JP2020508289A - 完全ヒトグリコシル化ヒトα−Ｌ−イズロニダーゼ（ＩＤＵＡ）を用いたムコ多糖症Ｉ型の治療 - Google Patents

完全ヒトグリコシル化ヒトα−Ｌ−イズロニダーゼ（ＩＤＵＡ）を用いたムコ多糖症Ｉ型の治療 Download PDF

Info

Publication number
JP2020508289A
JP2020508289A JP2019541264A JP2019541264A JP2020508289A JP 2020508289 A JP2020508289 A JP 2020508289A JP 2019541264 A JP2019541264 A JP 2019541264A JP 2019541264 A JP2019541264 A JP 2019541264A JP 2020508289 A JP2020508289 A JP 2020508289A
Authority
JP
Japan
Prior art keywords
human
idua
expression vector
mps
subject
Prior art date
Legal status (The legal status 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 status listed.)
Pending
Application number
JP2019541264A
Other languages
English (en)
Japanese (ja)
Other versions
JP2020508289A5 (https=
Inventor
ヨー ステファン
ヨー ステファン
ロバート ラインハルト リッキー
ロバート ラインハルト リッキー
マシュー シンプソン クラン
マシュー シンプソン クラン
ウー ズフチュン
ウー ズフチュン
Original Assignee
レジェンクスバイオ インコーポレーテッド
レジェンクスバイオ インコーポレーテッド
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 レジェンクスバイオ インコーポレーテッド, レジェンクスバイオ インコーポレーテッド filed Critical レジェンクスバイオ インコーポレーテッド
Publication of JP2020508289A publication Critical patent/JP2020508289A/ja
Publication of JP2020508289A5 publication Critical patent/JP2020508289A5/ja
Priority to JP2024079088A priority Critical patent/JP2024112876A/ja
Pending legal-status Critical Current

Links

Images

Classifications

    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • 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
    • A61K31/335—Heterocyclic compounds having oxygen as the only ring hetero atom, e.g. fungichromin
    • A61K31/365—Lactones
    • 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
    • A61K31/4353—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 ortho- or peri-condensed with heterocyclic ring systems
    • A61K31/436—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 ortho- or peri-condensed with heterocyclic ring systems the heterocyclic ring system containing a six-membered ring having oxygen as a ring hetero atom, e.g. rapamycin
    • 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
    • A61K31/56—Compounds containing cyclopenta[a]hydrophenanthrene ring systems; Derivatives thereof, e.g. steroids
    • A61K31/57—Compounds containing cyclopenta[a]hydrophenanthrene ring systems; Derivatives thereof, e.g. steroids substituted in position 17 beta by a chain of two carbon atoms, e.g. pregnane or progesterone
    • A61K31/573—Compounds containing cyclopenta[a]hydrophenanthrene ring systems; Derivatives thereof, e.g. steroids substituted in position 17 beta by a chain of two carbon atoms, e.g. pregnane or progesterone substituted in position 21, e.g. cortisone, dexamethasone, prednisone or aldosterone
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K45/00—Medicinal preparations containing active ingredients not provided for in groups A61K31/00 - A61K41/00
    • A61K45/06—Mixtures of active ingredients without chemical characterisation, e.g. antiphlogistics and cardiaca
    • 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
    • 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
    • A61K48/0083—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 administration regime
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • A61P25/28—Drugs for disorders of the nervous system for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia
    • 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K2300/00—Mixtures or combinations of active ingredients, wherein at least one active ingredient is fully defined in groups A61K31/00 - A61K41/00
    • 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
    • C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141—Use of virus, viral particle or viral elements as a vector
    • C12N2750/14143—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
    • 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)
    • C12Y302/01076—L-Iduronidase (3.2.1.76)

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • General Health & Medical Sciences (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Medicinal Chemistry (AREA)
  • Engineering & Computer Science (AREA)
  • Epidemiology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Organic Chemistry (AREA)
  • Genetics & Genomics (AREA)
  • Neurology (AREA)
  • Biomedical Technology (AREA)
  • Neurosurgery (AREA)
  • Molecular Biology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Biotechnology (AREA)
  • Immunology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Hospice & Palliative Care (AREA)
  • Psychiatry (AREA)
  • Biochemistry (AREA)
  • General Engineering & Computer Science (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
JP2019541264A 2017-01-31 2018-01-30 完全ヒトグリコシル化ヒトα−Ｌ−イズロニダーゼ（ＩＤＵＡ）を用いたムコ多糖症Ｉ型の治療 Pending JP2020508289A (ja)

