JP2013526856A5 - - Google Patents
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- Publication number
- JP2013526856A5 JP2013526856A5 JP2013506356A JP2013506356A JP2013526856A5 JP 2013526856 A5 JP2013526856 A5 JP 2013526856A5 JP 2013506356 A JP2013506356 A JP 2013506356A JP 2013506356 A JP2013506356 A JP 2013506356A JP 2013526856 A5 JP2013526856 A5 JP 2013526856A5
- Authority
- JP
- Japan
- Prior art keywords
- asn
- composition
- lal
- glycan structure
- mannose
- 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.)
- Granted
Links
- 239000000203 mixture Substances 0.000 claims 59
- 108010055297 Sterol Esterase Proteins 0.000 claims 43
- 102000000019 Sterol Esterase Human genes 0.000 claims 43
- 101001004953 Homo sapiens Lysosomal acid lipase/cholesteryl ester hydrolase Proteins 0.000 claims 13
- 238000000034 method Methods 0.000 claims 11
- WQZGKKKJIJFFOK-QTVWNMPRSA-N D-mannopyranose Chemical compound OC[C@H]1OC(O)[C@@H](O)[C@@H](O)[C@@H]1O WQZGKKKJIJFFOK-QTVWNMPRSA-N 0.000 claims 10
- SRBFZHDQGSBBOR-IOVATXLUSA-N D-xylopyranose Chemical compound O[C@@H]1COC(O)[C@H](O)[C@H]1O SRBFZHDQGSBBOR-IOVATXLUSA-N 0.000 claims 10
- 150000002703 mannose derivatives Chemical class 0.000 claims 10
- 230000009261 transgenic effect Effects 0.000 claims 9
- 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 8
- FWMNVWWHGCHHJJ-SKKKGAJSSA-N 4-amino-1-[(2r)-6-amino-2-[[(2r)-2-[[(2r)-2-[[(2r)-2-amino-3-phenylpropanoyl]amino]-3-phenylpropanoyl]amino]-4-methylpentanoyl]amino]hexanoyl]piperidine-4-carboxylic acid Chemical compound C([C@H](C(=O)N[C@H](CC(C)C)C(=O)N[C@H](CCCCN)C(=O)N1CCC(N)(CC1)C(O)=O)NC(=O)[C@H](N)CC=1C=CC=CC=1)C1=CC=CC=C1 FWMNVWWHGCHHJJ-SKKKGAJSSA-N 0.000 claims 7
- 241000271566 Aves Species 0.000 claims 7
- 210000004027 cell Anatomy 0.000 claims 7
- 229930182830 galactose Natural products 0.000 claims 7
- PYMYPHUHKUWMLA-UHFFFAOYSA-N arabinose Natural products OCC(O)C(O)C(O)C=O PYMYPHUHKUWMLA-UHFFFAOYSA-N 0.000 claims 5
- SRBFZHDQGSBBOR-UHFFFAOYSA-N beta-D-Pyranose-Lyxose Natural products OC1COC(O)C(O)C1O SRBFZHDQGSBBOR-UHFFFAOYSA-N 0.000 claims 5
- SQVRNKJHWKZAKO-UHFFFAOYSA-N beta-N-Acetyl-D-neuraminic acid Natural products CC(=O)NC1C(O)CC(O)(C(O)=O)OC1C(O)C(O)CO SQVRNKJHWKZAKO-UHFFFAOYSA-N 0.000 claims 5
- 210000003101 oviduct Anatomy 0.000 claims 5
- SQVRNKJHWKZAKO-OQPLDHBCSA-N sialic acid Chemical compound CC(=O)N[C@@H]1[C@@H](O)C[C@@](O)(C(O)=O)OC1[C@H](O)[C@H](O)CO SQVRNKJHWKZAKO-OQPLDHBCSA-N 0.000 claims 5
- SHZGCJCMOBCMKK-UHFFFAOYSA-N D-mannomethylose Natural products CC1OC(O)C(O)C(O)C1O SHZGCJCMOBCMKK-UHFFFAOYSA-N 0.000 claims 4
- PNNNRSAQSRJVSB-SLPGGIOYSA-N Fucose Natural products C[C@H](O)[C@@H](O)[C@H](O)[C@H](O)C=O PNNNRSAQSRJVSB-SLPGGIOYSA-N 0.000 claims 4
- 241000287828 Gallus gallus Species 0.000 claims 4
- SHZGCJCMOBCMKK-DHVFOXMCSA-N L-fucopyranose Chemical compound C[C@@H]1OC(O)[C@@H](O)[C@H](O)[C@@H]1O SHZGCJCMOBCMKK-DHVFOXMCSA-N 0.000 claims 4
- OVRNDRQMDRJTHS-UHFFFAOYSA-N N-acelyl-D-glucosamine Natural products CC(=O)NC1C(O)OC(CO)C(O)C1O OVRNDRQMDRJTHS-UHFFFAOYSA-N 0.000 claims 4
