JP2020519278A5 - - Google Patents
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- Publication number
- JP2020519278A5 JP2020519278A5 JP2019562275A JP2019562275A JP2020519278A5 JP 2020519278 A5 JP2020519278 A5 JP 2020519278A5 JP 2019562275 A JP2019562275 A JP 2019562275A JP 2019562275 A JP2019562275 A JP 2019562275A JP 2020519278 A5 JP2020519278 A5 JP 2020519278A5
- Authority
- JP
- Japan
- Prior art keywords
- enzyme
- nucleic acid
- cgl
- modified
- hyperhomocysteinemia
- 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
- 108090000790 Enzymes Proteins 0.000 claims 21
- 102000004190 Enzymes Human genes 0.000 claims 21
- 239000000203 mixture Substances 0.000 claims 8
- 102000039446 nucleic acids Human genes 0.000 claims 8
- 108020004707 nucleic acids Proteins 0.000 claims 8
- 150000007523 nucleic acids Chemical class 0.000 claims 8
- 241000282326 Felis catus Species 0.000 claims 6
- 101000737584 Homo sapiens Cystathionine gamma-lyase Proteins 0.000 claims 6
- 206010020365 Homocystinuria Diseases 0.000 claims 6
- 208000033892 Hyperhomocysteinemia Diseases 0.000 claims 6
- 210000004027 cell Anatomy 0.000 claims 6
- 230000007062 hydrolysis Effects 0.000 claims 6
- 238000006460 hydrolysis reaction Methods 0.000 claims 6
- 230000003225 hyperhomocysteinemia Effects 0.000 claims 6
- 108010045283 Cystathionine gamma-lyase Proteins 0.000 claims 5
- FFFHZYDWPBMWHY-VKHMYHEASA-N L-homocysteine Chemical compound OC(=O)[C@@H](N)CCS FFFHZYDWPBMWHY-VKHMYHEASA-N 0.000 claims 4
- ZTVZLYBCZNMWCF-UHFFFAOYSA-N homocystine Chemical compound [O-]C(=O)C([NH3+])CCSSCCC([NH3+])C([O-])=O ZTVZLYBCZNMWCF-UHFFFAOYSA-N 0.000 claims 4
- 108090000765 processed proteins & peptides Proteins 0.000 claims 4
- 239000002202 Polyethylene glycol Substances 0.000 claims 3
- 241000288906 Primates Species 0.000 claims 3
- 150000001413 amino acids Chemical group 0.000 claims 3
- KWIUHFFTVRNATP-UHFFFAOYSA-N glycine betaine Chemical compound C[N+](C)(C)CC([O-])=O KWIUHFFTVRNATP-UHFFFAOYSA-N 0.000 claims 3
- 229920001223 polyethylene glycol Polymers 0.000 claims 3
- 238000006467 substitution reaction Methods 0.000 claims 3
- 102000009027 Albumins Human genes 0.000 claims 2
- 108010088751 Albumins Proteins 0.000 claims 2
- 241000894006 Bacteria Species 0.000 claims 2
- 102100035429 Cystathionine gamma-lyase Human genes 0.000 claims 2
- 241000238631 Hexapoda Species 0.000 claims 2
- 230000000694 effects Effects 0.000 claims 2
- 150000004676 glycans Chemical class 0.000 claims 2
- 238000002347 injection Methods 0.000 claims 2
- 239000007924 injection Substances 0.000 claims 2
- 229930182817 methionine Natural products 0.000 claims 2
- 125000001360 methionine group Chemical group N[C@@H](CCSC)C(=O)* 0.000 claims 2
- 229920001282 polysaccharide Polymers 0.000 claims 2
- 239000005017 polysaccharide Substances 0.000 claims 2
- 230000002265 prevention Effects 0.000 claims 2
- LXNHXLLTXMVWPM-UHFFFAOYSA-N pyridoxine Chemical compound CC1=NC=C(CO)C(CO)=C1O LXNHXLLTXMVWPM-UHFFFAOYSA-N 0.000 claims 2
- 239000004475 Arginine Substances 0.000 claims 1
- 108020004705 Codon Proteins 0.000 claims 1
- 102000020018 Cystathionine gamma-Lyase Human genes 0.000 claims 1
- 241000588724 Escherichia coli Species 0.000 claims 1
- 241000233866 Fungi Species 0.000 claims 1
- DHMQDGOQFOQNFH-UHFFFAOYSA-N Glycine Natural products NCC(O)=O DHMQDGOQFOQNFH-UHFFFAOYSA-N 0.000 claims 1
- 239000004471 Glycine Substances 0.000 claims 1
