HRP20220407T1 - Pripravci i postupci za uporabu u liječenju homocistinurije - Google Patents
Pripravci i postupci za uporabu u liječenju homocistinurije Download PDFInfo
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- HRP20220407T1 HRP20220407T1 HRP20220407TT HRP20220407T HRP20220407T1 HR P20220407 T1 HRP20220407 T1 HR P20220407T1 HR P20220407T T HRP20220407T T HR P20220407TT HR P20220407 T HRP20220407 T HR P20220407T HR P20220407 T1 HRP20220407 T1 HR P20220407T1
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- mutant polypeptide
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- 239000000203 mixture Substances 0.000 title claims 10
- 238000000034 method Methods 0.000 title claims 8
- 206010020365 Homocystinuria Diseases 0.000 title claims 3
- 229920001184 polypeptide Polymers 0.000 claims 22
- 102000004196 processed proteins & peptides Human genes 0.000 claims 22
- 108090000765 processed proteins & peptides Proteins 0.000 claims 22
- 235000018417 cysteine Nutrition 0.000 claims 8
- XUJNEKJLAYXESH-UHFFFAOYSA-N cysteine Natural products SCC(N)C(O)=O XUJNEKJLAYXESH-UHFFFAOYSA-N 0.000 claims 8
- KWIUHFFTVRNATP-UHFFFAOYSA-N Betaine Natural products C[N+](C)(C)CC([O-])=O KWIUHFFTVRNATP-UHFFFAOYSA-N 0.000 claims 6
- KWIUHFFTVRNATP-UHFFFAOYSA-O N,N,N-trimethylglycinium Chemical compound C[N+](C)(C)CC(O)=O KWIUHFFTVRNATP-UHFFFAOYSA-O 0.000 claims 6
- 229960003237 betaine Drugs 0.000 claims 6
- MTCFGRXMJLQNBG-UHFFFAOYSA-N Serine Natural products OCC(N)C(O)=O MTCFGRXMJLQNBG-UHFFFAOYSA-N 0.000 claims 5
- 235000001014 amino acid Nutrition 0.000 claims 5
- 150000001413 amino acids Chemical group 0.000 claims 5
- 230000035772 mutation Effects 0.000 claims 5
- YPWSLBHSMIKTPR-UHFFFAOYSA-N Cystathionine Natural products OC(=O)C(N)CCSSCC(N)C(O)=O YPWSLBHSMIKTPR-UHFFFAOYSA-N 0.000 claims 4
- ILRYLPWNYFXEMH-UHFFFAOYSA-N D-cystathionine Natural products OC(=O)C(N)CCSCC(N)C(O)=O ILRYLPWNYFXEMH-UHFFFAOYSA-N 0.000 claims 4
- ILRYLPWNYFXEMH-WHFBIAKZSA-N L-cystathionine Chemical compound [O-]C(=O)[C@@H]([NH3+])CCSC[C@H]([NH3+])C([O-])=O ILRYLPWNYFXEMH-WHFBIAKZSA-N 0.000 claims 4
- 108010073644 Cystathionine beta-synthase Proteins 0.000 claims 3
- 102100034976 Cystathionine beta-synthase Human genes 0.000 claims 3
- FFFHZYDWPBMWHY-VKHMYHEASA-N L-homocysteine Chemical compound OC(=O)[C@@H](N)CCS FFFHZYDWPBMWHY-VKHMYHEASA-N 0.000 claims 3
- FFEARJCKVFRZRR-BYPYZUCNSA-N L-methionine Chemical compound CSCC[C@H](N)C(O)=O FFEARJCKVFRZRR-BYPYZUCNSA-N 0.000 claims 3
- ZJUKTBDSGOFHSH-WFMPWKQPSA-N S-Adenosylhomocysteine Chemical compound O[C@@H]1[C@H](O)[C@@H](CSCC[C@H](N)C(O)=O)O[C@H]1N1C2=NC=NC(N)=C2N=C1 ZJUKTBDSGOFHSH-WFMPWKQPSA-N 0.000 claims 3
- 229930182817 methionine Natural products 0.000 claims 3
- 230000037213 diet Effects 0.000 claims 2
- 235000005911 diet Nutrition 0.000 claims 2
- 208000037265 diseases, disorders, signs and symptoms Diseases 0.000 claims 2
- 210000004185 liver Anatomy 0.000 claims 2
- 238000002560 therapeutic procedure Methods 0.000 claims 2
- 101000946497 Homo sapiens Cystathionine beta-synthase Proteins 0.000 claims 1
- 208000001132 Osteoporosis Diseases 0.000 claims 1
- 208000018631 connective tissue disease Diseases 0.000 claims 1
- 230000002950 deficient Effects 0.000 claims 1
- 201000010099 disease Diseases 0.000 claims 1
