HUE030932T2 - Fokozott stabilitású, és fokozott katalitikus aktivitású, megváltoztatott, rekombináns, ß-glükocerebrozidáz proteinek - Google Patents

Fokozott stabilitású, és fokozott katalitikus aktivitású, megváltoztatott, rekombináns, ß-glükocerebrozidáz proteinek Download PDF

Info

Publication number
HUE030932T2
HUE030932T2 HUE11839428A HUE11839428A HUE030932T2 HU E030932 T2 HUE030932 T2 HU E030932T2 HU E11839428 A HUE11839428 A HU E11839428A HU E11839428 A HUE11839428 A HU E11839428A HU E030932 T2 HUE030932 T2 HU E030932T2
Authority
HU
Hungary
Prior art keywords
leu
ala
ser
gly
pro
Prior art date
Application number
HUE11839428A
Other languages
English (en)
Inventor
Hung Do
Original Assignee
Amicus Therapeutics Inc
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 Amicus Therapeutics Inc filed Critical Amicus Therapeutics Inc
Publication of HUE030932T2 publication Critical patent/HUE030932T2/hu

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
    • 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
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/52—Genes encoding for enzymes or proenzymes
    • 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)
    • A61K38/47—Hydrolases (3) acting on glycosyl compounds (3.2), e.g. cellulases, lactases
    • 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
    • A61K31/33—Heterocyclic compounds
    • A61K31/395—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins
    • A61K31/435—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having six-membered rings with one nitrogen as the only ring hetero atom
    • A61K31/44—Non condensed pyridines; Hydrogenated derivatives thereof
    • A61K31/445—Non condensed piperidines, e.g. piperocaine
    • 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
    • A61P3/00—Drugs for disorders of the metabolism
    • A61P3/06—Antihyperlipidemics
    • 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
    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14—Hydrolases (3)
    • C12N9/24—Hydrolases (3) acting on glycosyl compounds (3.2)
    • 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)
    • C12N9/24—Hydrolases (3) acting on glycosyl compounds (3.2)
    • C12N9/2402—Hydrolases (3) acting on glycosyl compounds (3.2) hydrolysing O- and S- glycosyl compounds (3.2.1)
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Y—ENZYMES
    • C12Y302/00—Hydrolases acting on glycosyl compounds, i.e. glycosylases (3.2)
    • C12Y302/01—Glycosidases, i.e. enzymes hydrolysing O- and S-glycosyl compounds (3.2.1)
    • C12Y302/01045—Glucosylceramidase (3.2.1.45), i.e. beta-glucocerebrosidase
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides

Landscapes

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

Claims (2)

