SI2704737T1 - Terapevtski sestavki nukleaze in postopki - Google Patents
Terapevtski sestavki nukleaze in postopki Download PDFInfo
- Publication number
- SI2704737T1 SI2704737T1 SI201231276T SI201231276T SI2704737T1 SI 2704737 T1 SI2704737 T1 SI 2704737T1 SI 201231276 T SI201231276 T SI 201231276T SI 201231276 T SI201231276 T SI 201231276T SI 2704737 T1 SI2704737 T1 SI 2704737T1
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- Slovenia
- Prior art keywords
- hybrid nuclease
- hybrid
- nuclease molecule
- molecule
- amino acid
- Prior art date
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- 101710163270 Nuclease Proteins 0.000 title claims abstract 13
- 238000000034 method Methods 0.000 title claims abstract 5
- 239000000203 mixture Substances 0.000 title claims 7
- 230000001225 therapeutic effect Effects 0.000 title claims 2
- 208000037265 diseases, disorders, signs and symptoms Diseases 0.000 claims abstract 4
- 201000010099 disease Diseases 0.000 claims abstract 3
- 125000003275 alpha amino acid group Chemical group 0.000 claims 6
- 239000000539 dimer Substances 0.000 claims 4
- 201000000596 systemic lupus erythematosus Diseases 0.000 claims 4
- 208000023275 Autoimmune disease Diseases 0.000 claims 2
- 206010067584 Type 1 diabetes mellitus Diseases 0.000 claims 2
- 230000001363 autoimmune Effects 0.000 claims 2
- 239000003937 drug carrier Substances 0.000 claims 2
- 239000013604 expression vector Substances 0.000 claims 2
- 230000035772 mutation Effects 0.000 claims 2
- 150000007523 nucleic acids Chemical class 0.000 claims 2
- 102000039446 nucleic acids Human genes 0.000 claims 2
- 108020004707 nucleic acids Proteins 0.000 claims 2
- 239000002777 nucleoside Substances 0.000 claims 2
- 150000003833 nucleoside derivatives Chemical class 0.000 claims 2
- 238000003259 recombinant expression Methods 0.000 claims 2
- 206010039073 rheumatoid arthritis Diseases 0.000 claims 2
- 208000026872 Addison Disease Diseases 0.000 claims 1
- 208000004300 Atrophic Gastritis Diseases 0.000 claims 1
- 208000032116 Autoimmune Experimental Encephalomyelitis Diseases 0.000 claims 1
- 206010071155 Autoimmune arthritis Diseases 0.000 claims 1
- 208000008439 Biliary Liver Cirrhosis Diseases 0.000 claims 1
- 208000033222 Biliary cirrhosis primary Diseases 0.000 claims 1
- 206010009900 Colitis ulcerative Diseases 0.000 claims 1
- 206010063075 Cryptogenic cirrhosis Diseases 0.000 claims 1
- 208000036495 Gastritis atrophic Diseases 0.000 claims 1
- 208000024869 Goodpasture syndrome Diseases 0.000 claims 1
- 206010072579 Granulomatosis with polyangiitis Diseases 0.000 claims 1
- 208000030836 Hashimoto thyroiditis Diseases 0.000 claims 1
- 208000035186 Hemolytic Autoimmune Anemia Diseases 0.000 claims 1
- 206010020850 Hyperthyroidism Diseases 0.000 claims 1
- 208000007466 Male Infertility Diseases 0.000 claims 1
- 206010034277 Pemphigoid Diseases 0.000 claims 1
- 201000011152 Pemphigus Diseases 0.000 claims 1
- 208000002500 Primary Ovarian Insufficiency Diseases 0.000 claims 1
- 208000012654 Primary biliary cholangitis Diseases 0.000 claims 1
- 206010039710 Scleroderma Diseases 0.000 claims 1
- 208000021386 Sjogren Syndrome Diseases 0.000 claims 1
- 206010042742 Sympathetic ophthalmia Diseases 0.000 claims 1
- 201000006704 Ulcerative Colitis Diseases 0.000 claims 1
- 206010046851 Uveitis Diseases 0.000 claims 1
- 230000002159 abnormal effect Effects 0.000 claims 1
- 208000007502 anemia Diseases 0.000 claims 1
- 201000000448 autoimmune hemolytic anemia Diseases 0.000 claims 1
- 230000015556 catabolic process Effects 0.000 claims 1
- 208000016644 chronic atrophic gastritis Diseases 0.000 claims 1
- 230000001684 chronic effect Effects 0.000 claims 1
- 208000025302 chronic primary adrenal insufficiency Diseases 0.000 claims 1
- 208000018631 connective tissue disease Diseases 0.000 claims 1
- 230000003013 cytotoxicity Effects 0.000 claims 1
- 231100000135 cytotoxicity Toxicity 0.000 claims 1
- 238000006731 degradation reaction Methods 0.000 claims 1
- 201000001981 dermatomyositis Diseases 0.000 claims 1