- MBLBDJOUHNCFQT-LXGUWJNJSA-N N-acetylglucosamine Natural products CC(=O)N[C@@H](C=O)[C@@H](O)[C@H](O)[C@H](O)CO MBLBDJOUHNCFQT-LXGUWJNJSA-N 0.000 claims 4
- WYUKJASPBYYQRJ-VSJOFRJTSA-N beta-D-GlcpNAc-(1->2)-alpha-D-Manp-(1->3)-[beta-D-GlcpNAc-(1->2)-alpha-D-Manp-(1->6)]-beta-D-Manp-(1->4)-beta-GlcpNAc-(1->4)-beta-D-GlcpNAc Chemical compound O[C@@H]1[C@@H](NC(=O)C)[C@H](O)O[C@H](CO)[C@H]1O[C@H]1[C@H](NC(C)=O)[C@@H](O)[C@H](O[C@H]2[C@H]([C@@H](O[C@@H]3[C@H]([C@@H](O)[C@H](O)[C@@H](CO)O3)O[C@H]3[C@@H]([C@@H](O)[C@H](O)[C@@H](CO)O3)NC(C)=O)[C@H](O)[C@@H](CO[C@@H]3[C@H]([C@@H](O)[C@H](O)[C@@H](CO)O3)O[C@H]3[C@@H]([C@@H](O)[C@H](O)[C@@H](CO)O3)NC(C)=O)O2)O)[C@@H](CO)O1 WYUKJASPBYYQRJ-VSJOFRJTSA-N 0.000 claims 4
- 239000008194 pharmaceutical composition Substances 0.000 claims 4
- OVRNDRQMDRJTHS-FMDGEEDCSA-N N-acetyl-beta-D-glucosamine Chemical compound CC(=O)N[C@H]1[C@H](O)O[C@H](CO)[C@@H](O)[C@@H]1O OVRNDRQMDRJTHS-FMDGEEDCSA-N 0.000 claims 3
- KRKNYBCHXYNGOX-UHFFFAOYSA-N citric acid Chemical compound OC(=O)CC(O)(C(O)=O)CC(O)=O KRKNYBCHXYNGOX-UHFFFAOYSA-N 0.000 claims 3
- 239000003814 drug Substances 0.000 claims 3
- 210000004602 germ cell Anatomy 0.000 claims 3
- 102000048394 human LIPA Human genes 0.000 claims 3
- 125000000311 mannosyl group Chemical group C1([C@@H](O)[C@@H](O)[C@H](O)[C@H](O1)CO)* 0.000 claims 3
- 229950006780 n-acetylglucosamine Drugs 0.000 claims 3
- 229940124597 therapeutic agent Drugs 0.000 claims 3
- QCVGEOXPDFCNHA-UHFFFAOYSA-N 5,5-dimethyl-2,4-dioxo-1,3-oxazolidine-3-carboxamide Chemical compound CC1(C)OC(=O)N(C(N)=O)C1=O QCVGEOXPDFCNHA-UHFFFAOYSA-N 0.000 claims 2
- 102000002322 Egg Proteins Human genes 0.000 claims 2
- 108010000912 Egg Proteins Proteins 0.000 claims 2
- 208000003221 Lysosomal acid lipase deficiency Diseases 0.000 claims 2
- ZTOKCBJDEGPICW-UHFFFAOYSA-N Man3GlcNAc2 Natural products OC1C(NC(=O)C)C(O)OC(CO)C1OC1C(NC(C)=O)C(O)C(OC2C(C(OC3C(C(O)C(O)C(CO)O3)O)C(O)C(COC3C(C(O)C(O)C(CO)O3)O)O2)O)C(CO)O1 ZTOKCBJDEGPICW-UHFFFAOYSA-N 0.000 claims 2
- 230000004988 N-glycosylation Effects 0.000 claims 2
- NBIIXXVUZAFLBC-UHFFFAOYSA-N Phosphoric acid Chemical compound OP(O)(O)=O NBIIXXVUZAFLBC-UHFFFAOYSA-N 0.000 claims 2
- 108700019146 Transgenes Proteins 0.000 claims 2
- 125000003275 alpha amino acid group Chemical group 0.000 claims 2
- GLEIMNFBCWCWPW-QOTBAUSGSA-N alpha-D-Man-(1->2)-alpha-D-Man-(1->2)-alpha-D-Man-(1->3)-[alpha-D-Man-(1->3)-[alpha-D-Man-(1->6)]-alpha-D-Man-(1->6)]-beta-D-Man-(1->4)-beta-D-GlcNAc-(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](NC(C)=O)[C@@H](O)[C@H](O[C@H]2[C@H]([C@@H](O[C@@H]3[C@H]([C@@H](O)[C@H](O)[C@@H](CO)O3)O[C@@H]3[C@H]([C@@H](O)[C@H](O)[C@@H](CO)O3)O[C@@H]3[C@H]([C@@H](O)[C@H](O)[C@@H](CO)O3)O)[C@H](O)[C@@H](CO[C@@H]3[C@H]([C@@H](O[C@@H]4[C@H]([C@@H](O)[C@H](O)[C@@H](CO)O4)O)[C@H](O)[C@@H](CO[C@@H]4[C@H]([C@@H](O)[C@H](O)[C@@H](CO)O4)O)O3)O)O2)O)[C@@H](CO)O1 GLEIMNFBCWCWPW-QOTBAUSGSA-N 0.000 claims 2
