HRP20191608T1 - Proteini s ponavljajućim dipeptidom kao terapeutsko ciljno mjesto kod neurodegenerativnih bolesti s ekspanzijom ponavljajućeg heksanukleotida - Google Patents
Proteini s ponavljajućim dipeptidom kao terapeutsko ciljno mjesto kod neurodegenerativnih bolesti s ekspanzijom ponavljajućeg heksanukleotida Download PDFInfo
- Publication number
- HRP20191608T1 HRP20191608T1 HRP20191608T HRP20191608T1 HR P20191608 T1 HRP20191608 T1 HR P20191608T1 HR P20191608 T HRP20191608 T HR P20191608T HR P20191608 T1 HRP20191608 T1 HR P20191608T1
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- Prior art keywords
- seq
- accordance
- sequence
- heavy chain
- antigen
- Prior art date
Links
- 230000004770 neurodegeneration Effects 0.000 title 1
- 208000015122 neurodegenerative disease Diseases 0.000 title 1
- 102000004169 proteins and genes Human genes 0.000 title 1
- 108090000623 proteins and genes Proteins 0.000 title 1
- 230000001225 therapeutic effect Effects 0.000 title 1
- 239000000427 antigen Substances 0.000 claims 5
- 102000036639 antigens Human genes 0.000 claims 5
- 108091007433 antigens Proteins 0.000 claims 5
- 239000012634 fragment Substances 0.000 claims 5
- 101100112922 Candida albicans CDR3 gene Proteins 0.000 claims 4
- 102100035360 Cerebellar degeneration-related antigen 1 Human genes 0.000 claims 4
- 102100035361 Cerebellar degeneration-related protein 2 Human genes 0.000 claims 4
- 101000737793 Homo sapiens Cerebellar degeneration-related antigen 1 Proteins 0.000 claims 4
- 101000737796 Homo sapiens Cerebellar degeneration-related protein 2 Proteins 0.000 claims 4
- 201000011240 Frontotemporal dementia Diseases 0.000 claims 3
- 125000003275 alpha amino acid group Chemical group 0.000 claims 3
- 206010002026 amyotrophic lateral sclerosis Diseases 0.000 claims 3
- 201000010099 disease Diseases 0.000 claims 2
- 208000037265 diseases, disorders, signs and symptoms Diseases 0.000 claims 2
- 108010016626 Dipeptides Proteins 0.000 claims 1
- VPZXBVLAVMBEQI-VKHMYHEASA-N Glycyl-alanine Chemical compound OC(=O)[C@H](C)NC(=O)CN VPZXBVLAVMBEQI-VKHMYHEASA-N 0.000 claims 1
- 108010079364 N-glycylalanine Proteins 0.000 claims 1
- VPZXBVLAVMBEQI-UHFFFAOYSA-N glycyl-DL-alpha-alanine Natural products OC(=O)C(C)NC(=O)CN VPZXBVLAVMBEQI-UHFFFAOYSA-N 0.000 claims 1
- 238000000338 in vitro Methods 0.000 claims 1
- 102000039446 nucleic acids Human genes 0.000 claims 1
- 108020004707 nucleic acids Proteins 0.000 claims 1
- 150000007523 nucleic acids Chemical class 0.000 claims 1
- 239000000825 pharmaceutical preparation Substances 0.000 claims 1
- 229920001184 polypeptide Polymers 0.000 claims 1
- 108090000765 processed proteins & peptides Proteins 0.000 claims 1
- 102000004196 processed proteins & peptides Human genes 0.000 claims 1
Classifications
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- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07K—PEPTIDES
- C07K16/00—Immunoglobulins [IGs], e.g. monoclonal or polyclonal antibodies
- C07K16/18—Immunoglobulins [IGs], e.g. monoclonal or polyclonal antibodies against material from animals or humans
-
- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07K—PEPTIDES
- C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
- C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
- C07K14/46—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
- C07K14/47—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
-
- G—PHYSICS
- G01—MEASURING; TESTING
- G01N—INVESTIGATING OR ANALYSING MATERIALS BY DETERMINING THEIR CHEMICAL OR PHYSICAL PROPERTIES
- G01N33/00—Investigating or analysing materials by specific methods not covered by groups G01N1/00 - G01N31/00
- G01N33/48—Biological material, e.g. blood, urine; Haemocytometers
- G01N33/50—Chemical analysis of biological material, e.g. blood, urine; Testing involving biospecific ligand binding methods; Immunological testing
