JPWO2020018665A5 - - Google Patents
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- Publication number
- JPWO2020018665A5 JPWO2020018665A5 JP2021502545A JP2021502545A JPWO2020018665A5 JP WO2020018665 A5 JPWO2020018665 A5 JP WO2020018665A5 JP 2021502545 A JP2021502545 A JP 2021502545A JP 2021502545 A JP2021502545 A JP 2021502545A JP WO2020018665 A5 JPWO2020018665 A5 JP WO2020018665A5
- Authority
- JP
- Japan
- Prior art keywords
- pharmaceutical composition
- human
- idua
- expression vector
- immunosuppressive therapy
- 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
Links
- 101001019502 Homo sapiens Alpha-L-iduronidase Proteins 0.000 claims description 68
- 239000013604 expression vector Substances 0.000 claims description 55
- 238000002650 immunosuppressive therapy Methods 0.000 claims description 54
- 102000056929 human IDUA Human genes 0.000 claims description 47
- 239000002773 nucleotide Substances 0.000 claims description 25
- 125000003729 nucleotide group Chemical group 0.000 claims description 25
- 102100035028 Alpha-L-iduronidase Human genes 0.000 claims description 21
- FDJKUWYYUZCUJX-KVNVFURPSA-N N-glycolylneuraminic acid Chemical compound OC[C@H](O)[C@H](O)[C@@H]1O[C@](O)(C(O)=O)C[C@H](O)[C@H]1NC(=O)CO FDJKUWYYUZCUJX-KVNVFURPSA-N 0.000 claims description 21
- 210000004556 brain Anatomy 0.000 claims description 21
- 206010056886 Mucopolysaccharidosis I Diseases 0.000 claims description 19
- 208000028781 Mucopolysaccharidosis type 1 Diseases 0.000 claims description 19
- 210000001175 cerebrospinal fluid Anatomy 0.000 claims description 16
- 208000036632 Brain mass Diseases 0.000 claims description 13
- QJJXYPPXXYFBGM-LFZNUXCKSA-N Tacrolimus Chemical compound C1C[C@@H](O)[C@H](OC)C[C@@H]1\C=C(/C)[C@@H]1[C@H](C)[C@@H](O)CC(=O)[C@H](CC=C)/C=C(C)/C[C@H](C)C[C@H](OC)[C@H]([C@H](C[C@H]2C)OC)O[C@@]2(O)C(=O)C(=O)N2CCCC[C@H]2C(=O)O1 QJJXYPPXXYFBGM-LFZNUXCKSA-N 0.000 claims description 12
- HPNSFSBZBAHARI-UHFFFAOYSA-N micophenolic acid Natural products OC1=C(CC=C(C)CCC(O)=O)C(OC)=C(C)C2=C1C(=O)OC2 HPNSFSBZBAHARI-UHFFFAOYSA-N 0.000 claims description 12
- 229960000951 mycophenolic acid Drugs 0.000 claims description 12
- HPNSFSBZBAHARI-RUDMXATFSA-N mycophenolic acid Chemical compound OC1=C(C\C=C(/C)CCC(O)=O)C(OC)=C(C)C2=C1C(=O)OC2 HPNSFSBZBAHARI-RUDMXATFSA-N 0.000 claims description 12
- ZAHRKKWIAAJSAO-UHFFFAOYSA-N rapamycin Natural products COCC(O)C(=C/C(C)C(=O)CC(OC(=O)C1CCCCN1C(=O)C(=O)C2(O)OC(CC(OC)C(=CC=CC=CC(C)CC(C)C(=O)C)C)CCC2C)C(C)CC3CCC(O)C(C3)OC)C ZAHRKKWIAAJSAO-UHFFFAOYSA-N 0.000 claims description 12
- 229960002930 sirolimus Drugs 0.000 claims description 12
- QFJCIRLUMZQUOT-HPLJOQBZSA-N sirolimus Chemical compound C1C[C@@H](O)[C@H](OC)C[C@@H]1C[C@@H](C)[C@H]1OC(=O)[C@@H]2CCCCN2C(=O)C(=O)[C@](O)(O2)[C@H](C)CC[C@H]2C[C@H](OC)/C(C)=C/C=C/C=C/[C@@H](C)C[C@@H](C)C(=O)[C@H](OC)[C@H](O)/C(C)=C/[C@@H](C)C(=O)C1 QFJCIRLUMZQUOT-HPLJOQBZSA-N 0.000 claims description 12
- 229960001967 tacrolimus Drugs 0.000 claims description 12
- QJJXYPPXXYFBGM-SHYZHZOCSA-N tacrolimus Natural products CO[C@H]1C[C@H](CC[C@@H]1O)C=C(C)[C@H]2OC(=O)[C@H]3CCCCN3C(=O)C(=O)[C@@]4(O)O[C@@H]([C@H](C[C@H]4C)OC)[C@@H](C[C@H](C)CC(=C[C@@H](CC=C)C(=O)C[C@H](O)[C@H]2C)C)OC QJJXYPPXXYFBGM-SHYZHZOCSA-N 0.000 claims description 12
- 101000718529 Saccharolobus solfataricus (strain ATCC 35092 / DSM 1617 / JCM 11322 / P2) Alpha-galactosidase Proteins 0.000 claims description 11
- NNISLDGFPWIBDF-MPRBLYSKSA-N alpha-D-Gal-(1->3)-beta-D-Gal-(1->4)-D-GlcNAc Chemical compound O[C@@H]1[C@@H](NC(=O)C)C(O)O[C@H](CO)[C@H]1O[C@H]1[C@H](O)[C@@H](O[C@@H]2[C@@H]([C@@H](O)[C@@H](O)[C@@H](CO)O2)O)[C@@H](O)[C@@H](CO)O1 NNISLDGFPWIBDF-MPRBLYSKSA-N 0.000 claims description 11
