SG11201805598WA - Methods and compositions for treating hunter syndrome - Google Patents
Methods and compositions for treating hunter syndromeInfo
- Publication number
- SG11201805598WA SG11201805598WA SG11201805598WA SG11201805598WA SG11201805598WA SG 11201805598W A SG11201805598W A SG 11201805598WA SG 11201805598W A SG11201805598W A SG 11201805598WA SG 11201805598W A SG11201805598W A SG 11201805598WA SG 11201805598W A SG11201805598W A SG 11201805598WA
- Authority
- SG
- Singapore
- Prior art keywords
- gil
- dong
- bojeong
- 30beon
- yongin
- Prior art date
Links
- 238000000034 method Methods 0.000 title abstract 4
- 239000000203 mixture Substances 0.000 title abstract 4
- 201000002273 mucopolysaccharidosis II Diseases 0.000 title abstract 3
- 208000022018 mucopolysaccharidosis type 2 Diseases 0.000 title abstract 3
- 210000004556 brain Anatomy 0.000 abstract 4
- 239000003795 chemical substances by application Substances 0.000 abstract 2
- 102100029199 Iduronate 2-sulfatase Human genes 0.000 abstract 1
- 101710096421 Iduronate 2-sulfatase Proteins 0.000 abstract 1
- 241000699670 Mus sp. Species 0.000 abstract 1
- 210000003169 central nervous system Anatomy 0.000 abstract 1
- 210000002216 heart Anatomy 0.000 abstract 1
- 229950005132 idursulfase beta Drugs 0.000 abstract 1
- 210000003734 kidney Anatomy 0.000 abstract 1
- 210000004185 liver Anatomy 0.000 abstract 1
- 210000004072 lung Anatomy 0.000 abstract 1
- 239000010445 mica Substances 0.000 abstract 1
- 229910052618 mica group Inorganic materials 0.000 abstract 1
- 230000008520 organization Effects 0.000 abstract 1
- 230000002093 peripheral effect Effects 0.000 abstract 1
- 210000000278 spinal cord Anatomy 0.000 abstract 1
- 210000000952 spleen Anatomy 0.000 abstract 1
- 208000024891 symptom Diseases 0.000 abstract 1
- 210000001519 tissue Anatomy 0.000 abstract 1
Classifications
-
- 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
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
- A61K47/06—Organic compounds, e.g. natural or synthetic hydrocarbons, polyolefins, mineral oil, petrolatum or ozokerite
- A61K47/26—Carbohydrates, e.g. sugar alcohols, amino sugars, nucleic acids, mono-, di- or oligo-saccharides; Derivatives thereof, e.g. polysorbates, sorbitan fatty acid esters or glycyrrhizin
-
- 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
- A61K9/00—Medicinal preparations characterised by special physical form
- A61K9/0012—Galenical forms characterised by the site of application
- A61K9/0019—Injectable compositions; Intramuscular, intravenous, arterial, subcutaneous administration; Compositions to be administered through the skin in an invasive manner
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K9/00—Medicinal preparations characterised by special physical form
- A61K9/0012—Galenical forms characterised by the site of application
- A61K9/0085—Brain, e.g. brain implants; Spinal cord
-
- 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
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12Y—ENZYMES
- C12Y301/00—Hydrolases acting on ester bonds (3.1)
- C12Y301/06—Sulfuric ester hydrolases (3.1.6)
- C12Y301/06013—Iduronate-2-sulfatase (3.1.6.13)
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Chemical & Material Sciences (AREA)
- General Health & Medical Sciences (AREA)
- Pharmacology & Pharmacy (AREA)
- Medicinal Chemistry (AREA)
- Veterinary Medicine (AREA)
- Public Health (AREA)
- Animal Behavior & Ethology (AREA)
- Engineering & Computer Science (AREA)
- Epidemiology (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Chemical Kinetics & Catalysis (AREA)
- General Chemical & Material Sciences (AREA)
- Organic Chemistry (AREA)
- Neurology (AREA)
- Biomedical Technology (AREA)
- Neurosurgery (AREA)
- Orthopedic Medicine & Surgery (AREA)
- Psychology (AREA)
- Gastroenterology & Hepatology (AREA)
- Immunology (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Biochemistry (AREA)
- Dermatology (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Oil, Petroleum & Natural Gas (AREA)
- Molecular Biology (AREA)
- Obesity (AREA)
- Hematology (AREA)
- Diabetes (AREA)