- 208000035475 disorder Diseases 0.000 claims 1
- 230000003442 weekly effect Effects 0.000 claims 1
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- A—HUMAN NECESSITIES
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- A61K31/19—Carboxylic acids, e.g. valproic acid
- A61K31/195—Carboxylic acids, e.g. valproic acid having an amino group
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- A—HUMAN NECESSITIES
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- 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
- A61K38/51—Lyases (4)
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- A61K31/185—Acids; Anhydrides, halides or salts thereof, e.g. sulfur acids, imidic, hydrazonic or hydroximic acids
- A61K31/19—Carboxylic acids, e.g. valproic acid
- A61K31/195—Carboxylic acids, e.g. valproic acid having an amino group
- A61K31/197—Carboxylic acids, e.g. valproic acid having an amino group the amino and the carboxyl groups being attached to the same acyclic carbon chain, e.g. gamma-aminobutyric acid [GABA], beta-alanine, epsilon-aminocaproic acid or pantothenic acid
- A61K31/198—Alpha-amino acids, e.g. alanine or edetic acid [EDTA]
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- A61K31/205—Amine addition salts of organic acids; Inner quaternary ammonium salts, e.g. betaine, carnitine
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- A61K31/38—Heterocyclic compounds having sulfur as a ring hetero atom
- A61K31/385—Heterocyclic compounds having sulfur as a ring hetero atom having two or more sulfur atoms in the same ring
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- A61K45/06—Mixtures of active ingredients without chemical characterisation, e.g. antiphlogistics and cardiaca
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- C12N9/96—Stabilising an enzyme by forming an adduct or a composition; Forming enzyme conjugates
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- C12Y402/00—Carbon-oxygen lyases (4.2)
- C12Y402/01—Hydro-lyases (4.2.1)
- C12Y402/01022—Cystathionine beta-synthase (4.2.1.22)
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Claims (15)
1. Pripravak koji sadrži izolirani mutantni polipeptid humane skraćene cistationin beta sintaze (htCBS) za uporabu u postupku liječenja homocistinurije kod ljudskog subjekta, navedeni postupak sadrži davanje pripravka subjektu, pri čemu je izolirani htCBS mutantni polipeptid PEGiliran i sadrži mutaciju cisteina na aminokiselinskom položaju 15 od SEQ ID NO: 3 prema serinu, i pri čemu se izolirani htCBS mutantni polipeptid daje u dozi između 0.33 i oko 10 mg/kg najmanje jednom tjedno tijekom najmanje šest tjedana.
2. Pripravak za uporabu prema zahtjevu 1, naznačen time što:
(a) izolirani htCBS mutantni polipeptid je PEGiliran s najmanje jednim odabranim iz skupine koju čine: ME-200GS, ME-200MA0B, ME-400MA, GL4-400MA i ME-050GS;
(b) PEG je linearni PEG niske molekularne mase;
(c) PEG je četverokraki razgranati PEG visoke molekularne mase;
(d) doza je količina odabrana iz skupine koju čine: 0.33, 0.66, oko 1, oko 2, i oko 5 mg/kg;
(e) doza je oko 0.4 mg/kg;
(f) izolirani htCBS mutantni polipeptid se daje zajedno s betainom;
(g) betain se daje prije izoliranog htCBS mutantnog polipeptida; ili
(h) htCBS mutantni polipeptid se daje najmanje dva puta tjedno.