- ZTOKCBJDEGPICW-GWPISINRSA-N alpha-D-Manp-(1->3)-[alpha-D-Manp-(1->6)]-beta-D-Manp-(1->4)-beta-D-GlcpNAc-(1->4)-beta-D-GlcpNAc Chemical compound O[C@@H]1[C@@H](NC(=O)C)[C@H](O)O[C@H](CO)[C@H]1O[C@H]1[C@H](NC(C)=O)[C@@H](O)[C@H](O[C@H]2[C@H]([C@@H](O[C@@H]3[C@H]([C@@H](O)[C@H](O)[C@@H](CO)O3)O)[C@H](O)[C@@H](CO[C@@H]3[C@H]([C@@H](O)[C@H](O)[C@@H](CO)O3)O)O2)O)[C@@H](CO)O1 ZTOKCBJDEGPICW-GWPISINRSA-N 0.000 claims 2
- 239000003795 chemical substances by application Substances 0.000 claims 2
- HVYWMOMLDIMFJA-DPAQBDIFSA-N cholesterol Chemical compound C1C=C2C[C@@H](O)CC[C@]2(C)[C@@H]2[C@@H]1[C@@H]1CC[C@H]([C@H](C)CCCC(C)C)[C@@]1(C)CC2 HVYWMOMLDIMFJA-DPAQBDIFSA-N 0.000 claims 2
- 208000024042 cholesterol ester storage disease Diseases 0.000 claims 2
- 208000013760 cholesteryl ester storage disease Diseases 0.000 claims 2
- 235000014103 egg white Nutrition 0.000 claims 2
- 210000000969 egg white Anatomy 0.000 claims 2
- 235000013601 eggs Nutrition 0.000 claims 2
- FJLGEFLZQAZZCD-MCBHFWOFSA-N (3R,5S)-fluvastatin Chemical compound C12=CC=CC=C2N(C(C)C)C(\C=C\[C@@H](O)C[C@@H](O)CC(O)=O)=C1C1=CC=C(F)C=C1 FJLGEFLZQAZZCD-MCBHFWOFSA-N 0.000 claims 1
- XUKUURHRXDUEBC-KAYWLYCHSA-N Atorvastatin Chemical compound C=1C=CC=CC=1C1=C(C=2C=CC(F)=CC=2)N(CC[C@@H](O)C[C@@H](O)CC(O)=O)C(C(C)C)=C1C(=O)NC1=CC=CC=C1 XUKUURHRXDUEBC-KAYWLYCHSA-N 0.000 claims 1
- XUKUURHRXDUEBC-UHFFFAOYSA-N Atorvastatin Natural products C=1C=CC=CC=1C1=C(C=2C=CC(F)=CC=2)N(CCC(O)CC(O)CC(O)=O)C(C(C)C)=C1C(=O)NC1=CC=CC=C1 XUKUURHRXDUEBC-UHFFFAOYSA-N 0.000 claims 1
- 102000008100 Human Serum Albumin Human genes 0.000 claims 1
- 108091006905 Human Serum Albumin Proteins 0.000 claims 1
- PCZOHLXUXFIOCF-UHFFFAOYSA-N Monacolin X Natural products C12C(OC(=O)C(C)CC)CC(C)C=C2C=CC(C)C1CCC1CC(O)CC(=O)O1 PCZOHLXUXFIOCF-UHFFFAOYSA-N 0.000 claims 1
- OVRNDRQMDRJTHS-RTRLPJTCSA-N N-acetyl-D-glucosamine Chemical compound CC(=O)N[C@H]1C(O)O[C@H](CO)[C@@H](O)[C@@H]1O OVRNDRQMDRJTHS-RTRLPJTCSA-N 0.000 claims 1
- 108091028043 Nucleic acid sequence Proteins 0.000 claims 1
- TUZYXOIXSAXUGO-UHFFFAOYSA-N Pravastatin Natural products C1=CC(C)C(CCC(O)CC(O)CC(O)=O)C2C(OC(=O)C(C)CC)CC(O)C=C21 TUZYXOIXSAXUGO-UHFFFAOYSA-N 0.000 claims 1
- RYMZZMVNJRMUDD-UHFFFAOYSA-N SJ000286063 Natural products C12C(OC(=O)C(C)(C)CC)CC(C)C=C2C=CC(C)C1CCC1CC(O)CC(=O)O1 RYMZZMVNJRMUDD-UHFFFAOYSA-N 0.000 claims 1
- 208000026589 Wolman disease Diseases 0.000 claims 1
- 239000000654 additive Substances 0.000 claims 1
- 230000000996 additive effect Effects 0.000 claims 1
- 229910000147 aluminium phosphate Inorganic materials 0.000 claims 1
- 239000007864 aqueous solution Substances 0.000 claims 1
- 229960005370 atorvastatin Drugs 0.000 claims 1
- 235000012000 cholesterol Nutrition 0.000 claims 1
- 239000003085 diluting agent Substances 0.000 claims 1
- 239000003937 drug carrier Substances 0.000 claims 1
- 229960003765 fluvastatin Drugs 0.000 claims 1
- 210000004907 gland Anatomy 0.000 claims 1
- 230000013595 glycosylation Effects 0.000 claims 1
- 238000006206 glycosylation reaction Methods 0.000 claims 1
- 229940125721 immunosuppressive agent Drugs 0.000 claims 1
- 239000003018 immunosuppressive agent Substances 0.000 claims 1
- 229960004844 lovastatin Drugs 0.000 claims 1