Priority Applications (1)

Application Number Priority Date Filing Date Title
JP2024079088A JP2024112876A (ja) 2017-01-31 2024-05-15 完全ヒトグリコシル化ヒトα－Ｌ－イズロニダーゼ（ＩＤＵＡ）を用いたムコ多糖症Ｉ型の治療

Applications Claiming Priority (11)

Application Number Priority Date Filing Date Title
US201762452769P 2017-01-31 2017-01-31
US62/452,769 2017-01-31
US201762485655P 2017-04-14 2017-04-14
US62/485,655 2017-04-14
US201762529366P 2017-07-06 2017-07-06
US62/529,366 2017-07-06
US201762579690P 2017-10-31 2017-10-31
US62/579,690 2017-10-31
US201862616234P 2018-01-11 2018-01-11
US62/616,234 2018-01-11
PCT/US2018/015910 WO2018144441A1 (en) 2017-01-31 2018-01-30 Treatment of mucopolysaccharidosis i with fully-human glycosylated human alpha-l-iduronidase (idua)

Related Child Applications (1)

Application Number Title Priority Date Filing Date
JP2024079088A Division JP2024112876A (ja) 2017-01-31 2024-05-15 完全ヒトグリコシル化ヒトα－Ｌ－イズロニダーゼ（ＩＤＵＡ）を用いたムコ多糖症Ｉ型の治療

Publications (2)

Publication Number Publication Date
JP2020508289A true JP2020508289A (ja) 2020-03-19
JP2020508289A5 JP2020508289A5 (https=) 2021-03-18

Family

ID=63041038

Family Applications (2)

Application Number Title Priority Date Filing Date
JP2019541264A Pending JP2020508289A (ja) 2017-01-31 2018-01-30 完全ヒトグリコシル化ヒトα−Ｌ−イズロニダーゼ（ＩＤＵＡ）を用いたムコ多糖症Ｉ型の治療
JP2024079088A Pending JP2024112876A (ja) 2017-01-31 2024-05-15 完全ヒトグリコシル化ヒトα－Ｌ－イズロニダーゼ（ＩＤＵＡ）を用いたムコ多糖症Ｉ型の治療

Family Applications After (1)

Application Number Title Priority Date Filing Date
JP2024079088A Pending JP2024112876A (ja) 2017-01-31 2024-05-15 完全ヒトグリコシル化ヒトα－Ｌ－イズロニダーゼ（ＩＤＵＡ）を用いたムコ多糖症Ｉ型の治療

Country Status (12)

Country Link
US (2) US20190358303A1 (https=)
EP (1) EP3576768A4 (https=)
JP (2) JP2020508289A (https=)
KR (2) KR20250022877A (https=)
AU (1) AU2018216807A1 (https=)
BR (1) BR112019015482A2 (https=)
CA (1) CA3049915A1 (https=)
IL (1) IL268076B2 (https=)
MA (1) MA47436A (https=)
SG (1) SG11201906452PA (https=)
UY (1) UY37587A (https=)
WO (1) WO2018144441A1 (https=)

Cited By (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2023552443A (ja) * 2020-12-07 2023-12-15 スペースクラフト セブン リミテッド ライアビリティ カンパニー ダノン病の治療

Families Citing this family (6)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CA3068328A1 (en) 2017-07-06 2019-01-10 The Trustees Of The University Of Pennsylvania Aav9-mediated gene therapy for treating mucopolysaccharidosis type i
KR102883778B1 (ko) 2017-09-22 2025-11-12 더 트러스티스 오브 더 유니버시티 오브 펜실베니아 Ii형 점액다당류증의 치료를 위한 유전자 요법
IL280198B2 (en) * 2018-07-18 2026-01-01 Regenxbio Inc Treatment of mucopolysaccharidosis I with fully glycosylated human iduronidase-alpha-L (idus)
KR20220131522A (ko) * 2020-01-22 2022-09-28 리젠엑스바이오 인크. 완전-인간 글리코실화된 인간 알파-l-이두로니다아제 (idua)를 사용한 점액다당류증 i의 치료
WO2022165313A1 (en) 2021-02-01 2022-08-04 Regenxbio Inc. Gene therapy for neuronal ceroid lipofuscinoses
EP4423264A4 (en) * 2021-10-27 2025-12-17 Univ North Carolina Chapel Hill AAV-IDUA VECTOR FOR TREATING MPS I