3. Pripravak koji sadrži izolirani mutantni polipeptid humane skraćene cistationin beta sintaze (htCBS) za uporabu u terapijskom postupku povećanja cistationina i/ili cisteina kod ljudskog subjekta koji ima nedostatak CBS-a, navedeni postupak sadrži davanje pripravka subjektu, pri čemu je navedeni izolirani htCBS mutantni polipeptid je PEGiliran i sadrži mutaciju cisteina na aminokiselinskom položaju 15 od SEQ ID NO: 3 prema serinu, i daje se u dozi između 0.33 i oko 10 mg/kg najmanje jednom tjedno tijekom najmanje šest tjedana.
4. Pripravak za uporabu prema zahtjevu 3, naznačen time što:
(a) izolirani htCBS mutantni polipeptid je PEGiliran s najmanje jednim odabranim iz skupine koju čine: ME-200GS, ME-200MA0B, ME-400MA, GL4-400MA i ME-050GS;
(b) htCBS mutantni polipeptid se daje najmanje dva puta tjedno;
(c) PEG je linearni PEG niske molekularne mase;
(d) PEG je četverokraki razgranati PEG visoke molekularne mase;
(e) doza je količina odabrana iz skupine koju čine: 0.33, 0.66, oko 1, oko 2, i oko 5 mg/kg;
(f) doza je oko 0.4 mg/kg;
(g) količina cistationina je povećana na iznad 0.008 µM;
(h) izolirani htCBS mutantni polipeptid se daje zajedno s betainom; ili
(i) betain se daje prije primjene izoliranog htCBS mutantnog polipeptida.
5. Pripravak za uporabu prema zahtjevu 4 opcije (g), naznačen time što:
(i) količina cistationina je povećana na između 0.005-0.35 µM;
(ii) količina cisteina je povećana na iznad 140 µM; ili
(iii) količina cisteina je povećana na između 200 µM do 400 µM.
6. Pripravak koji sadrži izolirani mutantni polipeptid humane skraćene cistationin beta sintaze (htCBS) za uporabu u terapijskom postupku smanjenja homocisteina, metionina, i/ili S-adenozil homocisteina kod čovjeka koji ima nedostatak CBS-a, navedeni postupak sadrži davanje pripravka subjektu, pri čemu je navedeni izolirani htCBS mutantni polipeptid PEGiliran i sadrži mutaciju cisteina na aminokiselinskom položaju 15 od SEQ ID NO: 3 prema serinu, i daje se u dozi između 0.33 i 10 mg/kg najmanje jednom tjedno tijekom najmanje šest tjedana.
7. Pripravak za uporabu prema zahtjevu 6, naznačen time što:
(a) PEG je linearni PEG niske molekularne mase;
(b) PEG je četverokraki razgranati PEG visoke molekularne mase;
(c) doza je količina odabrana iz skupine koju čine: 0.33, 0.66, oko 1, oko 2, i oko 5 mg/kg;
(d) doza je oko 0.4 mg/kg;
(e) količina homocisteina je manja od 100 µM;
(f) izolirani htCBS se istovremeno daje s betainom; ili
(g) betain se daje prije izoliranog htCBS mutantnog polipeptida.
8. Pripravak za uporabu prema zahtjevu 7 opcije (e), naznačen time što je količina homocisteina oko 10 µM.
9. Pripravak za uporabu prema zahtjevu 7 opcije (e), naznačen time što je količina metionina manja od 50 µM pri čemu, izborno:
(i) količina metionina je oko 30 µM; ili
(ii) količina S-adenozil homocisteina je smanjena na manje od 0.14 µM, poželjno, količina S-adenozil homocisteina je smanjena na oko 0.015 µM.
10. Pripravak koji sadrži izolirani mutantni polipeptid humane cistationin beta sintaze (htCBS) za uporabu u postupku liječenja oštećenja parenhima jetre, navedeni postupak sadrži davanje pripravka ljudskom subjektu, pri čemu je navedeni izolirani htCBS mutantni polipeptid PEGiliran i sadrži mutaciju cistein na aminokiselinskom položaju 15 od SEQ ID NO: 3 prema serinu, i pri čemu se izolirani htCBS mutantni polipeptid daje u dozi između 0.33 i 10 mg/kg najmanje jednom dnevno tijekom najmanje šest tjedana.
11. Pripravak za uporabu prema zahtjevu 10, naznačen time što:
(a) PEG je linearni PEG niske molekularne mase;
(b) PEG je četverokraki razgranati PEG visoke molekularne mase;
(c) doza je količina odabrana iz skupine koju čine: 0.33, 0.66, oko 1, oko 2, i oko 5 mg/kg;
(d) doza je oko 0.4 mg/kg; ili
(e) oštećenje parenhima jetre je smanjeno.