  1. mmmn βΤΛΒΙΜΤΑβϋ, ÉS FOKOZOTT KATALITIKUS AKTIVITÁSI', MK<n\\)/F02TA' TÓIT, RHKO.WIAÁNS, sVGLÍXKOCKRkiírozhm£ kROTkJVFk Äskhadsl ml i'^i-nyp^iUik i> 'àœ#ÿ»«&amp; íkHMijA £:KéyéiKp2#k kökSS válsSkksR fíloA és UHF: FH 45L.; BU5F; ®45LS:FfMÁ m LMm &amp;4A; és K.32 s V; &amp; IlQ 115 N(3; F s®sïims saiií'öSi^-'S^kveHíááljöz kipást I; &amp;&amp;-i; 'í|é%fiö«t «sæiiisiij. MagválKíssaskk {«komM«te§> teásán •^'•fgläköeefeko.z-Äg· ^rteas.; -»te# a kpvskeKÖ asuiác sókat: F 3 l.í>Á vH 1.31 IF; vagy If 14 31:,; ès: SsstíXp:(SX:lí>vábM ftiMíáe|it,s?FsÍjí s kövHteaók kfelsl vâî&amp;ssîe#: 102 IRpROIA,ásd 031 V». atef â ysxilstteks SEQ1Ö RO: h svssteE &amp;tóöösS¥*s^kvemdáá«>-2 ä*ö»>1ms msitstedk, &amp; Ad L vagy t, iKÄÖ, MpIfliiHi. i«fc$$#isâss< hamsa ^3 - glöklxcöfsröi;kíá± |ïs-ï>îô ïíí, ©RCk 3 sybHMí sOkteskás vagy a *>EQ U.VRÖ; 4Á6 ssstísii aáíiíK!·' SSV-ÍXiA Vi:SÜfk ΡόΠϊΟ Vite. 4 , A.a 1- 3, sgiAgposuvk tesassAyfte bökte? teEbRösösphRtedgg pRtete, âoa| MlspiéyVig tegÿ IbköÁte stssfe O itSξ?1 SuíKsS: vad íipásd^rskösakftáss il^gKäkJVPidkskäddbliöK kd·· fVSí 5, ä 4, igéns'poîd ssvmte siegvteosassosi. AMgsMtteá, teötel E •gÄRörpdmaidÄK:pyoiv|ö:i dbpls i> kömU stedlMa ikssslÜ EAsEdgiés kdäXliHSHyi'k: kdpöd, 4> A: 4, lgÉ5JgXM ste Itei * mag VtevAsJetg syksltekasap îsmsaki p*gÂœsr«toæ$lfe' £î'0h$m,·: shal h. If-köMk;siáisdkás testek scask-ges pH as 4s 3'!'C-oss. f; Ac«· 3*3k ^í8í«lph8'^«dft?if-:'tMgMhá^4h^K,^j#£ó^hs4AhSkh^&amp;iiisi ?Egstevaete4tei4te gmteA ava&amp;l jdifeiösgvg, tegy lagalted káteer kems&amp;àm »5«§»&amp;V«It β#1|# HMialklsje Vas,,.|M s \M': &amp; À 7, igènypagi sssrisks, s$ßgv#M2iä$öt%: yekóihfe|%te Jaftláí? fpy««% testet: akpttdváh táSstelagvs íafateiád stellsgas pEHs és 37te;sssi k?tete, básteö drap lipfglAkMaRp mktesÄ tsas Stet 9, Λ:··: ί ο. sjíéavíXÍi'sKík Mrsmlylks «amsn. îSisii^âpSïStsï-tssd:;. biMjs β:·|ΐΜ|ΐ0ΐ:δ5·|Ιίφ£ΐίΐ3έ pröteJu. ίΐχφ^ΐβΐ^φκίφ; ttegÿ tekeaetJ kafálteiilms âàiixâSàss vas s vsd öp«:s5v mtemfewaös ' esel«.}·« ktepm. lő, ,A. §. jgépsgsspt gsmmís, :asagstel?ù4telk4b röfe>irs£tíís> kassas |:k''gask<aïaSîbisa:id8?: pr&amp;îsiSy sisal s tiJkmoli keisIlHkus aktivitás sssíísgca pH>s da 3? ''Φόΐί sülté ép., bseoreetss keresmé saítsj mkijeslas stetes mm, U V .¾ Φ. s:gé,tíY|K?m .visirask sssegmdisadStete fekmsbssiS.sjs, Issss® preseste: ások a. fekoÂÂfetei^ikWÂi'^îàsf^sîsâl.l fiateksum kőrülteússysk kötete. 12. K.éssfesésvy iiíssly s «xaslsas itempeistek bamssHylke ternîtes, msgá'áifebíáSibf! spsíteüsteáss, hu-mák tk-y:!âka)Sfi)sdf0akdsa psteteist ás :gvőpyas:í:sssa.g: "ika:gkidbskí bsmkmb: isutaiussa, $χ Mbsætettêev, sbitely as M 1. igsayysstsk: bármelyike: ssmstîi, smgválmsíaíokt, teketelteUteSí Pmssteä |-glSkx:grébn>Sídáy pteí#k :# gyágyfealjlsg eil ogteilpilp pu l:lm taslskuss,
  2. 14. Az leli. ifésv^wmk Mrmelyite? assduii, íságvsgimlslpíl, φφφΦίΜφ, hüstelte ^-gMksiesi-sfem-akláz pfotßis MsosteltesBs dre láss betegségi tesMsúbm lislbbü alfesl smitessskí - 15. A 14. ígásypöet esssiipí sreg:uá|léete?f45j isíkeskuaslss, hsmás d--glk^s4'«ivbiSísláá® prcásis á® áll píeglddn>eéibá|ka]ssaáásra, sboi.S iikosgómálls tástetesi betegség öaue&amp;ss'-lmtegség.
HUE11839428A 2010-11-08 2011-11-08 Fokozott stabilitású, és fokozott katalitikus aktivitású, megváltoztatott, rekombináns, ß-glükocerebrozidáz proteinek HUE030932T2 (hu)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US41133110P 2010-11-08 2010-11-08
US41218010P 2010-11-10 2010-11-10

Publications (1)

Publication Number Publication Date
HUE030932T2 true HUE030932T2 (hu) 2017-06-28

Family

ID=46051505

Family Applications (1)