Citations (7)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO1999011665A1 (en) * 1997-09-01 1999-03-11 Kirin Brewery Company, Limited Human thrombopoietin produced by established human cell lines, process for producing the same, and medicinal compositions containing the same
JP2002514429A (ja) * 1998-05-13 2002-05-21 ハーバー−ユーシーエルエー 組換えα−Ｌ−イズロニダーゼ、その生成及び精製方法及びその欠損により引き起こされる疾病の治療方法
JP2004504016A (ja) * 2000-06-30 2004-02-12 マキシゲン・エイピーエス ペプチド拡張されたグリコシル化ポリペプチド
JP2007528862A (ja) * 2003-07-16 2007-10-18 アールブイエックス セラピューティクス， インコーポレイテッド ＴＧＦ−βの効果を下方制御するための化合物および方法
JP2008506389A (ja) * 2004-07-13 2008-03-06 セル ジェネシス インコーポレイテッド Ａａｖベクター組成物および免疫グロブリンの発現の増強のための方法ならびにその使用方法
JP2016523835A (ja) * 2013-05-15 2016-08-12 リージェンツ オブ ザ ユニバーシティ オブ ミネソタ 中枢神経系へのアデノ随伴ウイルスを介した遺伝子導入
WO2016200543A2 (en) * 2015-05-13 2016-12-15 The Trustees Of The University Of Pennsylvania Aav-mediated expression of anti-inluenza antibodies and methods of use thereof