- Genetics & Genomics (AREA)
- General Engineering & Computer Science (AREA)
- Wood Science & Technology (AREA)
- Zoology (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Medicinal Preparation (AREA)
- Enzymes And Modification Thereof (AREA)
- External Artificial Organs (AREA)
- Peptides Or Proteins (AREA)
Abstract
INTERNATIONAL APPLICATION PUBLISHED UNDER THE PATENT COOPERATION TREATY (PCT) (19) World Intellectual Property -, Organization M11111011101010111110101111 101E3 01111 0011011111111101111011# 11 International Bureau .. .... ..Yejd (10) International Publication Number (43) International Publication Date ..... .....!;,,, WO 2017/116066 Al 6July 2017 (06.07.2017) WI P0 1 PCT (51) International Patent Classification: 30beon-gil, Giheung-gu, Yongin-si, Gyeonggi-do 16924 A61K 38/43 (2006.01) A61K 9/00 (2006.01) (KR). (21) International Application Number: (74) Agent: DOOHO IP LAW FIRM; (Seocho-dong, Daehy- PCT/KR2016/015060 un Blue Tower) 7th Floor, 1, Gangnam-daero 51-gil, (22) International Filing Date: Seocho-gu, Seoul 06628 (KR). 21 December 2016 (21.12.2016) (81) Designated States (unless otherwise indicated, for every kind of national protection available): AE, AG, AL, AM, (25) Filing Language: English AO, AT, AU, AZ, BA, BB, BG, BH, BN, BR, BW, BY, (26) Publication Language: English BZ, CA, CH, CL, CN, CO, CR, CU, CZ, DE, DJ, DK, DM, DO, DZ, EC, EE, EG, ES, FI, GB, GD, GE, GH, GM, GT, (30) Priority Data: HN, HR, HU, ID, IL, IN, IR, IS, JP, KE, KG, KH, KN, 62/272,843 30 December 2015 (30.12.2015) US KP, KR, KW, KZ, LA, LC, LK, LR, LS, LU, LY, MA, 62/369,970 2 August 2016 (02.08.2016) US MD, ME, MG, MK, MN, MW, MX, MY, MZ, NA, NG, (71) Applicants: GREEN CROSS CORPORATION NI, NO, NZ, OM, PA, PE, PG, PH, PL, PT, QA, RO, RS, [KR/KR]; (Bojeong-dong) 107, Ihyeon-ro 30beon-gil, Gi- RU, RW, SA, SC, SD, SE, SG, SK, SL, SM, ST, SV, SY, heung-gu, Yongin-si, Gyeonggi-do 16924 (KR). MEDI- TH, TJ, TM, TN, TR, TT, TZ, UA, UG, US, UZ, VC, VN, GENEBIO CORPORATION [KR/KR]; (Bojeong-dong) ZA, ZM, ZW. 107, lhyeon-ro 30beon-gil, Giheung-gu, Yongin-si, (84) Designated States (unless otherwise indicated, for every Gyeonggi-do 16924 (KR). kind of regional protection available): ARIPO (BW, GH, (72) Inventors: OKUYAMA, Torayuki; 2-10-1, Okura, GM, KE, LR, LS, MW, MZ, NA, RW, SD, SL, ST, SZ, Setagaya-ku, Tokyo, Tokyo 157-8535 (JP). JIN, Thong- TZ, UG, ZM, ZW), Eurasian (AM, AZ, BY, KG, KZ, RU, — Gyu; E-3806, 57, Eonju-ro 30-gil, Seoul 06293 (KR). TJ, TM), European (AL, AT, BE, BG, CH, CY, CZ, DE, BYUN, Han-Yeul; (Bojeong-dong) 107, Ihyeon-ro DK, EE, ES, FI, FR, GB, GR, HR, HU, IE, IS, IT, LT, LU, 30beon-gil, Giheung-gu, Yongin-si, Gyeonggi-do 16924 LV, MC, MK, MT, NL, NO, PL, PT, RO, RS, SE, SI, SK, = (KR). SEO, Jin-Wook; (Bojeong-dong) 107, Ihyeon-ro SM, TR), OAPI (BF, BJ, CF, CG, CI, CM, GA, GN, GQ, 30beon-gil, Giheung-gu, Yongin-si, Gyeonggi-do 16924 GW, KM, ML, MR, NE, SN, TD, TG). (KR). LEE, Byoung-Ju; (Bojeong-dong) 107, Ihyeon-ro Published: = 30beon-gil, Giheung-gu, Yongin-si, Gyeonggi-do 16924 — (KR). KIM, Yong-Chul; (Bojeong-dong) 107, lhyeon-ro with international search report (Art. 21(3)) = 30beon-gil, Giheung-gu, Yongin-si, Gyeonggi-do 16924 — with sequence listing part of description (Rule 5.2(a)) = (KR). JANG, In-Young; (Bojeong-dong) 107, Ihyeon-ro = = = = = = = Title: METHODS AND COMPOSITIONS FOR TREATING HUNTER SYNDROME = (54) Brain GAG (D7) Brain GAG (D14) Brain GAG (D28) S' b. En 3 t - h. E 2 3 I E 2 1-1 .4 a g • • a 0 < Pt (.0 0 Il g , 0 • ti` ,e , ,,, , b ., , f t , , f , 0 c,' ''. 5 , ; . ' g 0 4' ,a , .,, , f , ,,,O , 4 ,4 , -4. , ,,,i ,; b • S , Q ‹> 41' e At` , ..,, , f° ,0% . 5 ,4 , -e .) 2 f , i e 11 KO mice KO nice KO mica Ir ... ' 1-1 (57) : The present invention provides, among other things, compositions and methods for CNS delivery of Idursulfase-beta, 0 a human recombinant iduronate-2-sulfatase protein, for effective treatment of Hunter Syndrome. The compositions and methods \" provided by the present invention effectively reduce symptoms not only in brain and spinal cord but also in peripheral tissues includ - 0 ing heart, liver, spleen, lung, and kidney.
