HUE030799T2 - Módszerek és készítmények lizoszomális tárolási betegségek kezelésére - Google Patents

Módszerek és készítmények lizoszomális tárolási betegségek kezelésére Download PDF

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Publication number
HUE030799T2
HUE030799T2 HUE13715059A HUE13715059A HUE030799T2 HU E030799 T2 HUE030799 T2 HU E030799T2 HU E13715059 A HUE13715059 A HU E13715059A HU E13715059 A HUE13715059 A HU E13715059A HU E030799 T2 HUE030799 T2 HU E030799T2
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Hungary
Prior art keywords
cell
gene
protein
transgene
dna
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HUE13715059A
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English (en)
Inventor
Edward J Rebar
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Sangamo Biosciences Inc
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First worldwide family litigation filed litigation Critical https://patents.darts-ip.com/?family=48050927&utm_source=google_patent&utm_medium=platform_link&utm_campaign=public_patent_search&patent=HUE030799(T2) "Global patent litigation dataset” by Darts-ip is licensed under a Creative Commons Attribution 4.0 International License.
Application filed by Sangamo Biosciences Inc filed Critical Sangamo Biosciences Inc
Publication of HUE030799T2 publication Critical patent/HUE030799T2/hu

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    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K35/00—Medicinal preparations containing materials or reaction products thereof with undetermined constitution
    • A61K35/12—Materials from mammals; Compositions comprising non-specified tissues or cells; Compositions comprising non-embryonic stem cells; Genetically modified cells
    • A61K35/28—Bone marrow; Haematopoietic stem cells; Mesenchymal stem cells of any origin, e.g. adipose-derived stem 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
    • A61K38/46—Hydrolases (3)
    • 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
    • A61K38/46—Hydrolases (3)
    • A61K38/465—Hydrolases (3) acting on ester bonds (3.1), e.g. lipases, ribonucleases
    • 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P3/00—Drugs for disorders of the metabolism
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
    • 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
    • C12N15/09—Recombinant DNA-technology
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86—Viral vectors
    • C12N15/864—Parvoviral vectors, e.g. parvovirus, densovirus
    • C12N15/8645—Adeno-associated virus
    • 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
    • C12N15/09—Recombinant DNA-technology
    • C12N15/87—Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
    • C12N15/90—Stable introduction of foreign DNA into chromosome
    • C12N15/902—Stable introduction of foreign DNA into chromosome using homologous recombination
    • C12N15/907—Stable introduction of foreign DNA into chromosome using homologous recombination in mammalian 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
    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14—Hydrolases (3)
    • C12N9/16—Hydrolases (3) acting on ester bonds (3.1)
    • C12N9/22—Ribonucleases RNAses, DNAses
    • 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
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C07K2319/80—Fusion polypeptide containing a DNA binding domain, e.g. Lacl or Tet-repressor
    • C07K2319/81—Fusion polypeptide containing a DNA binding domain, e.g. Lacl or Tet-repressor containing a Zn-finger domain for DNA binding

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • General Health & Medical Sciences (AREA)
  • Organic Chemistry (AREA)
  • Biotechnology (AREA)
  • Medicinal Chemistry (AREA)
  • Zoology (AREA)
  • Molecular Biology (AREA)
  • Wood Science & Technology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Biomedical Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Epidemiology (AREA)
  • Immunology (AREA)
  • Microbiology (AREA)
  • Biochemistry (AREA)
  • Cell Biology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Physics & Mathematics (AREA)
  • Biophysics (AREA)
  • Plant Pathology (AREA)
  • Developmental Biology & Embryology (AREA)
  • Virology (AREA)
  • Hematology (AREA)
  • Mycology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Diabetes (AREA)
  • Obesity (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)

Claims (2)