- 239000000427 antigen Substances 0.000 claims description 11
- 102000036639 antigens Human genes 0.000 claims description 11
- 108091007433 antigens Proteins 0.000 claims description 11
- 238000004113 cell culture Methods 0.000 claims description 10
- 210000003061 neural cell Anatomy 0.000 claims description 10
- 230000006107 tyrosine sulfation Effects 0.000 claims description 8
- VHRSUDSXCMQTMA-PJHHCJLFSA-N 6alpha-methylprednisolone Chemical compound C([C@@]12C)=CC(=O)C=C1[C@@H](C)C[C@@H]1[C@@H]2[C@@H](O)C[C@]2(C)[C@@](O)(C(=O)CO)CC[C@H]21 VHRSUDSXCMQTMA-PJHHCJLFSA-N 0.000 claims description 6
- 239000003246 corticosteroid Substances 0.000 claims description 6
- 229960004584 methylprednisolone Drugs 0.000 claims description 6
- 229960005205 prednisolone Drugs 0.000 claims description 6
- OIGNJSKKLXVSLS-VWUMJDOOSA-N prednisolone Chemical compound O=C1C=C[C@]2(C)[C@H]3[C@@H](O)C[C@](C)([C@@](CC4)(O)C(=O)CO)[C@@H]4[C@@H]3CCC2=C1 OIGNJSKKLXVSLS-VWUMJDOOSA-N 0.000 claims description 6
- 210000002569 neuron Anatomy 0.000 claims description 5
- 230000002463 transducing effect Effects 0.000 claims description 5
- 210000004498 neuroglial cell Anatomy 0.000 claims description 3
- NBSCHQHZLSJFNQ-QTVWNMPRSA-N D-Mannose-6-phosphate Chemical compound OC1O[C@H](COP(O)(O)=O)[C@@H](O)[C@H](O)[C@@H]1O NBSCHQHZLSJFNQ-QTVWNMPRSA-N 0.000 claims description 2
- OUYCCCASQSFEME-QMMMGPOBSA-N L-tyrosine Chemical compound OC(=O)[C@@H](N)CC1=CC=C(O)C=C1 OUYCCCASQSFEME-QMMMGPOBSA-N 0.000 claims description 2
- 108010076504 Protein Sorting Signals Proteins 0.000 claims description 2
- 150000001413 amino acids Chemical group 0.000 claims description 2
- OUYCCCASQSFEME-UHFFFAOYSA-N tyrosine Natural products OC(=O)C(N)CC1=CC=C(O)C=C1 OUYCCCASQSFEME-UHFFFAOYSA-N 0.000 claims description 2
- 239000008194 pharmaceutical composition Substances 0.000 claims 34
- 230000001537 neural effect Effects 0.000 claims 2
- 230000026683 transduction Effects 0.000 claims 1
- 238000010361 transduction Methods 0.000 claims 1
- 238000000034 method Methods 0.000 description 46
- 102000004627 Iduronidase Human genes 0.000 description 20
- 108010003381 Iduronidase Proteins 0.000 description 20
- 230000001419 dependent effect Effects 0.000 description 4
- 239000013598 vector Substances 0.000 description 4
- 230000015572 biosynthetic process Effects 0.000 description 3
- 230000013595 glycosylation Effects 0.000 description 2
- 238000006206 glycosylation reaction Methods 0.000 description 2
- 150000004676 glycans Chemical class 0.000 description 1
Priority Applications (1)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| JP2024040767A JP2024095680A (ja) | 2018-07-18 | 2024-03-15 | 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 (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201862699923P | 2018-07-18 | 2018-07-18 | |
| US62/699,923 | 2018-07-18 | ||
| PCT/US2019/042205 WO2020018665A1 (en) | 2018-07-18 | 2019-07-17 | Treatment of mucopolysaccharidosis i with fully-human glycosylated human alpha-l-iduronidase (idus) |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2024040767A Division JP2024095680A (ja) | 2018-07-18 | 2024-03-15 | 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 (3)
| Publication Number | Publication Date |
|---|---|
| JP2021531276A JP2021531276A (ja) | 2021-11-18 |
| JP2021531276A5 JP2021531276A5 (https=) | 2022-07-26 |
| JPWO2020018665A5 true JPWO2020018665A5 (https=) | 2022-07-26 |
Family
ID=69164759