- ROHFNLRQFUQHCH-UHFFFAOYSA-N Leucine Natural products CC(C)CC(N)C(O)=O ROHFNLRQFUQHCH-UHFFFAOYSA-N 0.000 claims 1
- 241000124008 Mammalia Species 0.000 claims 1
- 102220482716 Paraneoplastic antigen Ma6E_S63L_mutation Human genes 0.000 claims 1
- 102220533727 Ribonuclease pancreatic_R119D_mutation Human genes 0.000 claims 1
- MTCFGRXMJLQNBG-UHFFFAOYSA-N Serine Natural products OCC(N)C(O)=O MTCFGRXMJLQNBG-UHFFFAOYSA-N 0.000 claims 1
- KZSNJWFQEVHDMF-UHFFFAOYSA-N Valine Natural products CC(C)C(N)C(O)=O KZSNJWFQEVHDMF-UHFFFAOYSA-N 0.000 claims 1
- 239000002253 acid Substances 0.000 claims 1
- ODKSFYDXXFIFQN-UHFFFAOYSA-N arginine Natural products OC(=O)C(N)CCCNC(N)=N ODKSFYDXXFIFQN-UHFFFAOYSA-N 0.000 claims 1
- 125000000637 arginyl group Chemical group N[C@@H](CCCNC(N)=N)C(=O)* 0.000 claims 1
- 235000003704 aspartic acid Nutrition 0.000 claims 1
- CKLJMWTZIZZHCS-REOHCLBHSA-N aspartic acid group Chemical group N[C@@H](CC(=O)O)C(=O)O CKLJMWTZIZZHCS-REOHCLBHSA-N 0.000 claims 1
- 230000001580 bacterial effect Effects 0.000 claims 1
- OQFSQFPPLPISGP-UHFFFAOYSA-N beta-carboxyaspartic acid Natural products OC(=O)C(N)C(C(O)=O)C(O)=O OQFSQFPPLPISGP-UHFFFAOYSA-N 0.000 claims 1
- 229960003237 betaine Drugs 0.000 claims 1
- 230000037213 diet Effects 0.000 claims 1
- 235000005911 diet Nutrition 0.000 claims 1
- 239000003814 drug Substances 0.000 claims 1
- 239000003937 drug carrier Substances 0.000 claims 1
- 239000013604 expression vector Substances 0.000 claims 1
- 238000009472 formulation Methods 0.000 claims 1
- 230000002538 fungal effect Effects 0.000 claims 1
- 125000003630 glycyl group Chemical group [H]N([H])C([H])([H])C(*)=O 0.000 claims 1
- 108010079916 homocysteinase Proteins 0.000 claims 1
- 238000001802 infusion Methods 0.000 claims 1
- 238000001361 intraarterial administration Methods 0.000 claims 1
- 238000007918 intramuscular administration Methods 0.000 claims 1
- 238000007912 intraperitoneal administration Methods 0.000 claims 1
- 238000001990 intravenous administration Methods 0.000 claims 1
- 229960000310 isoleucine Drugs 0.000 claims 1
- AGPKZVBTJJNPAG-UHFFFAOYSA-N isoleucine Natural products CCC(C)C(N)C(O)=O AGPKZVBTJJNPAG-UHFFFAOYSA-N 0.000 claims 1
- 125000000741 isoleucyl group Chemical group [H]N([H])C(C(C([H])([H])[H])C([H])([H])C([H])([H])[H])C(=O)O* 0.000 claims 1
- 125000001909 leucine group Chemical group [H]N(*)C(C(*)=O)C([H])([H])C(C([H])([H])[H])C([H])([H])[H] 0.000 claims 1
- 125000003588 lysine group Chemical group [H]N([H])C([H])([H])C([H])([H])C([H])([H])C([H])([H])C([H])(N([H])[H])C(*)=O 0.000 claims 1
- 210000004962 mammalian cell Anatomy 0.000 claims 1
- 238000004519 manufacturing process Methods 0.000 claims 1
- 239000002773 nucleotide Substances 0.000 claims 1
- 125000003729 nucleotide group Chemical group 0.000 claims 1
- 230000010412 perfusion Effects 0.000 claims 1
- 239000000825 pharmaceutical preparation Substances 0.000 claims 1
- 229920000642 polymer Polymers 0.000 claims 1
- 229920001184 polypeptide Polymers 0.000 claims 1
- 102000004196 processed proteins & peptides Human genes 0.000 claims 1
- RADKZDMFGJYCBB-UHFFFAOYSA-N pyridoxal hydrochloride Natural products CC1=NC=C(CO)C(C=O)=C1O RADKZDMFGJYCBB-UHFFFAOYSA-N 0.000 claims 1
- 102200115297 rs121918072 Human genes 0.000 claims 1
- 102220056008 rs184866544 Human genes 0.000 claims 1
- 102220140316 rs200681141 Human genes 0.000 claims 1
- 125000003607 serino group Chemical group [H]N([H])[C@]([H])(C(=O)[*])C(O[H])([H])[H] 0.000 claims 1