- PCZOHLXUXFIOCF-BXMDZJJMSA-N lovastatin Chemical compound C([C@H]1[C@@H](C)C=CC2=C[C@H](C)C[C@@H]([C@H]12)OC(=O)[C@@H](C)CC)C[C@@H]1C[C@@H](O)CC(=O)O1 PCZOHLXUXFIOCF-BXMDZJJMSA-N 0.000 claims 1
- QLJODMDSTUBWDW-UHFFFAOYSA-N lovastatin hydroxy acid Natural products C1=CC(C)C(CCC(O)CC(O)CC(O)=O)C2C(OC(=O)C(C)CC)CC(C)C=C21 QLJODMDSTUBWDW-UHFFFAOYSA-N 0.000 claims 1
- 150000007523 nucleic acids Chemical group 0.000 claims 1
- 150000002482 oligosaccharides Polymers 0.000 claims 1
- 229960002797 pitavastatin Drugs 0.000 claims 1
- VGYFMXBACGZSIL-MCBHFWOFSA-N pitavastatin Chemical compound OC(=O)C[C@H](O)C[C@H](O)\C=C\C1=C(C2CC2)N=C2C=CC=CC2=C1C1=CC=C(F)C=C1 VGYFMXBACGZSIL-MCBHFWOFSA-N 0.000 claims 1
- 229960002965 pravastatin Drugs 0.000 claims 1
- TUZYXOIXSAXUGO-PZAWKZKUSA-N pravastatin Chemical compound C1=C[C@H](C)[C@H](CC[C@@H](O)C[C@@H](O)CC(O)=O)[C@H]2[C@@H](OC(=O)[C@@H](C)CC)C[C@H](O)C=C21 TUZYXOIXSAXUGO-PZAWKZKUSA-N 0.000 claims 1
- 229960000672 rosuvastatin Drugs 0.000 claims 1
- BPRHUIZQVSMCRT-VEUZHWNKSA-N rosuvastatin Chemical compound CC(C)C1=NC(N(C)S(C)(=O)=O)=NC(C=2C=CC(F)=CC=2)=C1\C=C\[C@@H](O)C[C@@H](O)CC(O)=O BPRHUIZQVSMCRT-VEUZHWNKSA-N 0.000 claims 1
- 229960002855 simvastatin Drugs 0.000 claims 1
- RYMZZMVNJRMUDD-HGQWONQESA-N simvastatin Chemical compound C([C@H]1[C@@H](C)C=CC2=C[C@H](C)C[C@@H]([C@H]12)OC(=O)C(C)(C)CC)C[C@@H]1C[C@@H](O)CC(=O)O1 RYMZZMVNJRMUDD-HGQWONQESA-N 0.000 claims 1
- 239000001509 sodium citrate Substances 0.000 claims 1
- HRXKRNGNAMMEHJ-UHFFFAOYSA-K trisodium citrate Chemical compound [Na+].[Na+].[Na+].[O-]C(=O)CC(O)(CC([O-])=O)C([O-])=O HRXKRNGNAMMEHJ-UHFFFAOYSA-K 0.000 claims 1
- 229940038773 trisodium citrate Drugs 0.000 claims 1
Applications Claiming Priority (11)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US34317710P | 2010-04-23 | 2010-04-23 | |
| US61/343,177 | 2010-04-23 | ||
| US39637610P | 2010-05-26 | 2010-05-26 | |
| US61/396,376 | 2010-05-26 | ||
| US40301110P | 2010-09-09 | 2010-09-09 | |
| US61/403,011 | 2010-09-09 | ||
| US45601410P | 2010-10-29 | 2010-10-29 | |
| US61/456,014 | 2010-10-29 | ||
| US201161432372P | 2011-01-13 | 2011-01-13 | |
| US61/432,372 | 2011-01-13 | ||
| PCT/US2011/033699 WO2011133960A2 (en) | 2010-04-23 | 2011-04-23 | Lysosomal storage disease enzyme |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2015160139A Division JP6212081B2 (ja) | 2010-04-23 | 2015-08-14 | リソソーム蓄積症酵素 |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2013526856A JP2013526856A (ja) | 2013-06-27 |
| JP2013526856A5 true JP2013526856A5 (https=) | 2015-01-29 |
| JP5925187B2 JP5925187B2 (ja) | 2016-05-25 |
Family
ID=44834852
Family Applications (4)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2013506356A Active JP5925187B2 (ja) | 2010-04-23 | 2011-04-23 | リソソーム蓄積症酵素 |
| JP2015160139A Active JP6212081B2 (ja) | 2010-04-23 | 2015-08-14 | リソソーム蓄積症酵素 |
| JP2016151739A Active JP6496690B2 (ja) | 2010-04-23 | 2016-08-02 | リソソーム蓄積症酵素 |
| JP2018023329A Pending JP2018100289A (ja) | 2010-04-23 | 2018-02-13 | リソソーム蓄積症酵素 |