Application Number Title Priority Date Filing Date
HUE11839428A HUE030932T2 (hu) 2010-11-08 2011-11-08 Fokozott stabilitású, és fokozott katalitikus aktivitású, megváltoztatott, rekombináns, ß-glükocerebrozidáz proteinek

Country Status (19)

Country Link
US (4) US8962564B2 (hu)
EP (2) EP3144386B1 (hu)
JP (2) JP6073796B2 (hu)
KR (1) KR101901467B1 (hu)
CN (2) CN103314105B (hu)
BR (1) BR112013011152A2 (hu)
CA (1) CA2817011C (hu)
CY (1) CY1118843T1 (hu)
DK (1) DK2638152T3 (hu)
ES (2) ES2743825T3 (hu)
HR (1) HRP20161560T1 (hu)
HU (1) HUE030932T2 (hu)
LT (1) LT2638152T (hu)
PL (1) PL2638152T3 (hu)
PT (1) PT2638152T (hu)
RS (1) RS55405B1 (hu)
SI (1) SI2638152T1 (hu)
SM (1) SMT201600431B (hu)
WO (1) WO2012064709A2 (hu)

Families Citing this family (8)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP3917558A1 (en) * 2019-02-01 2021-12-08 Oxyrane UK Limited Glucocerebrosidase polypeptides
EP4028048A1 (en) 2019-09-09 2022-07-20 F. Hoffmann-La Roche AG Glucocerebrosidase mutants
MX2023001137A (es) * 2020-07-29 2023-02-22 Freeline Therapeutics Ltd Beta-glucocerebrosidasa mutada con estabilidad mejorada.
JPWO2023145814A1 (hu) * 2022-01-31 2023-08-03
JPWO2023145812A1 (hu) * 2022-01-31 2023-08-03
WO2023145813A1 (ja) * 2022-01-31 2023-08-03 株式会社日本触媒 グルコセレブロシダーゼ活性を有するタンパク質およびその製造方法
WO2023198661A1 (en) 2022-04-12 2023-10-19 F. Hoffmann-La Roche Ag Fusion proteins targeted to the central nervous system
CN118715316A (zh) * 2022-10-08 2024-09-27 凌意(杭州)生物科技有限公司 用于治疗GCase缺乏相关疾病的多核苷酸

Family Cites Families (16)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
ATE138686T1 (de) 1988-12-23 1996-06-15 Genzyme Corp Cho-zellen befähigt enzymatisch aktive rekombinante-glukocerebrosidase zu produzieren
JPH03503721A (ja) * 1988-12-23 1991-08-22 ジェンザイム　コーポレイション 酵素的に活性な組換えグルコセレブロシダーゼ
US6524613B1 (en) 1997-04-30 2003-02-25 Regents Of The University Of Minnesota Hepatocellular chimeraplasty
CA2288209A1 (en) * 1997-04-30 1998-11-05 Regents Of The University Of Minnesota In vivo use of recombinagenic oligonucleobases to correct genetic lesions in hepatocytes
EP2147681A1 (en) * 1997-10-29 2010-01-27 Genzyme Corporation Compositions and methods for treating lysosomal storage disease
IL141403A0 (en) * 1998-08-28 2002-03-10 Univ Duke Deleted adenoviruses and methods of making and administering the same
US20020127219A1 (en) * 1999-12-30 2002-09-12 Okkels Jens Sigurd Lysosomal enzymes and lysosomal enzyme activators
AU2352201A (en) * 1999-12-30 2001-07-16 Maxygen Aps Improved lysosomal enzymes and lysosomal enzyme activators
WO2001077307A2 (en) 2000-04-06 2001-10-18 Exegenics, Inc. Expression system for efficiently producing clinically effective lysosomal enzymes (glucocerebrosidase)
WO2002002597A2 (en) 2000-06-30 2002-01-10 Maxygen Aps Peptide extended glycosylated polypeptides
US7855063B2 (en) * 2003-04-16 2010-12-21 Yeda Research And Development Co. Ltd. Partially deglycosylated glucocerebrosidase polypeptide and crystals thereof
WO2005089047A2 (en) 2004-03-18 2005-09-29 Glycofi, Inc. Glycosylated glucocerebrosidase expression in fungal hosts
WO2008134628A2 (en) * 2007-04-26 2008-11-06 Amicus Therapeutics, Inc. Dosing regimens for the treatment of lysosomal storage diseases using pharmacological chaperones
AU2008254594B2 (en) * 2007-05-22 2013-06-27 Amicus Therapeutics, Inc. New method for preparing isofagomine and its derivatives
US20090148887A1 (en) 2007-11-02 2009-06-11 The Scripps Research Institute Genetically encoded boronate amino acid
JP5513398B2 (ja) 2007-11-02 2014-06-04 ザ スクリプス リサーチ インスティチュート 非天然アミノ酸を含有する蛋白質を使用する指向的進化