- G01N33/68—Chemical analysis of biological material, e.g. blood, urine; Testing involving biospecific ligand binding methods; Immunological testing involving proteins, peptides or amino acids
- G01N33/6893—Chemical analysis of biological material, e.g. blood, urine; Testing involving biospecific ligand binding methods; Immunological testing involving proteins, peptides or amino acids related to diseases not provided for elsewhere
-
- G—PHYSICS
- G01—MEASURING; TESTING
- G01N—INVESTIGATING OR ANALYSING MATERIALS BY DETERMINING THEIR CHEMICAL OR PHYSICAL PROPERTIES
- G01N33/00—Investigating or analysing materials by specific methods not covered by groups G01N1/00 - G01N31/00
- G01N33/48—Biological material, e.g. blood, urine; Haemocytometers
- G01N33/50—Chemical analysis of biological material, e.g. blood, urine; Testing involving biospecific ligand binding methods; Immunological testing
- G01N33/68—Chemical analysis of biological material, e.g. blood, urine; Testing involving biospecific ligand binding methods; Immunological testing involving proteins, peptides or amino acids
- G01N33/6893—Chemical analysis of biological material, e.g. blood, urine; Testing involving biospecific ligand binding methods; Immunological testing involving proteins, peptides or amino acids related to diseases not provided for elsewhere
- G01N33/6896—Neurological disorders, e.g. Alzheimer's disease
-
- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07K—PEPTIDES
- C07K2317/00—Immunoglobulins specific features
- C07K2317/20—Immunoglobulins specific features characterized by taxonomic origin
-
- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07K—PEPTIDES
- C07K2317/00—Immunoglobulins specific features
- C07K2317/70—Immunoglobulins specific features characterized by effect upon binding to a cell or to an antigen
- C07K2317/76—Antagonist effect on antigen, e.g. neutralization or inhibition of binding
-
- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07K—PEPTIDES
- C07K2317/00—Immunoglobulins specific features
- C07K2317/90—Immunoglobulins specific features characterized by (pharmaco)kinetic aspects or by stability of the immunoglobulin
- C07K2317/92—Affinity (KD), association rate (Ka), dissociation rate (Kd) or EC50 value
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Chemical & Material Sciences (AREA)
- Engineering & Computer Science (AREA)
- Molecular Biology (AREA)
- Biomedical Technology (AREA)
- Immunology (AREA)
- Hematology (AREA)
- Urology & Nephrology (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Biochemistry (AREA)
- Medicinal Chemistry (AREA)
- General Health & Medical Sciences (AREA)
- Organic Chemistry (AREA)
- Cell Biology (AREA)
- Pathology (AREA)
- Microbiology (AREA)
- Physics & Mathematics (AREA)
- Analytical Chemistry (AREA)
- Biotechnology (AREA)
- Food Science & Technology (AREA)
- General Physics & Mathematics (AREA)
- Biophysics (AREA)
- Genetics & Genomics (AREA)
- Neurosurgery (AREA)
- Neurology (AREA)
- Toxicology (AREA)
- Zoology (AREA)
- Gastroenterology & Hepatology (AREA)
- Peptides Or Proteins (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Measuring Or Testing Involving Enzymes Or Micro-Organisms (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
Claims (3)
1. Antitijelo ili njegov antigen-vezujući fragment koji se specifično vežu za polipeptid koji se sastoji od (Gly-Ala)a ponavljajućeg dipeptida, pri čemu je a cijeli broj od 16 ili više, pri čemu antitijelo ili njegov antigen-vezujući fragment sadrži CDR3 sekvencu lakog lanca koja sadrži aminokiselinsku sekvencu u skladu sa SEQ ID NO: 53; CDR2 sekvencu lakog lanca koja sadrži aminokiselinsku sekvencu u skladu sa SEQ ID NO: 54; i CDR1 sekvencu lakog lanca koja sadrži aminokiselinsku sekvencu u skladu sa SEQ ID NO: 55.