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201562272843P | 2015-12-30 | 2015-12-30 | |
| US201662369970P | 2016-08-02 | 2016-08-02 | |
| PCT/KR2016/015060 WO2017116066A1 (en) | 2015-12-30 | 2016-12-21 | Methods and compositions for treating hunter syndrome |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| SG11201805598WA true SG11201805598WA (en) | 2018-07-30 |
Family
ID=59224844
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| SG11201805598WA SG11201805598WA (en) | 2015-12-30 | 2016-12-21 | Methods and compositions for treating hunter syndrome |
Country Status (25)
| Country | Link |
|---|---|
| US (1) | US11052135B2 (en) |
| EP (1) | EP3397270B1 (en) |
| JP (2) | JP2019504053A (en) |
| KR (2) | KR20180090387A (en) |
| CN (1) | CN108430494A (en) |
| BR (1) | BR112018013421A2 (en) |
| CL (1) | CL2018001782A1 (en) |
| CO (1) | CO2018007251A2 (en) |
| DK (1) | DK3397270T5 (en) |
| EA (1) | EA201891533A1 (en) |
| ES (1) | ES2978197T3 (en) |
| FI (1) | FI3397270T3 (en) |
| HK (1) | HK1258054A1 (en) |
| HU (1) | HUE066511T2 (en) |
| MX (1) | MX2018008029A (en) |
| MY (1) | MY193846A (en) |
| PE (1) | PE20181329A1 (en) |
| PH (1) | PH12018550102A1 (en) |
| PL (1) | PL3397270T3 (en) |
| PT (1) | PT3397270T (en) |
| RS (1) | RS65513B1 (en) |
| SG (1) | SG11201805598WA (en) |
| SI (1) | SI3397270T1 (en) |
| UA (1) | UA123704C2 (en) |
| WO (1) | WO2017116066A1 (en) |
Families Citing this family (2)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| KR20200058510A (en) | 2017-10-02 | 2020-05-27 | 데날리 테라퓨틱스 인크. | Fusion protein containing enzyme replacement therapy enzyme |
| KR102671857B1 (en) * | 2018-05-30 | 2024-06-04 | 주식회사 녹십자 | Method and composition for treating hunter syndrome by administration of lateral ventricle of cerebrum |
Family Cites Families (10)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US5932211A (en) | 1991-11-12 | 1999-08-03 | Women's And Children's Hospital | Glycosylation variants of iduronate 2-sulfatase |
| US6537785B1 (en) | 1999-09-14 | 2003-03-25 | Genzyme Glycobiology Research Institute, Inc. | Methods of treating lysosomal storage diseases |
| KR102663407B1 (en) * | 2010-06-25 | 2024-05-10 | 샤이어 휴먼 지네틱 테라피즈 인크. | CNS delivery of therapeutic agents |
| KR20230159646A (en) * | 2010-06-25 | 2023-11-21 | 샤이어 휴먼 지네틱 테라피즈 인크. | Methods and compositions for cns delivery of iduronate-2-sulfatase |
| LT3626257T (en) * | 2010-06-25 | 2021-12-10 | Shire Human Genetic Therapies, Inc. | Methods and compositions for cns delivery of arylsulfatase a |
| JP2012062312A (en) | 2010-08-19 | 2012-03-29 | Yoshikatsu Eto | Agent for treatment of hunter syndrome |
| ES2582928T3 (en) | 2011-02-11 | 2016-09-16 | Swedish Orphan Biovitrum Ab (Publ) | Citrate-free pharmaceutical compositions comprising anakinra |
| KR101158673B1 (en) * | 2011-06-24 | 2012-07-03 | 주식회사 지씨바이오 | Composition and formulation comprising recombinant human iduronate-2-sulfatase and preparation method thereof |
| BR112014015352A2 (en) | 2011-12-23 | 2017-06-13 | Shire Human Genetic Therapies | treatment of cognitive impairment of hunter syndrome by intrathecal distribution of iduronate-2-sulfatase |