- 210000003954 umbilical cord Anatomy 0.000 claims 1
- 239000004474 valine Substances 0.000 claims 1
- 125000002987 valine group Chemical group [H]N([H])C([H])(C(*)=O)C([H])(C([H])([H])[H])C([H])([H])[H] 0.000 claims 1
- 239000011726 vitamin B6 Substances 0.000 claims 1
- 235000019158 vitamin B6 Nutrition 0.000 claims 1
- 229940011671 vitamin b6 Drugs 0.000 claims 1
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201762505493P | 2017-05-12 | 2017-05-12 | |
| US62/505,493 | 2017-05-12 | ||
| PCT/US2018/032246 WO2018209192A1 (en) | 2017-05-12 | 2018-05-11 | Human-enzyme mediated depletion of homocysteine for treating patients with hyperhomocysteinemia and homocystinuria |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2020519278A JP2020519278A (ja) | 2020-07-02 |
| JP2020519278A5 true JP2020519278A5 (enExample) | 2021-05-27 |
| JP7187040B2 JP7187040B2 (ja) | 2022-12-12 |
Family
ID=62685099
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2019562275A Active JP7187040B2 (ja) | 2017-05-12 | 2018-05-11 | 高ホモシステイン血症およびホモシスチン尿症の患者を処置するための、ヒト酵素介在性のホモシステイン枯渇 |
Country Status (14)
| Country | Link |
|---|---|
| US (2) | US11033612B2 (enExample) |
| EP (1) | EP3622066A1 (enExample) |
| JP (1) | JP7187040B2 (enExample) |
| KR (1) | KR102680776B1 (enExample) |
| CN (1) | CN110603324A (enExample) |
| AU (1) | AU2018266879B2 (enExample) |
| BR (1) | BR112019023800A2 (enExample) |
| CA (1) | CA3060988A1 (enExample) |
| CL (1) | CL2019003248A1 (enExample) |
| CO (1) | CO2019012484A2 (enExample) |
| IL (1) | IL270503B2 (enExample) |
| MX (1) | MX386601B (enExample) |
| WO (1) | WO2018209192A1 (enExample) |
| ZA (1) | ZA201907018B (enExample) |
Families Citing this family (5)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US10363311B2 (en) | 2013-08-29 | 2019-07-30 | Board Of Regents, The University Of Texas System | Engineered primate cystine/cysteine degrading enzymes as antineogenic agents |
| US20190309271A1 (en) | 2018-03-20 | 2019-10-10 | Rubius Therapeutics, Inc. | Therapeutic cell systems and methods for treating homocystinuria |
| EP3870081A4 (en) | 2018-10-26 | 2023-01-18 | Board of Regents, The University of Texas System | GENETIC CYSTINE/CYSTEIN DEGRADING PRIMATE ENZYMES FOR THERAPEUTIC USES |
| EP3952857A4 (en) * | 2019-04-10 | 2023-01-04 | The Regents of the University of Colorado, a Body Corporate | COMPOSITIONS AND METHODS FOR THE TREATMENT OF HOMOCYSTINURIA AND OTHER INFLAMMATORY DISEASES |
| AU2020449880A1 (en) * | 2020-05-29 | 2023-01-19 | The Board Of Regents Of The University Of Texas System | Treatment of homocystinuria and hyperhomocysteinemia using cystathionine-gamma-lyase |
Family Cites Families (21)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US5824311A (en) | 1987-11-30 | 1998-10-20 | Trustees Of The University Of Pennsylvania | Treatment of tumors with monoclonal antibodies against oncogene antigens |
| US4870287A (en) | 1988-03-03 | 1989-09-26 | Loma Linda University Medical Center | Multi-station proton beam therapy system |
| US5329028A (en) | 1992-08-05 | 1994-07-12 | Genentech, Inc. | Carbohydrate-directed cross-linking reagents |
| US5801029A (en) | 1993-02-16 | 1998-09-01 | Onyx Pharmaceuticals, Inc. | Cytopathic viruses for therapy and prophylaxis of neoplasia |
| US5801005A (en) | 1993-03-17 | 1998-09-01 | University Of Washington | Immune reactivity to HER-2/neu protein for diagnosis of malignancies in which the HER-2/neu oncogene is associated |