i
(i) CDR1 sekvencu teškog lanca koja sadrži GYTFTGYWIE u skladu sa SEQ ID NO: 68, CDR2 sekvencu teškog lanca koja sadrži EILPGSGSTK u skladu sa SEQ ID NO: 64 i CDR3 sekvencu teškog lanca koja sadrži GDFTNSHFAY u skladu sa SEQ ID NO: 60;
ili
(ii) CDR1 sekvencu teškog lanca koja sadrži GYTFTGYWIE u skladu sa SEQ ID NO: 68, CDR2 sekvencu teškog lanca koja sadrži ENLPGSGSTK u skladu sa SEQ ID NO: 65 i CDR3 sekvencu teškog lanca koja sadrži GDYSNSHFAY u skladu sa SEQ ID NO: 57;
ili
(iii) CDR1 sekvencu teškog lanca koja sadrži GYKFIGYWIE u skladu sa SEQ ID NO: 69, CDR2 sekvencu teškog lanca koja sadrži ENLPGSGTTK u skladu sa SEQ ID NO: 66 i CDR3 sekvencu teškog lanca koja sadrži GDYSNSHFTY u skladu sa SEQ ID NO: 59.
2. Farmaceutski preparat koji sadrži antitijelo ili njegov antigen-vezujući fragment u skladu s patentnim zahtjevom 1 i/ili nukleinsku kiselinu koja kodira navedeno antitijelo ili njegov antigen-vezujući fragment.
3. Uporaba antitijela ili njegovog antigen-vezujućeg fragmenta u skladu s patentnim zahtjevom 1 u in vitro dijagnostici bolesti koja je naznačena ekspanzijom genomskih GGGGCC ponovaka, pri čemu je bolest izabrana iz grupe koja se sastoji od amiotrofične lateralne skleroze (ALS), frontotemporalne demencije (FTD), i amiotrofične lateralne skleroze-frontotemporalne demencije (ALS-FTD).
Applications Claiming Priority (4)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| PCT/EP2013/000190 WO2014114303A1 (en) | 2013-01-22 | 2013-01-22 | Dipeptide-repeat proteins as therapeutic target in neurodegenerative diseases with hexanucleotide repeat expansion |
| EP13185419 | 2013-09-20 | ||
| EP14701089.6A EP2948777B1 (en) | 2013-01-22 | 2014-01-22 | Dipeptide-repeat proteins as therapeutic target in neurodegenerative diseases with hexanucleotide repeat expansion |
| PCT/EP2014/051204 WO2014114660A1 (en) | 2013-01-22 | 2014-01-22 | Dipeptide-repeat proteins as therapeutic target in neurodegenerative diseases with hexanucleotide repeat expansion |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| HRP20191608T1 true HRP20191608T1 (hr) | 2019-12-13 |
Family
ID=49999965
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| HRP20191608 HRP20191608T1 (hr) | 2013-01-22 | 2019-09-05 | Proteini s ponavljajućim dipeptidom kao terapeutsko ciljno mjesto kod neurodegenerativnih bolesti s ekspanzijom ponavljajućeg heksanukleotida |
Country Status (13)
| Country | Link |
|---|---|
| US (1) | US10066007B2 (hr) |
| EP (2) | EP3588091A1 (hr) |
| CY (1) | CY1122079T1 (hr) |
| DK (1) | DK2948777T3 (hr) |
| ES (1) | ES2744804T3 (hr) |
| HR (1) | HRP20191608T1 (hr) |
| HU (1) | HUE046434T2 (hr) |
| LT (1) | LT2948777T (hr) |
| PL (1) | PL2948777T3 (hr) |
| PT (1) | PT2948777T (hr) |
| RS (1) | RS59283B1 (hr) |
| SI (1) | SI2948777T1 (hr) |
| WO (1) | WO2014114660A1 (hr) |
Families Citing this family (23)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| ES2762326T5 (es) | 2012-10-15 | 2023-04-27 | Ionis Pharmaceuticals Inc | Métodos para modular la expresión de C9ORF72 |
| WO2014062736A1 (en) | 2012-10-15 | 2014-04-24 | Isis Pharmaceuticals, Inc. | Methods for monitoring c9orf72 expression |