  1. IMEMPi IS EÁSMTMÉdYdlK LIAC>SZÖMM,tS TÁKÖLASfBMSCSÉSEK Sznhod&amp;hoi Igénypontok L !$ XjMt etjárás égy btyils ESSpí ESSilö sdl gdpéfáte#Á éisély hitódik égy jléoszöníálls fásölMs betegregben '-.?. >>. <. do ál&amp;nj. bzn. ahol .¾ ehntsn. oug.sbz iuyi&amp;he δ d1·'·>Ά<.. .%<. t n fehérjéi kódold b&amp;nvzgéo integnhása s sejt endogén alb·,tone UíKuszába, nem természetesen «. lídoí-ddé nukîenz használatával oh? módon, hogy a sejt megtermeli a?? &amp; fel-etjét, nmelv tnányzlk tt ikoszondih drotási d Az 1. igénypont szennt; in γ$ΐ;ο élj.mo,, ahol 0 lízoszotnáíis tárolási betegség n ksjvethez.ohlm: ál ki csoportból Robiit kivák;s.zíásta: üa«*he.k.l'abry, Hoai«rx Hurler, é&amp; M«mtan»:-Pl«fc. ' V' ,ζρ, Γ ..»'wn s > ΐί η % n<.hbe, tho ο V ,ot' &amp; \Ove k\ό ό d|v >s !' h' kiválasztott fehérjét kódolja glükoeetebrozidáz a galahtoæîdàz. tnnonttb2''Sztüikäz. aiíh-L idiifonkinz es sill sap mt se Hit - losz fed lásateraz
  2. 4, Az Id. igénypontok ontmelylke azerií-ts s n vitro élj árát., ahol « transzáén expresszié-tat egy eaóogéts giéésóisr vezérig % A« 5 -4. igény pmbok bânot'O-lke szeriru» »o vitro eljárás. ahol a UáJtsz.gett egy iuzi^fsMfie, M^Îÿ· olyars aíPmov^tík»»5 .ittak' SJ, unwïheket a ironscge»., salomon 0, traos gént magába záró eô4og^:!ôkttÿZ: kódol· g A,<: 1-5 »góuypontok bármelyike szerinti h* sthx> eljárás, ahol o, uxmsxgén &amp; höveikepkef hiAghgggo t&amp;Ä. fehértói fcwdok ia) egy- membrán feherje olyan e-omcoïlddts doonloje. <.4) oxévkm. hogy expresszsói követhess a htxíhs feltétje a sejt feistzhsess lakahzâlôdlk, d. vagy ib) egy reeeptodsnx vad hginxhmg ntodysté? expressziéi követbe0 a hkshs fehérje htjot a ygr-agÿ gâteru ?, A z l-ô. igenypmslok bàrsttdxike szermii in xdro eh órás, ahoi a telt a következőket Sarhihnadl csoportból kerül ktvdaxzlihta vorösvérsejt. mâjsejb »znmsest és A A ?, Igén) poni tzetinti in vItto eijérút.. ahol az. ö-v^ejt verkcgrb ftvseu vagy Indukált giUtigotóiB ŐsEji g, .\e gk. Igenygosífok bársaciv ike nzeriml in . ara diásS, áros a n jklca/cjuk-ug taskh.U <ΡΝκ ΓΛi h îtiikieÂziTALbMj ès.'v3gy CRISPR/Cát sansltzer. i(i. Az IS. ígénypoui'ik hánnelvike r.-setinis i'ijgaAaa; genenik raj·. iizoszoTtsáiis t:irs>hAi iretagthg kezdtSe s«raii thrfdnö aikahn^sura, ahoi az eijárds suseáivto híglalja a sejt hendà^s a íászartiiA idany tèsæére. U, Sgy vagy több îsakieèz es sgj vagy íöbb toasszgén hzoszosnáhs tarolás! betegség kezelése során történd á!ksí>o;u-asr;k ahoi az eljárás snagábar: foglalja az égj vag> tAbb aokleá? -H az egy vagy iöirb trar«sz#n ä, V V zos ' « « 0 a.a o í>"' iMuu el' *< ' » w *fa sí> lt>d * ^ t alanyban, ahol a fehérjét kódoló transzgéo noeg áiédik &amp; sejt endogén alhutnin ioiatsotába egy aero tertnészstle^n eloh' h 51 î'iîvk V ík ouvíui '! nvs" , , m de í z h/.-vo-ndd í uoheí hetégneg e-ftS hwr.víO lein ; et 12 a H>- i-enygv'in s.vnesisSg j. <-ή -nedri'amz. 0 hkdeu st.t, vvhoiewi.’i .eg ::ndd •-'ssed\ed pn^ttr/or sejt, és tíz. eljárás magéban foglalja az. őssejt vagy greSin-zer sejt beadás előtti expanzióját H diiTerencláí-isát Di Az ID> ^»É*:sôî:l glfiDD à IDD, s$e?j&amp;i hás^álsaé^.síiJt'-'Vl^··^ U;. igggvpjfg sMäM ^Âà^ÂV^y Dlÿ ÖÄÄ?··# ggÿ vsg;g fögl>gfDDgégs g&amp;öig ih^sg-p# à H:g|?y§ «gy ylflgs y&amp;ktig,. Dlgt |><Mdíű·; ;v. ΛΛΥ vD:;o? sDPíífe
HUE13715059A 2012-07-11 2013-03-15 Módszerek és készítmények lizoszomális tárolási betegségek kezelésére HUE030799T2 (hu)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201261670463P 2012-07-11 2012-07-11
US201261704072P 2012-09-21 2012-09-21

Publications (1)

Publication Number Publication Date
HUE030799T2 true HUE030799T2 (hu) 2017-06-28

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ID=48050927

Family Applications (3)

Application Number Title Priority Date Filing Date
HUE18198271A HUE051612T2 (hu) 2012-07-11 2013-03-15 Eljárások és készítmények lizoszomális tárolási betegségek kezelésére
HUE16198961A HUE041553T2 (hu) 2012-07-11 2013-03-15 Lizoszomális tárolási betegségek (LSD) kezelése és génterápia
HUE13715059A HUE030799T2 (hu) 2012-07-11 2013-03-15 Módszerek és készítmények lizoszomális tárolási betegségek kezelésére

Family Applications Before (2)

Application Number Title Priority Date Filing Date
HUE18198271A HUE051612T2 (hu) 2012-07-11 2013-03-15 Eljárások és készítmények lizoszomális tárolási betegségek kezelésére
HUE16198961A HUE041553T2 (hu) 2012-07-11 2013-03-15 Lizoszomális tárolási betegségek (LSD) kezelése és génterápia

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US (6) US9877988B2 (hu)
EP (4) EP3444342B1 (hu)
JP (5) JP6225182B2 (hu)
AU (1) AU2013289206B2 (hu)
CA (1) CA2875618C (hu)
DK (3) DK3196301T3 (hu)
ES (3) ES2613691T3 (hu)
HK (1) HK1209788A1 (hu)
HU (3) HUE051612T2 (hu)
WO (1) WO2014011237A1 (hu)

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