12. Pripravak za uporabu prema zahtjevu 1, naznačen time što je navedeni izolirani htCBS mutantni polipeptid PEGiliran sa ME-200GS PEG molekulom.
13. Pripravak za uporabu prema zahtjevu 12, naznačen time što se pripravak primjenjuje u dozi odabranoj iz skupine koju čine: 0.33, 0.66, oko 1, oko 2, i oko 5 mg/kg.
14. Pripravak koji sadrži izolirani mutantni polipeptid humane skraćene cistationin B sintaze (htCBS) koji sadrži mutaciju cisteina na aminokiselinskom položaju 15 od SEQ ID NO: 3 prema serinu za uporabu u postupku liječenja najmanje jedne bolesti, poremećaja, ili stanja odabranog iz skupine koju čine osteoporoza, ili poremećaj vezivnog tkiva koji utječe na okularni sustav, postupak koji sadrži davanje pripravka ljudskom subjektu, pri čemu je navedeni izolirani htCBS mutantni polipeptid PEGiliran s ME-200GS PEG molekulom;
pri čemu se pripravak daje u dozi odabranoj iz raspona od 0.33 mg/kg do oko 10 mg/kg najmanje jednom tjedno tijekom najmanje šest tjedana.
15. Pripravak za uporabu prema zahtjevu 14, naznačen time što subjekt nije na dijeti s ograničenom metioninom; ili je subjekt na dijeti s ograničenom metioninom.
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US14/935,690 US9675678B2 (en) | 2013-01-29 | 2015-11-09 | Compositions and methods for treatment of homocystinuria |
| PCT/US2016/061050 WO2017083327A1 (en) | 2015-11-09 | 2016-11-09 | Compositions and methods for treatment of homocystinuria |
| EP16864878.0A EP3373922B1 (en) | 2015-11-09 | 2016-11-09 | Compositions and methods for use in the treatment of homocystinuria |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| HRP20220407T1 true HRP20220407T1 (hr) | 2022-05-27 |
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Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| HRP20220407TT HRP20220407T1 (hr) | 2015-11-09 | 2016-11-09 | Pripravci i postupci za uporabu u liječenju homocistinurije |
Country Status (25)
| Country | Link |
|---|---|
| US (3) | US11077175B2 (hr) |
| EP (2) | EP3998067A1 (hr) |
| JP (2) | JP7009364B2 (hr) |
| KR (1) | KR20180074703A (hr) |
| CN (2) | CN115920016A (hr) |
| AU (2) | AU2016354030B2 (hr) |
| BR (1) | BR112018007768A2 (hr) |
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Families Citing this family (4)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US9675678B2 (en) | 2013-01-29 | 2017-06-13 | The Regents Of The University Of Colorado, A Body Corporate | Compositions and methods for treatment of homocystinuria |
| SG10201912972SA (en) | 2015-11-09 | 2020-03-30 | Univ Colorado Regents | Compositions and methods for treatment of homocystinuria |
| AU2018254410A1 (en) | 2017-04-17 | 2019-10-10 | The Regents Of The University Of Colorado, A Body Corporate | Optimization of enzyme replacement therapy for treatment of homocystinuria |
| MX2021015966A (es) * | 2019-06-26 | 2022-04-06 | Travere Therapeutics Switzerland Gmbh | Producto farmacológico para terapia enzimática para el tratamiento de la homocistinuria. |
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| JPS5576559A (en) | 1978-12-01 | 1980-06-09 | Chiyou Lsi Gijutsu Kenkyu Kumiai | Electron beam fine adjusting device |
| US5656425A (en) | 1993-08-17 | 1997-08-12 | Regents Of The University Of Colorado | Screening for mutations by expressing cDNA segments |
| US5523225A (en) | 1993-09-13 | 1996-06-04 | Regents Of The University Of Colorado | DNA sequence encoding human cystathionine β-synthase |