Family Applications After (3)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2015160139A Active JP6212081B2 (ja) | 2010-04-23 | 2015-08-14 | リソソーム蓄積症酵素 |
| JP2016151739A Active JP6496690B2 (ja) | 2010-04-23 | 2016-08-02 | リソソーム蓄積症酵素 |
| JP2018023329A Pending JP2018100289A (ja) | 2010-04-23 | 2018-02-13 | リソソーム蓄積症酵素 |
Country Status (21)
| Country | Link |
|---|---|
| US (4) | US20130209436A1 (https=) |
| EP (3) | EP4241854A3 (https=) |
| JP (4) | JP5925187B2 (https=) |
| KR (1) | KR101742346B1 (https=) |
| CN (2) | CN102985538B (https=) |
| AU (1) | AU2011242461B2 (https=) |
| BR (1) | BR112012027143B1 (https=) |
| CA (2) | CA2796607C (https=) |
| DK (2) | DK2561069T3 (https=) |
| ES (2) | ES2627535T3 (https=) |
| FI (1) | FI3205351T3 (https=) |
| HK (1) | HK1219655A1 (https=) |
| HU (2) | HUE062163T2 (https=) |
| IL (2) | IL222513A0 (https=) |
| LT (1) | LT3205351T (https=) |
| MX (2) | MX338426B (https=) |
| PL (2) | PL2561069T3 (https=) |
| PT (2) | PT2561069T (https=) |
| RU (2) | RU2015151189A (https=) |
| SI (2) | SI2561069T1 (https=) |
| WO (1) | WO2011133960A2 (https=) |
Families Citing this family (15)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| ES2328446T5 (es) | 2000-02-04 | 2014-02-27 | Children's Hospital Research Foundation | Uso de lipasa ácida lisosomal para tratar la aterosclerosis y enfermedades asociadas |
| WO2007030375A2 (en) * | 2005-09-08 | 2007-03-15 | Children's Hospital Medical Center | Lysosomal acid lipase therapy for nafld and related diseases |
| AU2007345347B2 (en) * | 2007-01-26 | 2013-11-07 | Synageva Biopharma Corp | Transgene expression in avians |
| PT2561069T (pt) | 2010-04-23 | 2017-05-22 | Alexion Pharma Inc | Enzima de doença de armazenamento lisossomal |
| CA2810999C (en) | 2010-09-09 | 2023-10-03 | Anthony Quinn | Use of lysosomal acid lipase for treating lysosomal acid lipase deficiency in patients |
| EP2675472A4 (en) | 2011-02-15 | 2014-09-17 | Synageva Biopharma Corp | METHOD FOR THE TREATMENT OF LACK OF LYSOSOMAL SAURER LIPASE |
| WO2013131014A1 (en) * | 2012-03-02 | 2013-09-06 | Synageva Biopharma Corp. | Truncated lysosomal acid lipase |
| BR112016024586B1 (pt) * | 2014-04-23 | 2022-03-29 | Alexion Pharmaceuticals, Inc | Método de preparação de clara de ovo para o processamento cromatográfico em massa |
| US11235036B2 (en) | 2016-06-17 | 2022-02-01 | Alexion Pharmaceuticals, Inc. | Lysosomal acid lipase deficiency compositions and methods |
| JP7027347B2 (ja) * | 2016-08-23 | 2022-03-01 | アレクシオン ファーマシューティカルズ インコーポレイテッド | 卵白から異種タンパク質を精製する方法 |
| ES2888126T3 (es) | 2016-09-30 | 2021-12-30 | Alexion Pharma Inc | Sebelipasa alfa para su uso en fibrosis hepática y para tratar la deficiencia de lipasa ácida lisosomal en pacientes según el estadio de fibrosis de Ishak |
| US10633705B2 (en) | 2016-10-14 | 2020-04-28 | Alexion Pharmaceuticals, Inc. | N-acetyl-alpha-D-glucosaminidase deficiency compositions and methods |
| WO2019152343A1 (en) | 2018-02-01 | 2019-08-08 | Alexion Pharmaceuticals, Inc. | Methods of desensitizing against infusion associated reactions in lysosomal acid lipase deficiency (lal-d) patients treated with exogenous lysosomal acid lipase (lal |