| US9603908B2 (en) * | 2012-03-30 | 2017-03-28 | Shire Human Genetic Therapies, Inc. | Subcutaneous administration of iduronate-2-sulfatase |
-
2016
- 2016-12-21 PE PE2018001230A patent/PE20181329A1/en unknown
- 2016-12-21 US US16/066,187 patent/US11052135B2/en active Active
- 2016-12-21 SI SI201631823T patent/SI3397270T1/en unknown
- 2016-12-21 SG SG11201805598WA patent/SG11201805598WA/en unknown
- 2016-12-21 RS RS20240556A patent/RS65513B1/en unknown
- 2016-12-21 WO PCT/KR2016/015060 patent/WO2017116066A1/en active Application Filing
- 2016-12-21 CN CN201680076842.2A patent/CN108430494A/en active Pending
- 2016-12-21 PL PL16882018.1T patent/PL3397270T3/en unknown
- 2016-12-21 EP EP16882018.1A patent/EP3397270B1/en active Active
- 2016-12-21 ES ES16882018T patent/ES2978197T3/en active Active
- 2016-12-21 UA UAA201808279A patent/UA123704C2/en unknown
- 2016-12-21 DK DK16882018.1T patent/DK3397270T5/en active
- 2016-12-21 FI FIEP16882018.1T patent/FI3397270T3/en active
- 2016-12-21 MX MX2018008029A patent/MX2018008029A/en unknown
- 2016-12-21 MY MYPI2018001192A patent/MY193846A/en unknown
- 2016-12-21 KR KR1020187021970A patent/KR20180090387A/en not_active Ceased
- 2016-12-21 PT PT168820181T patent/PT3397270T/en unknown
- 2016-12-21 HU HUE16882018A patent/HUE066511T2/en unknown
- 2016-12-21 EA EA201891533A patent/EA201891533A1/en unknown
- 2016-12-21 BR BR112018013421A patent/BR112018013421A2/en active Search and Examination
- 2016-12-21 KR KR1020207023352A patent/KR102272399B1/en active Active
- 2016-12-21 JP JP2018534600A patent/JP2019504053A/en active Pending
-
2018
- 2018-06-26 PH PH12018550102A patent/PH12018550102A1/en unknown
- 2018-06-28 CL CL2018001782A patent/CL2018001782A1/en unknown
- 2018-07-11 CO CONC2018/0007251A patent/CO2018007251A2/en unknown
-
2019
- 2019-01-11 HK HK19100431.6A patent/HK1258054A1/en unknown
-
2020
- 2020-05-26 JP JP2020091152A patent/JP6987924B2/en active Active
Also Published As
| Publication number | Publication date |
|---|---|
| MY193846A (en) | 2022-10-28 |
| WO2017116066A1 (en) | 2017-07-06 |
| SI3397270T1 (en) | 2024-07-31 |
| KR20180090387A (en) | 2018-08-10 |
| JP2019504053A (en) | 2019-02-14 |
| EA201891533A1 (en) | 2018-12-28 |
| KR20200099621A (en) | 2020-08-24 |
| FI3397270T3 (en) | 2024-05-10 |
| RS65513B1 (en) | 2024-06-28 |
| CN108430494A (en) | 2018-08-21 |
| CL2018001782A1 (en) | 2018-11-09 |
| EP3397270B1 (en) | 2024-04-17 |
| US20200268857A1 (en) | 2020-08-27 |
| KR102272399B1 (en) | 2021-07-05 |
| US11052135B2 (en) | 2021-07-06 |
| HUE066511T2 (en) | 2024-08-28 |
| JP2020147578A (en) | 2020-09-17 |
| EP3397270A4 (en) | 2019-09-18 |
| BR112018013421A2 (en) | 2018-12-18 |
| MX2018008029A (en) | 2018-08-23 |
| HK1258054A1 (en) | 2019-11-01 |
| ES2978197T3 (en) | 2024-09-06 |
| DK3397270T5 (en) | 2024-09-23 |
| PL3397270T3 (en) | 2024-08-19 |
| CO2018007251A2 (en) | 2018-07-19 |
| PE20181329A1 (en) | 2018-08-20 |
| DK3397270T3 (en) | 2024-05-06 |
| JP6987924B2 (en) | 2022-01-05 |
| PH12018550102A1 (en) | 2019-02-11 |
| EP3397270A1 (en) | 2018-11-07 |
| UA123704C2 (en) | 2021-05-19 |
| PT3397270T (en) | 2024-05-09 |
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