Family Cites Families (29)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5196315A (en) * 1990-05-01 1993-03-23 The Johns Hopkins University Human neuronal cell line
CA2099503C (en) * 1991-11-14 2007-10-09 Hamish Steele Scott Synthetic alpha-l-iduronidase and genetic sequences encoding same
CA2379166C (en) 1999-08-09 2013-03-26 Targeted Genetics Corporation Enhancement of expression of a single-stranded, heterologous nucleotide sequence from recombinant viral vectors by designing the sequence such that it forms instrastrand base pairs
US6426208B1 (en) * 1999-11-12 2002-07-30 Harbor-Ucla Research And Education Institute Recombinant α-L-iduronidase, methods for producing and purifying the same and methods for treating diseases caused by deficiencies thereof
US6585971B1 (en) * 1999-11-12 2003-07-01 Harbor-Ucla Research And Education Institute Recombinant α-L-iduronidase, methods for producing and purifying the same and methods for treating disease caused by deficiencies thereof
MX359371B (es) 2001-11-13 2018-09-25 Univ Pennsylvania Un metodo para detectar y/o identificar secuencias del virus adeno-asociado y aislamiento de secuencias novedosas identificadas de ese modo.
PT1453547T (pt) 2001-12-17 2016-12-28 Univ Pennsylvania Sequências do vírus adeno-associado (aav) do serotipo 8, vetores contendo as mesmas, e utilizações destas
EP2345731B1 (en) 2003-09-30 2015-10-21 The Trustees of the University of Pennsylvania Adeno-associated virus (AAV) clades, sequences, vectors containing same, and uses thereof
ES2525067T3 (es) 2005-04-07 2014-12-17 The Trustees Of The University Of Pennsylvania Método de incremento de la función de un vector de AAV
JP4495210B2 (ja) 2005-06-09 2010-06-30 パナソニック株式会社 振幅誤差補償装置及び直交度誤差補償装置
US9375440B2 (en) * 2006-11-03 2016-06-28 Medtronic, Inc. Compositions and methods for making therapies delivered by viral vectors reversible for safety and allele-specificity
US8734809B2 (en) 2009-05-28 2014-05-27 University Of Massachusetts AAV's and uses thereof
US8927514B2 (en) 2010-04-30 2015-01-06 City Of Hope Recombinant adeno-associated vectors for targeted treatment
US8628966B2 (en) 2010-04-30 2014-01-14 City Of Hope CD34-derived recombinant adeno-associated vectors for stem cell transduction and systemic therapeutic gene transfer
WO2012057363A1 (ja) 2010-10-27 2012-05-03 学校法人自治医科大学 神経系細胞への遺伝子導入のためのアデノ随伴ウイルスビリオン
WO2012109570A1 (en) 2011-02-10 2012-08-16 The University Of North Carolina At Chapel Hill Viral vectors with modified transduction profiles and methods of making and using the same
CN105755044A (zh) 2011-04-22 2016-07-13 加利福尼亚大学董事会 具有变异衣壳的腺相关病毒病毒体及其使用方法
ES2857773T5 (es) 2011-08-24 2024-06-04 Univ Leland Stanford Junior Nuevas proteínas de la cápside de AAV para la transferencia de ácidos nucleicos
WO2013170078A1 (en) 2012-05-09 2013-11-14 Oregon Health & Science University Adeno associated virus plasmids and vectors
CA2905952A1 (en) 2013-03-13 2014-10-02 The Children's Hospital Of Philadelphia Adeno-associated virus vectors and methods of use thereof
JP6479777B2 (ja) * 2013-06-11 2019-03-06 ポルタゲ ファーマシューティカルス エルティーディー． 生物学的に活性な特異的カーゴペプチドと結合したヒト由来の細胞透過性ペプチドの構造、製造および使用
WO2015009961A1 (en) * 2013-07-19 2015-01-22 Armagen Technologies, Inc. Compositions and methods related to structures that cross the blood brain barrier
ES2739288T3 (es) 2013-09-13 2020-01-30 California Inst Of Techn Recuperación selectiva
CN106232618A (zh) 2013-10-11 2016-12-14 马萨诸塞眼科耳科诊所 预测祖先病毒序列的方法及其用途
GB201403684D0 (en) 2014-03-03 2014-04-16 King S College London Vector
WO2015164757A1 (en) 2014-04-25 2015-10-29 Oregon Health & Science University Methods of viral neutralizing antibody epitope mapping
EP3151866B1 (en) 2014-06-09 2023-03-08 Voyager Therapeutics, Inc. Chimeric capsids
WO2016064737A1 (en) * 2014-10-20 2016-04-28 Neuralstem, Inc. Stable neural stem cells comprising an exogenous polynucleotide coding for a growth factor and methods of use thereof
US20180185495A1 (en) * 2014-11-10 2018-07-05 Alexion Pharmaceuticals, Inc. Therapeutic compositions of alpha-l-iduronidase, iduronate-2-sulfatase, and alpha-galactosidase a and methods of use thereof

Patent Citations (7)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO1999011665A1 (en) * 1997-09-01 1999-03-11 Kirin Brewery Company, Limited Human thrombopoietin produced by established human cell lines, process for producing the same, and medicinal compositions containing the same
JP2002514429A (ja) * 1998-05-13 2002-05-21 ハーバー−ユーシーエルエー 組換えα−Ｌ−イズロニダーゼ、その生成及び精製方法及びその欠損により引き起こされる疾病の治療方法
JP2004504016A (ja) * 2000-06-30 2004-02-12 マキシゲン・エイピーエス ペプチド拡張されたグリコシル化ポリペプチド
JP2007528862A (ja) * 2003-07-16 2007-10-18 アールブイエックス セラピューティクス， インコーポレイテッド ＴＧＦ−βの効果を下方制御するための化合物および方法
JP2008506389A (ja) * 2004-07-13 2008-03-06 セル ジェネシス インコーポレイテッド Ａａｖベクター組成物および免疫グロブリンの発現の増強のための方法ならびにその使用方法
JP2016523835A (ja) * 2013-05-15 2016-08-12 リージェンツ オブ ザ ユニバーシティ オブ ミネソタ 中枢神経系へのアデノ随伴ウイルスを介した遺伝子導入
WO2016200543A2 (en) * 2015-05-13 2016-12-15 The Trustees Of The University Of Pennsylvania Aav-mediated expression of anti-inluenza antibodies and methods of use thereof

Non-Patent Citations (10)