Family Applications (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2021502545A Pending JP2021531276A (ja) | 2018-07-18 | 2019-07-17 | 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 |
| JP2024040767A Pending JP2024095680A (ja) | 2018-07-18 | 2024-03-15 | 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 |
|---|---|---|---|
| JP2024040767A Pending JP2024095680A (ja) | 2018-07-18 | 2024-03-15 | 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 (10)
| Country | Link |
|---|---|
| US (1) | US20210275647A1 (https=) |
| EP (1) | EP3823590A4 (https=) |
| JP (2) | JP2021531276A (https=) |
| KR (1) | KR20210034037A (https=) |
| AU (1) | AU2019306236A1 (https=) |
| BR (1) | BR112021000830A2 (https=) |
| CA (1) | CA3106651A1 (https=) |
| IL (1) | IL280198B2 (https=) |
| SG (1) | SG11202100423YA (https=) |
| WO (1) | WO2020018665A1 (https=) |
Families Citing this family (3)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| MA50016A (fr) | 2017-07-06 | 2020-07-08 | Univ Pennsylvania | Thérapie génique médiée par aav9 pour traiter la mucopolysaccharidose de type i |
| TWI835747B (zh) | 2017-09-22 | 2024-03-21 | 賓州大學委員會 | 用於治療黏多醣病 ii 型之基因治療 |
| US20190114858A1 (en) * | 2017-10-16 | 2019-04-18 | Raritan Americas, Inc. | System for controlling access to an equipment rack and intelligent power distribution unit and control unit used therein |
Family Cites Families (9)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| WO2001097829A2 (en) * | 2000-06-19 | 2001-12-27 | Genzyme Corporation | Combination enzyme replacement, gene therapy and small molecule therapy for lysosomal storage diseases |
| US7560425B2 (en) * | 2002-06-07 | 2009-07-14 | Waratah Pharmaceuticals Inc. | Pharmaceutical composition consisting of rapamycine and gastrin 17(LEU15) and a method for treating diabetes |
| EP2052083B1 (en) * | 2006-09-21 | 2014-01-08 | The Scripps Research Institute | Genetically programmed expression of selectively sulfated proteins in eubacteria |
| EP3230438B1 (en) * | 2014-12-12 | 2024-12-04 | University of Copenhagen | N-glycosylation |
| HK1256341A1 (zh) * | 2015-05-15 | 2019-09-20 | 明尼苏达大学董事会 | 用於治疗性递送到中枢神经系统的腺相关物 |
| EP3411488A1 (en) * | 2016-02-03 | 2018-12-12 | The Trustees Of The University Of Pennsylvania | Gene therapy for treating mucopolysaccharidosis type i |
| PL3411484T3 (pl) * | 2016-02-05 | 2024-02-19 | Emory University | Wstrzykiwanie jednoniciowego lub samokomplementarnego wirusa adenosatelitarnego 9 do płynu mózgowego |
| EP3541946A1 (en) * | 2016-11-15 | 2019-09-25 | Regents Of The University Of Minnesota | Method for improving neurological function in mpsi and mpsii and other neurological disorders |
| CA3049915A1 (en) * | 2017-01-31 | 2018-08-09 | Stephen YOO | Treatment of mucopolysaccharidosis i with fully-human glycosylated human alpha-l-iduronidase (idua) |
-
2019
- 2019-07-16 IL IL280198A patent/IL280198B2/en unknown
- 2019-07-17 JP JP2021502545A patent/JP2021531276A/ja active Pending
- 2019-07-17 KR KR1020217004709A patent/KR20210034037A/ko active Pending
- 2019-07-17 EP EP19837268.2A patent/EP3823590A4/en active Pending
- 2019-07-17 WO PCT/US2019/042205 patent/WO2020018665A1/en not_active Ceased
- 2019-07-17 CA CA3106651A patent/CA3106651A1/en active Pending
- 2019-07-17 US US17/260,732 patent/US20210275647A1/en active Pending
- 2019-07-17 BR BR112021000830-6A patent/BR112021000830A2/pt not_active Application Discontinuation
- 2019-07-17 AU AU2019306236A patent/AU2019306236A1/en not_active Abandoned
- 2019-07-17 SG SG11202100423YA patent/SG11202100423YA/en unknown
-
2024
- 2024-03-15 JP JP2024040767A patent/JP2024095680A/ja active Pending
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