- 208000012997 experimental autoimmune encephalomyelitis Diseases 0.000 claims 1
- 208000003816 familial cirrhosis Diseases 0.000 claims 1
- 239000000710 homodimer Substances 0.000 claims 1
- 230000028993 immune response Effects 0.000 claims 1
- 201000002364 leukopenia Diseases 0.000 claims 1
- 231100001022 leukopenia Toxicity 0.000 claims 1
- 238000012423 maintenance Methods 0.000 claims 1
- 238000004519 manufacturing process Methods 0.000 claims 1
- 201000006417 multiple sclerosis Diseases 0.000 claims 1
- 206010028417 myasthenia gravis Diseases 0.000 claims 1
- 201000001976 pemphigus vulgaris Diseases 0.000 claims 1
- 208000005987 polymyositis Diseases 0.000 claims 1
- 206010036601 premature menopause Diseases 0.000 claims 1
- 206010043778 thyroiditis Diseases 0.000 claims 1
- 208000005057 thyrotoxicosis Diseases 0.000 claims 1
- 208000035408 type 1 diabetes mellitus 1 Diseases 0.000 claims 1
- 241000124008 Mammalia Species 0.000 abstract 2
- 208000035475 disorder Diseases 0.000 abstract 1
- 239000008194 pharmaceutical composition Substances 0.000 abstract 1
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- C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
- C12N9/14—Hydrolases (3)
- C12N9/16—Hydrolases (3) acting on ester bonds (3.1)
- C12N9/22—Ribonucleases RNAses, DNAses
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- A61K47/68—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 the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent the modifying agent being an antibody, an immunoglobulin or a fragment thereof, e.g. an Fc-fragment
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- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07K—PEPTIDES
- C07K16/00—Immunoglobulins [IGs], e.g. monoclonal or polyclonal antibodies
- C07K16/44—Immunoglobulins [IGs], e.g. monoclonal or polyclonal antibodies against material not provided for elsewhere, e.g. haptens, metals, DNA, RNA, amino acids
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- C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
- C12N15/09—Recombinant DNA-technology
- C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
- C12N15/62—DNA sequences coding for fusion proteins
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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
- A61K38/46—Hydrolases (3)
- A61K38/465—Hydrolases (3) acting on ester bonds (3.1), e.g. lipases, ribonucleases
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- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07K—PEPTIDES
- C07K2319/00—Fusion polypeptide
- C07K2319/30—Non-immunoglobulin-derived peptide or protein having an immunoglobulin constant or Fc region, or a fragment thereof, attached thereto
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- C12Y—ENZYMES
- C12Y301/00—Hydrolases acting on ester bonds (3.1)
- C12Y301/27—Endoribonucleases producing 3'-phosphomonoesters (3.1.27)
- C12Y301/27005—Pancreatic ribonuclease (3.1.27.5)
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Claims (12)
- Terapevtski sestavki nukleaze in postopki Patentni zahtevki1. Molekula hibridne nukleaze, ki vsebuje humano Rnazo in mutirano domeno Gc humanega IgGl, kjer je humana Rnaza operativno spojena z domeno Fc in kjer domena Fc vsebuje mutacijo P238S in mutacijo P331S in ima zmanjšano citotoksičnost v primerjavi z molekulo hibridne nukleaze, ki ima nespremenjeno domeno Fc in kjer molekula hibridne nukleaze vsebuje: zaporedje amino kislin, prikazano v SEQ ID NO: 96, 92, 62 ali 78; ali zaporedje amino kislin, prikazano v SEQ ID NO: 98 ali 94.
- 2. Molekula hibridne nukleaze po zahtevku 1, ki vsebuje zaporedje amino kislin prikazano v SEQ ID NO: 96.
- 3. Molekula hibridne nukleaze po zahtevku 1, ki vsebuje zaporedje amino kislin prikazano v SEQ ID NO: 98.
- 4. Dimer, ki vsebuje molekulo hibridne nukleaze po kateremkoli zahtevku od 1 do 3, neobvezno homodimer.
- 5. Sestavek, ki vsebuje molekulo hibridne nukleaze po kateremkoli zahtevku od 1 do 3ali dimer po zahtevku 4 in farmacevtsko sprejemljiv nosilec.