* Cited by examiner, † Cited by third party
Title
BIOCHEM. J., 1994, VOL.304, PP.43-49, JPN6022004080, ISSN: 0005237574 *
BIOCHEMISTRY, 1991, VOL.30, PP.1533-1537, JPN6022042318, ISSN: 0005237582 *
BIOCHIM. BIOPHYS. ACTA., 2000, VOL.1474, NO.3, PP.273-282, JPN6022042320, ISSN: 0005237580 *
EUR. J. BIOCHEM., 1985, VOL.152, PP.21-28, JPN6022004083, ISSN: 0005237575 *
GENE THER., 2013, VOL.20, PP.450-459, JPN6022004085, ISSN: 0005237577 *
JOURNAL OF PHARMACEUTICAL SCIENCES, 2009, VOL.98, NO.4, PP.1223-1245, JPN6022004088, ISSN: 0005237578 *
MOLECULAR THERAPY, VOL.23, SUPPL.1, P.S281, #706, JPN6022004084, ISSN: 0005237576 *
PNAS, 2013, VOL.110, NO.36, PP.14628-14633, JPN6022042319, ISSN: 0005063659 *
SCIENTIFIC REPORTS, VOL.5, #8926, PP.1-7, JPN6022004089, ISSN: 0005237579 *
比較内分泌学, 2010, VOL.36, NO.136, PP.13-19, JPN6022042321, ISSN: 0005237581 *

Cited By (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2023552443A (ja) * 2020-12-07 2023-12-15 スペースクラフト セブン リミテッド ライアビリティ カンパニー ダノン病の治療

Also Published As

Publication number Publication date
EP3576768A1 (en) 2019-12-11
SG11201906452PA (en) 2019-08-27
BR112019015482A2 (pt) 2020-03-31
IL268076B2 (en) 2024-10-01
IL268076A (en) 2019-09-26
WO2018144441A1 (en) 2018-08-09
IL268076B1 (en) 2024-06-01
CA3049915A1 (en) 2018-08-09
AU2018216807A1 (en) 2019-08-08
JP2024112876A (ja) 2024-08-21
US20190358303A1 (en) 2019-11-28
KR102761412B1 (ko) 2025-02-03
MA47436A (fr) 2019-12-11
KR20250022877A (ko) 2025-02-17
EP3576768A4 (en) 2020-11-04
UY37587A (es) 2018-08-31
KR20190109506A (ko) 2019-09-25
US20240050537A1 (en) 2024-02-15

Similar Documents

Publication Publication Date Title
US20240050537A1 (en) Treatment of mucopolysaccharidosis i with fully-human glycosylated human alpha-l-iduronidase (idua)
US20250051740A1 (en) Treatment of mucopolysaccharidosis ii with recombinant human iduronate-2-sulfatase (ids) produced by human neural or glial cells
JP2024095680A (ja) 完全ヒトグリコシル化ヒトα－Ｌ－イズロニダーゼ（ＩＤＵＡ）を用いたムコ多糖症Ｉ型の治療
US20230059395A1 (en) Treatment of mucopolysaccharidosis i with fully-human glycosylated human alpha-l-iduronidase (idua)

Legal Events

Date Code Title Description
A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20191212

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20210128

A621 Written request for application examination

Free format text: JAPANESE INTERMEDIATE CODE: A621

Effective date: 20210128

A131 Notification of reasons for refusal

Free format text: JAPANESE INTERMEDIATE CODE: A131

Effective date: 20220201

A601 Written request for extension of time

Free format text: JAPANESE INTERMEDIATE CODE: A601

Effective date: 20220427

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20220726

A131 Notification of reasons for refusal

Free format text: JAPANESE INTERMEDIATE CODE: A131

Effective date: 20221011

A601 Written request for extension of time

Free format text: JAPANESE INTERMEDIATE CODE: A601

Effective date: 20230106

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20230308

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A821

Effective date: 20230309

A131 Notification of reasons for refusal

Free format text: JAPANESE INTERMEDIATE CODE: A131

Effective date: 20230523

A601 Written request for extension of time

Free format text: JAPANESE INTERMEDIATE CODE: A601

Effective date: 20230815

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20231122

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A821

Effective date: 20231122

A02 Decision of refusal

Free format text: JAPANESE INTERMEDIATE CODE: A02

Effective date: 20240116