<*878 1 restrikciós enzimek, I l. A r. Igénypont: szerinti eljárás, aboi pp&amp;téte továbbá tartalmaz legalább egy kromatin tpédosifó domalnt, IX Μ 1 igéHvp>tií sKfö'iRö: eljárás, akoli a gofeukleotki bevezetése tartalmsa&amp;a a jxdiauk'íeötiá· locus-· specifikus iazercicpk
  7. 13. M i. igêsypoïiî szermíi epres, aboi a polmaldcoíid vek-orbas vsa tariakiáácva. M Az l, igésypoïst szeriuti eprás, síiéi a vektor viras v«k&amp;>*.
HUE07870731A 2006-07-26 2007-07-26 Eljárások és kompozíciók betegségek kezelésére HUE027142T2 (hu)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US82038106P 2006-07-26 2006-07-26
US88909507P 2007-02-09 2007-02-09

Publications (1)

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HUE027142T2 true HUE027142T2 (hu) 2016-08-29

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Country Status (17)

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US (3) US20100003226A1 (hu)
EP (1) EP2043662B1 (hu)
JP (1) JP2009544711A (hu)
KR (1) KR20090035011A (hu)
AU (1) AU2007332980A1 (hu)
CA (1) CA2658836C (hu)
DK (1) DK2043662T3 (hu)
ES (1) ES2553332T3 (hu)
HK (1) HK1129596A1 (hu)
HU (1) HUE027142T2 (hu)
IL (1) IL196638A (hu)
MX (1) MX2009000966A (hu)
NZ (1) NZ575075A (hu)
PL (1) PL2043662T3 (hu)
PT (1) PT2043662E (hu)
RU (1) RU2468820C2 (hu)
WO (1) WO2008073154A2 (hu)

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WO2016133907A1 (en) * 2015-02-17 2016-08-25 Cantex Pharmaceuticals, Inc. Adoptive cell transfer methods
EP3258941A4 (en) 2015-02-17 2018-09-26 Cantex Pharmaceuticals, Inc. Treatment of cancers and hematopoietic stem cell disorders privileged by cxcl12-cxcr4 interaction
CA3004742A1 (en) 2015-11-11 2017-05-18 Intrexon Corporation Compositions and methods for expression of multiple biologically active polypeptides from a single vector for treatment of cardiac conditions and other pathologies
WO2018207808A1 (ja) * 2017-05-09 2018-11-15 学校法人 慶應義塾 脳腫瘍治療用細胞製剤

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Also Published As

Publication number Publication date
CA2658836C (en) 2017-11-28
WO2008073154A3 (en) 2008-12-04
US20100003226A1 (en) 2010-01-07
CA2658836A1 (en) 2008-06-19
HK1129596A1 (en) 2009-12-04
NZ575075A (en) 2011-10-28
US20170191027A1 (en) 2017-07-06
US20150132265A1 (en) 2015-05-14
EP2043662B1 (en) 2015-09-09
KR20090035011A (ko) 2009-04-08
JP2009544711A (ja) 2009-12-17
IL196638A (en) 2013-11-28
PL2043662T3 (pl) 2016-03-31
EP2043662A2 (en) 2009-04-08
WO2008073154A2 (en) 2008-06-19
RU2468820C2 (ru) 2012-12-10
RU2009103208A (ru) 2010-09-10
DK2043662T3 (en) 2015-12-14
EP2043662A4 (en) 2010-04-14
IL196638A0 (en) 2009-11-18
AU2007332980A1 (en) 2008-06-19
MX2009000966A (es) 2009-03-05
ES2553332T3 (es) 2015-12-07
PT2043662E (pt) 2015-11-25

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