Also Published As

Publication number Publication date
KR20140009238A (ko) 2014-01-22
US9254313B2 (en) 2016-02-09
JP6457559B2 (ja) 2019-01-23
JP6073796B2 (ja) 2017-02-01
EP3144386A1 (en) 2017-03-22
EP2638152B1 (en) 2016-08-31
ES2604490T3 (es) 2017-03-07
LT2638152T (lt) 2016-12-12
KR101901467B1 (ko) 2018-11-02
JP2017099390A (ja) 2017-06-08
US9821038B2 (en) 2017-11-21
EP3144386B1 (en) 2019-05-29
CY1118843T1 (el) 2018-01-10
ES2743825T3 (es) 2020-02-20
CA2817011A1 (en) 2012-05-18
PL2638152T3 (pl) 2017-02-28
US20170157220A1 (en) 2017-06-08
JP2014500722A (ja) 2014-01-16
WO2012064709A2 (en) 2012-05-18
US20160184408A1 (en) 2016-06-30
US8962564B2 (en) 2015-02-24
WO2012064709A3 (en) 2012-08-02
CN103314105B (zh) 2015-11-25
US20130344054A1 (en) 2013-12-26
EP2638152A2 (en) 2013-09-18
EP2638152A4 (en) 2014-04-09
HRP20161560T1 (hr) 2016-12-30
DK2638152T3 (en) 2016-12-12
SI2638152T1 (sl) 2016-12-30
BR112013011152A2 (pt) 2016-11-29
US20150216950A1 (en) 2015-08-06
RS55405B1 (sr) 2017-04-28
SMT201600431B (it) 2017-01-10
CN105296447A (zh) 2016-02-03
US9566316B2 (en) 2017-02-14
CA2817011C (en) 2019-04-02
CN103314105A (zh) 2013-09-18
PT2638152T (pt) 2016-10-25

Similar Documents

Publication Publication Date Title
HUE030932T2 (hu) Fokozott stabilitású, és fokozott katalitikus aktivitású, megváltoztatott, rekombináns, ß-glükocerebrozidáz proteinek
Hermans et al. Human lysosomal alpha-glucosidase. Characterization of the catalytic site
JP4342731B2 (ja) プロテアーゼ感受性ｉｉ
Pizzo et al. Ribonucleases with angiogenic and bactericidal activities from the Atlantic salmon
US6673587B1 (en) Histone deacetylase, and uses therefor
KR20160131037A (ko) 그리몬티아·홀리새 유래 재조합 콜라게나아제 및 세포 분리용 효소제
US6548284B1 (en) Membrane-bound metalloprotease and soluble secreted form thereof
US20020119139A1 (en) Cloning and recombinant expression of mammalian group XII secreted phospholipase A2
JP2829397B2 (ja) フィブリン結合活性ポリペプチド
Ahmad et al. Assembly of Multimeric Proteins
NZ275817A (en) Endothelin converting enzymes, preparation and use
Tarullo Expression and Purification of Human Lysosomal β-galactosidase from Pichia Pastoris
JPS61192288A (ja) ヒトエラスタ−ゼ２の生物工学的製造
JP2780160B2 (ja) 機能性ポリペプチド
JP2641273B2 (ja) 変異型ヒトリゾチーム
JPH0454199A (ja) 機能性ポリペプチド
Hoke The cDNA cloning of murine HIP/L29 and the functional analysis of HIP/L29 domains
AU9340301A (en) Therapy for alpha-galactosidase a deficiency
JPH04234988A (ja) ヒトプロラクチン−プロテインａ融合蛋白発現遺伝子
KR20010043415A (ko) 사람 탈아데닐화 뉴클레아제, 이의 제조 방법 및 용도
JPH02307000A (ja) ヒトカルパスタチン様ポリペプチド
JPH0256498A (ja) ヒトカルパスタチン　ポリペプチド
JP2003516756A (ja) ヒトＣｄｃ２５ホスファターゼの取得方法及びヒトＣｄｃ２５ホスファターゼモジュレーターの同定方法