| US12492412B2 (en) * | 2019-07-02 | 2025-12-09 | M6P Terapeutics (Switzerland) Llc | Vector compositions and methods of using same for treatment of lysosomal storage disorders |
| CN114369620B (zh) * | 2021-12-31 | 2024-05-24 | 广州博垚生物科技有限公司 | 一种可持续分泌lal的载体、脐带间充质干细胞及其构建方法和应用 |
Family Cites Families (62)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| SE9001985D0 (sv) | 1990-06-01 | 1990-06-01 | Astra Ab | New chemical products |
| AU1580292A (en) | 1991-03-13 | 1992-10-21 | United States of America, as represented by the Secretary, U.S. Department of Commerce, The | Increasing the therapeutic efficiency of macrophage-targeted therapeutic agents by up-regulating the mannose lectin on macrophages |
| US5631365A (en) | 1993-09-21 | 1997-05-20 | Schering Corporation | Hydroxy-substituted azetidinone compounds useful as hypocholesterolemic agents |
| AU3275595A (en) | 1994-08-05 | 1996-03-04 | Molecular/Structural Biotechnologies, Inc. | Site-specific biomolecular complexes |
| US6118045A (en) | 1995-08-02 | 2000-09-12 | Pharming B.V. | Lysosomal proteins produced in the milk of transgenic animals |
| DE69637875D1 (de) | 1995-09-14 | 2009-04-30 | Virginia Tech Intell Prop | Produktion von lysosomalen enzymen in pflanzlichen expressionssystemen |
| JP4001925B2 (ja) | 1996-09-13 | 2007-10-31 | トランスカーヨティック セラピーズ インク． | α―ガラクトシダーゼＡ欠損症の治療 |
| US5897998A (en) | 1997-08-04 | 1999-04-27 | The University Of Georgia Research Foundation, Inc. | Method for manipulating avian eggs |
| IL135578A0 (en) | 1997-10-29 | 2001-05-20 | Genzyme Corp | Compositions and methods for treating lysosomal storage disease |
| US20030166132A1 (en) | 1998-08-26 | 2003-09-04 | Genentech, Inc. | Secreted and transmembrane polypeptides and nucleic acids encoding the same |
| DK1137762T3 (da) | 1998-12-07 | 2009-02-02 | Genzyme Corp | Behandling af Pompes sygdom |
| US7033780B1 (en) | 1999-06-14 | 2006-04-25 | Millennium Pharmaceuticals, Inc. | Nucleic acids corresponding to TANGO 294 a gene encoding a lipase—like protein |
| US7927587B2 (en) | 1999-08-05 | 2011-04-19 | Regents Of The University Of Minnesota | MAPC administration for the treatment of lysosomal storage disorders |
| US6537785B1 (en) | 1999-09-14 | 2003-03-25 | Genzyme Glycobiology Research Institute, Inc. | Methods of treating lysosomal storage diseases |
| ES2328446T5 (es) | 2000-02-04 | 2014-02-27 | Children's Hospital Research Foundation | Uso de lipasa ácida lisosomal para tratar la aterosclerosis y enfermedades asociadas |
| WO2001097829A2 (en) | 2000-06-19 | 2001-12-27 | Genzyme Corporation | Combination enzyme replacement, gene therapy and small molecule therapy for lysosomal storage diseases |
| US20040038365A1 (en) | 2000-10-31 | 2004-02-26 | Yonghong Xiao | Regulation of human lysosomal acid lipase |
| US20030064437A1 (en) | 2000-11-15 | 2003-04-03 | Nick Wan | Expression system for recombinant proteins |
| US20020193303A1 (en) | 2001-01-25 | 2002-12-19 | Millennium Pharmaceuticals, Inc. | 58860, a human cholesteryl ester hydrolase and uses therefor |