- 6. Molekula nukleinske kisline, ki vsebuje molekulo hibridne nukleaze po kateremkoli zahtevku od 1 do 3.
- 7. Rekombinantni ekpresijski vektor, ki vsebuje vsebuje molekulo hibridne nukleaze po zahtevku 6.
- 8. Gostiteljska celica transformirana z rekombinantnim ekpresijskim vektorjem po zahtevku 7.
- 9. Postopek izdelave molekule hibridne nukleaze po kateremkoli zahtevku od 1 do 3, ki obsega: zagotavljanje gostiteljske celice, ki vsebuje zaporedje amino kislin, ki kodira molekulo hibridne nukleaze; in vzdrževanje gostiteljske celice pri pogojih v katerih je molekula hibridne nukleaze izražena.
- 10. Molekula hibridne nukleaze po kateremkoli zahtevku od 1 do 3, dimer po zahtevku 4 ali sestavek po zahtevku 5 za uporabo v postopku zdravljenja ali preprečevanja stanja poezanega z abnormalnim imunskim odgovorom, neobvezno, kjer je stanje avtoimuna bolezen, prednostno, kjer je avtoimuna bolezen izbrana iz skupine, ki vsebuje od inzulina odvisen diabetes mellitus, multiplo sklerozo, eksperimentalni avtoimunski encefalomielitis, revmatoidni artritis, eksperimentalni avtoimunski artritis, miastenijo gravis, tiroiditis, eksperimentalno obliko uveoretinitis, Hashimoto-jev tiroiditis, primarni miksedem, tirotoksikozo, pemiciozno anaemijo, avtoimuni atrofični gastritis, Addison-ovo bolezen, prezgodnjo menopavzo, moško neplodnost, juvenilni diabetes, Goodpasture-jev sindrom, pemfigus vulgaris, pemfigoid, simpatično oftalmijo, fakogeni uveitis, avtoimuno hemolitično anemijo, idiopatsko levkopenijo, primarno žolčno cirozo, aktivni kronični Hbs-ve, kriptogeno cirozo, ulcerozni kolitis, Sjogren-sov sindrom, sklerodermo, Wegener-jevo granulomatozo, polimiozitis, dermatomiozitis, diskoidni LE, sistemski lupus eritematozus (SLE) in bolezen veznega tkiva.
- 11. Molekula hibridne nukleaze, dimer ali sestavek za uporabo po zahtevku 10, kjer je avtoimuna bolezen SLE.
- 12. Sestavek, ki vsebuje nukleazo, učinkovit v razgradnji imunskih kompleksov, ki vsebujejo RNK, DNK ali obe RNK in DNK in, kjer sestavek vsebuje farmacevtsko sprejemljiv nosilec in molekulo hibridne nukleaze, ki vsebuje zaporedje amino kislin prikazano v SEQ ID NO: 96, 98, 92, 94, 62 ali 78, za uporabo v postopku zdravljenja SLE.
Applications Claiming Priority (4)
Application Number | Priority Date | Filing Date | Title |
---|---|---|---|
US201161480961P | 2011-04-29 | 2011-04-29 | |
US201261617241P | 2012-03-29 | 2012-03-29 | |
EP12777116.0A EP2704737B1 (en) | 2011-04-29 | 2012-04-27 | Therapeutic nuclease compositions and methods |
PCT/US2012/035614 WO2012149440A2 (en) | 2011-04-29 | 2012-04-27 | Therapeutic nuclease compositions and methods |
Publications (1)
Publication Number | Publication Date |
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SI2704737T1 true SI2704737T1 (sl) | 2018-06-29 |
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ID=47073103
Family Applications (1)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
SI201231276T SI2704737T1 (sl) | 2011-04-29 | 2012-04-27 | Terapevtski sestavki nukleaze in postopki |
Country Status (27)
Country | Link |
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US (5) | US10202588B2 (sl) |
EP (2) | EP2704737B1 (sl) |
JP (5) | JP6063450B2 (sl) |
KR (5) | KR102161657B1 (sl) |
CN (2) | CN103930127B (sl) |
AU (5) | AU2012249360B2 (sl) |
BR (1) | BR112013027547B1 (sl) |
CA (1) | CA2834626A1 (sl) |
CL (1) | CL2013003123A1 (sl) |
CO (1) | CO6811815A2 (sl) |
CR (1) | CR20130598A (sl) |
DK (1) | DK2704737T3 (sl) |
DO (1) | DOP2013000250A (sl) |
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