| US5919455A (en) | 1993-10-27 | 1999-07-06 | Enzon, Inc. | Non-antigenic branched polymer conjugates |
| US5643575A (en) | 1993-10-27 | 1997-07-01 | Enzon, Inc. | Non-antigenic branched polymer conjugates |
| US5730990A (en) | 1994-06-24 | 1998-03-24 | Enzon, Inc. | Non-antigenic amine derived polymers and polymer conjugates |
| US5635375A (en) | 1995-01-09 | 1997-06-03 | Regents Of The University Of Colorado | Method of increasing the yield and heme saturation of cystathione β-synthase |
| EP1221615A2 (en) | 1995-04-25 | 2002-07-10 | Oridigm Corporation | S-adenosyl methionine regulation of metabolic pathways and its use in diagnosis and therapy |
| US5705151A (en) | 1995-05-18 | 1998-01-06 | National Jewish Center For Immunology & Respiratory Medicine | Gene therapy for T cell regulation |
| US6153655A (en) | 1998-04-17 | 2000-11-28 | Enzon, Inc. | Terminally-branched polymeric linkers and polymeric conjugates containing the same |
| US6174696B1 (en) | 1999-11-12 | 2001-01-16 | Genzyme Corporation | Method for the determination of homocysteine |
| AU5781800A (en) | 1999-07-06 | 2001-01-22 | General Atomics | Methods and compositions for assaying analytes |
| US20030091543A1 (en) | 2001-10-26 | 2003-05-15 | Klein Matthew B. | Therapeutic cell preparation grafts and methods of use thereof |
| WO2003106971A2 (en) | 2002-06-17 | 2003-12-24 | The Regents Of The University Of Colorado | HUMAN CYSTATHIONINE β-SYNTHASE VARIANTS AND METHODS OF PRODU CTION THEREOF |
| GB0216001D0 (en) | 2002-07-10 | 2002-08-21 | Nat Blood Authority | Process and composition |
| EP1396537A1 (en) * | 2002-09-04 | 2004-03-10 | Avidis SA | Treatment of elevated plasma homocysteine |
| CN1552905A (zh) | 2003-12-18 | 2004-12-08 | 中国农业科学院茶叶研究所 | 一类茶树胱硫醚-γ-合成酶特异表达序列标签及其生物芯片 |
| US8500720B2 (en) | 2005-05-09 | 2013-08-06 | Medtronic, Inc | Method and apparatus for treatment of cardiac disorders |
| US20070010492A1 (en) | 2005-07-11 | 2007-01-11 | Generale Robert J | Composition and method for reducing homocysteine caused by drugs containing methyl compounds |
| EP1878739A1 (en) | 2006-07-14 | 2008-01-16 | LEK Pharmaceuticals D.D. | One step IMAC (MCAC) purification of proteins |
| JP5074146B2 (ja) | 2007-03-30 | 2012-11-14 | オリンパス株式会社 | カプセル型医療装置 |
| CN101322840A (zh) | 2008-05-28 | 2008-12-17 | 中国人民解放军军事医学科学院卫生学环境医学研究所 | 一种人胱硫醚β-合成酶重组蛋白及应用 |
| AU2011212885B2 (en) | 2010-02-04 | 2015-10-01 | Aemase, Inc. | Engineered enzymes with methionine-gamma-lyase enzymes and pharmacological preparations thereof |
| WO2012001336A1 (en) | 2010-07-01 | 2012-01-05 | Isis Innovation Limited | Treatment of cognitive disorders |
| 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 |
| US9243239B2 (en) | 2012-03-26 | 2016-01-26 | The Regents Of The University Of Colorado, A Body Corporate | Purification of cystathionine beta-synthase |
| US9675678B2 (en) | 2013-01-29 | 2017-06-13 | The Regents Of The University Of Colorado, A Body Corporate | Compositions and methods for treatment of homocystinuria |
| WO2015033279A1 (en) * | 2013-09-04 | 2015-03-12 | Mahesh Kandula | Compositions and methods for the treatment of homocystinuria |
| SG10201912972SA (en) | 2015-11-09 | 2020-03-30 | Univ Colorado Regents | Compositions and methods for treatment of homocystinuria |
| AU2018254410A1 (en) * | 2017-04-17 | 2019-10-10 | The Regents Of The University Of Colorado, A Body Corporate | Optimization of enzyme replacement therapy for treatment of homocystinuria |
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