| US6387680B1 (en) * | 2001-03-29 | 2002-05-14 | Pe Corporation (Ny) | Isolated human lipase proteins, nucleic acid molecules encoding human lipase proteins, and uses thereof |
| US6800472B2 (en) | 2001-12-21 | 2004-10-05 | Genzyme Glycobiology Research Institute, Inc. | Expression of lysosomal hydrolase in cells expressing pro-N-acetylglucosamine-1-phosphodiester α-N-acetyl glucosimanidase |
| AU2003207744A1 (en) | 2002-01-30 | 2003-09-02 | Yale University | Transport peptides and uses therefor |
| ES2498369T3 (es) | 2002-04-16 | 2014-09-24 | Vlaams Interuniversitair Instituut Voor Biotechnologie Vzw. | Un marcador para medir cirrosis hepática |
| EP1546397A4 (en) | 2002-09-27 | 2007-10-31 | Cold Spring Harbor Lab | CELL BASED RNA INTERFERENCE AND ASSOCIATED METHODS AND COMPOSITIONS |
| US20050208090A1 (en) | 2004-03-18 | 2005-09-22 | Medtronic, Inc. | Methods and systems for treatment of neurological diseases of the central nervous system |
| WO2006002053A2 (en) | 2004-06-15 | 2006-01-05 | Mayo Foundation For Medical Education And Research | Helicases |
| NZ553420A (en) | 2004-08-19 | 2010-02-26 | Biogen Idec Inc | Refolding transforming growth factor beta family proteins |
| US20070270367A1 (en) | 2005-05-18 | 2007-11-22 | University Of Kentucky Research Foundation | Rybozyme-catalyzed insertion of targeted sequences into RNA |
| US8124732B2 (en) * | 2005-06-24 | 2012-02-28 | Synageva Biopharma Corp. | Composition comprising isolated human CTLA4-Fc fusion protein produced in a transgenic chicken |
| WO2007030375A2 (en) | 2005-09-08 | 2007-03-15 | Children's Hospital Medical Center | Lysosomal acid lipase therapy for nafld and related diseases |
| PL377180A1 (pl) | 2005-09-21 | 2007-04-02 | Instytut Farmaceutyczny | Zastosowanie izoflawonów i ich pochodnych w leczeniu mukopolisacharydoz |
| US7524626B2 (en) | 2005-10-05 | 2009-04-28 | Synageva Biopharma Corp. | Rapid production of high titer virus |
| AU2006304856A1 (en) | 2005-10-21 | 2007-04-26 | Synageva Biopharma Corp. | Glycolated and glycosylated poultry derived therapeutic proteins |
| AR059089A1 (es) | 2006-01-20 | 2008-03-12 | Genzyme Corp | Administracion intraventricular de una enzima para enfermedades de almacenamiento lisosomal |
| WO2008054544A2 (en) | 2006-05-22 | 2008-05-08 | Immune Disease Institute, Inc. | Method for delivery across the blood brain barrier |
| WO2007137303A2 (en) | 2006-05-24 | 2007-11-29 | Myelin Repair Foundation, Inc. | Permeability of blood-brain barrier |
| AU2007345347B2 (en) | 2007-01-26 | 2013-11-07 | Synageva Biopharma Corp | Transgene expression in avians |
| US8143265B2 (en) | 2007-04-16 | 2012-03-27 | Meharry Medical College | Method of treating atherosclerosis |
| US8466118B2 (en) | 2007-04-23 | 2013-06-18 | Saint Louis University | Modulation of blood brain barrier protein expression |
| EP3486653A1 (en) | 2007-05-24 | 2019-05-22 | The United States Government as represented by The Department of Veterans Affairs | Treatment of skeletal muscle disorder using ent2 |