| US5690959A (en) | 1993-05-29 | 1997-11-25 | Smithkline Beecham Corporation | Pharmaceutical thermal infusion process |
| GB9506466D0 (en) | 1994-08-26 | 1995-05-17 | Prolifix Ltd | Cell cycle regulated repressor and dna element |
| US5760395A (en) | 1996-04-18 | 1998-06-02 | Universities Research Assoc., Inc. | Method and apparatus for laser-controlled proton beam radiology |
| US5739169A (en) | 1996-05-31 | 1998-04-14 | Procept, Incorporated | Aromatic compounds for inhibiting immune response |
| US7118903B2 (en) | 2000-09-05 | 2006-10-10 | Shionogi & Co., Ltd. | L-methionine $g(g)-lyase with modified function |
| WO2002088301A2 (en) | 2001-05-02 | 2002-11-07 | Gavish Galilee Bio Applications Ltd. | Increased methionine in transgenic plants expressing mutant cystathionine gamma-synthase |
| WO2004112717A2 (en) | 2003-06-17 | 2004-12-29 | Harrison Roger G | Conjugate for the specific targeting of anticancer agents to cancer cells and production thereof |
| US20070207158A1 (en) | 2003-06-17 | 2007-09-06 | Harrison Roger G | Conjugate for the specific targeting of anticancer agents to tumor cells or tumor vasculature and production thereof |
| US20050036981A1 (en) | 2003-07-31 | 2005-02-17 | Shigeo Yagi | USE OF PLP WITH PEG-rMETase IN VIVO FOR ENHANCED EFFICACY |
| US20060275279A1 (en) | 2005-06-06 | 2006-12-07 | Rozzell J D | Methods for dissolving cystine stones and reducing cystine in urine |
| BRPI1013141A2 (pt) | 2009-06-15 | 2015-10-06 | Cellagenics B V | novos marcadores selecionaveis rigorosos |
| WO2011097381A2 (en) * | 2010-02-04 | 2011-08-11 | The Board Of Regents Of Use University Of Texas System | Engineered enzymes with methionine-gamma-lyase enzymes and pharmacological preparations therof |
| US9034318B2 (en) * | 2011-08-30 | 2015-05-19 | The Regents Of The University Of Colorado, A Body Corporate | Chemically modified cystathionine beta-synthase enzyme for treatment of homocystinuria |
| WO2015031726A2 (en) * | 2013-08-29 | 2015-03-05 | Board Of Regents, The University Of Texas System | Engineered primate l-methioninase for therapeutic purposes |
| US10363311B2 (en) | 2013-08-29 | 2019-07-30 | Board Of Regents, The University Of Texas System | Engineered primate cystine/cysteine degrading enzymes as antineogenic agents |
| KR20190026813A (ko) | 2016-07-06 | 2019-03-13 | 보드 오브 리전츠, 더 유니버시티 오브 텍사스 시스템 | 시스틴의 인간-효소 매개된 고갈 |
-
2018
- 2018-05-11 CN CN201880029082.9A patent/CN110603324A/zh active Pending
- 2018-05-11 EP EP18732985.9A patent/EP3622066A1/en active Pending
- 2018-05-11 US US15/977,299 patent/US11033612B2/en active Active
- 2018-05-11 JP JP2019562275A patent/JP7187040B2/ja active Active
- 2018-05-11 IL IL270503A patent/IL270503B2/en unknown
- 2018-05-11 KR KR1020197036481A patent/KR102680776B1/ko active Active
- 2018-05-11 CA CA3060988A patent/CA3060988A1/en active Pending
- 2018-05-11 BR BR112019023800-0A patent/BR112019023800A2/pt active Search and Examination
- 2018-05-11 WO PCT/US2018/032246 patent/WO2018209192A1/en not_active Ceased
- 2018-05-11 MX MX2019013458A patent/MX386601B/es unknown
- 2018-05-11 AU AU2018266879A patent/AU2018266879B2/en active Active
-
2019
- 2019-10-24 ZA ZA2019/07018A patent/ZA201907018B/en unknown
- 2019-11-07 CO CONC2019/0012484A patent/CO2019012484A2/es unknown
- 2019-11-11 CL CL2019003248A patent/CL2019003248A1/es unknown
-
2021
- 2021-05-25 US US17/330,106 patent/US12036269B2/en active Active
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