| AU2008255565B2 (en) | 2007-05-31 | 2013-10-24 | Paradigm Biopharmaceuticals Ltd | Sulphated xylans for treatment or prophylaxis of respiratory diseases |
| CA2693742A1 (en) | 2007-07-12 | 2009-01-15 | Prosensa Technologies B.V. | Molecules for targeting compounds to various selected organs, tissues or tumor cells |
| EP2185187A1 (en) | 2007-08-29 | 2010-05-19 | Shire Human Genetic Therapies, Inc. | Subcutaneous administration of alpha-galatosidase a |
| WO2009088998A2 (en) * | 2008-01-07 | 2009-07-16 | Synageva Biopharma Corp. | Glycosylation in avians |
| FR2937322B1 (fr) | 2008-10-22 | 2013-02-22 | Vect Horus | Derives peptidiques et leur utilisation comme vecteurs de molecules sous forme de conjugues |
| US8232073B2 (en) | 2009-01-02 | 2012-07-31 | Zacharon Pharmaceuticals, Inc. | Quantification of non-reducing end glycan residual compounds |
| WO2010078515A2 (en) | 2009-01-02 | 2010-07-08 | Zacharon Pharmaceuticals, Inc. | Polymer end group detection |
| US8257694B2 (en) | 2009-05-14 | 2012-09-04 | Product Partners, Llc | Nutritional compositions for reducing oxidative damage |
| ES2576853T3 (es) | 2009-07-03 | 2016-07-11 | Inserm - Institut National De La Santé Et De La Recherche Médicale | Compuestos dirigidos al receptor del manosa 6-fosfato independiente de cationes |
| WO2011049737A1 (en) | 2009-10-19 | 2011-04-28 | Amicus Therapeutics, Inc. | Novel compositions for preventing and/or treating lysosomal storage disorders |
| KR101077618B1 (ko) | 2009-11-13 | 2011-10-27 | 경북대학교 산학협력단 | 뇌혈관 장벽을 통과하여 퇴행성 뇌신경 질환 부위의 세포사멸을 표적하는 펩타이드 및 이의 용도 |
| US9095541B2 (en) | 2009-11-24 | 2015-08-04 | Arch Cancer Therapeutics, Inc. | Brain tumor targeting peptides and methods |
| PT2561069T (pt) * | 2010-04-23 | 2017-05-22 | Alexion Pharma Inc | Enzima de doença de armazenamento lisossomal |
| US20150030582A1 (en) * | 2010-04-23 | 2015-01-29 | Synageva Biopharma Corp. | Lysosomal Storage Disease Enzyme |
| CA2810999C (en) * | 2010-09-09 | 2023-10-03 | Anthony Quinn | Use of lysosomal acid lipase for treating lysosomal acid lipase deficiency in patients |
| WO2012112677A2 (en) | 2011-02-15 | 2012-08-23 | Children's Hospital Medical Center | Methods for treating lysosomal acid lipase deficiency |
| EP2675472A4 (en) | 2011-02-15 | 2014-09-17 | Synageva Biopharma Corp | METHOD FOR THE TREATMENT OF LACK OF LYSOSOMAL SAURER LIPASE |
| US8865881B2 (en) | 2011-02-22 | 2014-10-21 | California Institute Of Technology | Delivery of proteins using adeno-associated virus (AAV) vectors |
| EP2709670A4 (en) | 2011-05-18 | 2015-01-21 | Childrens Hosp Medical Center | TARGETED TRANSFER OF PROTEINS OVER THE BLOOD BRAIN BARRIER |
| WO2012177778A1 (en) | 2011-06-20 | 2012-12-27 | Mount Sinai School Of Medicine | Anti-tnf- therapy for the mucopolysaccharidoses and other lysosomal disorders |
| WO2012177639A2 (en) | 2011-06-22 | 2012-12-27 | Alnylam Pharmaceuticals, Inc. | Bioprocessing and bioproduction using avian cell lines |
| WO2013020064A1 (en) | 2011-08-03 | 2013-02-07 | Lotus Tissue Repair Inc. | Collagen 7 and related methods |
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