| RU2020127664A (ru) | 2012-10-15 | 2020-09-17 | Ионис Фармасьютикалз, Инк. | Композиции для модуляции экспрессии гена c9orf72 |
| EP3588091A1 (en) * | 2013-01-22 | 2020-01-01 | Deutsches Zentrum für Neurodegenerative Erkrankungen e.V. (DZNE) | Dipeptide-repeat proteins as therapeutic target in neurodegenerative diseases with hexanucleotide repeat expansion |
| US9448232B2 (en) | 2013-01-24 | 2016-09-20 | Mayo Foundation For Medical Education And Research | Methods and materials for detecting C9ORF72 hexanucleotide repeat expansion positive frontotemporal lobar degeneration or C9ORF72 hexanucleotide repeat expansion positive amyotrophic lateral sclerosis |
| JP6628285B2 (ja) | 2013-03-14 | 2020-01-08 | ユニバーシティ オブ フロリダ リサーチ ファンデーション インコーポレーティッド | Ａｌｓ関連ジアミノ酸リピート含有タンパク質 |
| MY192689A (en) | 2013-10-11 | 2022-09-01 | Ionis Pharmaceuticals Inc | Compositions for modulating c9orf72 expression |
| KR20230104759A (ko) | 2014-09-30 | 2023-07-10 | 뉴리뮨 홀딩 아게 | 인간-유래의 항-디펩티드 반복체(dpr) 항체 |
| KR102258516B1 (ko) * | 2015-04-16 | 2021-05-31 | 아이오니스 파마수티컬즈, 인코포레이티드 | C9orf72 발현을 조절하기 위한 조성물 |
| US10509045B2 (en) | 2015-05-29 | 2019-12-17 | University Of Florida Research Foundation, Incorporated | Methods for diagnosing Huntington's disease |
| US11260073B2 (en) | 2015-11-02 | 2022-03-01 | Ionis Pharmaceuticals, Inc. | Compounds and methods for modulating C90RF72 |
| CA3019847A1 (en) | 2016-04-04 | 2017-10-12 | University Of Florida Research Foundation, Incorporated | Manipulation of eif3 to modulate repeat associated non-atg (ran) translation |
| WO2018195110A1 (en) | 2017-04-17 | 2018-10-25 | University Of Florida Research Foundation, Incorporated | Regolation of ran translation by pkr and eif2a-p pathways |
| JP6959632B2 (ja) * | 2017-05-15 | 2021-11-02 | 学校法人近畿大学 | 筋萎縮性側索硬化症又は前頭側頭型認知症の予防又は治療剤 |
| US11903910B2 (en) | 2017-09-26 | 2024-02-20 | University Of Florida Research Foundation, Incorporated | Use of metformin and analogs thereof to reduce RAN protein levels in the treatment of neurological disorders |
| CN108795935B (zh) * | 2018-05-23 | 2022-06-21 | 中国农业科学院上海兽医研究所(中国动物卫生与流行病学中心上海分中心) | 日本血吸虫SjELAV-like 1基因的siRNA及其应用 |
| CA3114657A1 (en) * | 2018-10-01 | 2020-04-09 | United Neuroscience | Peptide immunogen constructs directed against dipeptide repeat proteins from c9orf72 |
| WO2020221937A1 (en) * | 2019-05-02 | 2020-11-05 | Deutsches Zentrum Für Neurodegenerative Erkrankungen E.V. (Dzne) | Immunogen for preventing or treating familial frontotemporal dementia (ftd) and/or amyotrophic lateral sclerosis (als) |
| EP4031562A4 (en) * | 2019-09-20 | 2023-11-01 | University of Florida Research Foundation, Incorporated | DETECTION OF ANTIBODIES AGAINST RAN PROTEINS FROM SERUM AND TISSUE LYSATES |
| AU2020354387A1 (en) * | 2019-09-23 | 2022-04-07 | University Of Florida Research Foundation, Incorporated | Vaccine therapy for ran protein diseases |
| WO2023244542A1 (en) * | 2022-06-13 | 2023-12-21 | Yeefan Med Inc | Methods for detecting b-isox precipitates or captured proteins as biofluid biomarkers |
| WO2024097756A1 (en) * | 2022-11-01 | 2024-05-10 | University Of Florida Research Foundation, Incorporated | Interrupted ran proteins in disease |
| WO2024102988A1 (en) * | 2022-11-11 | 2024-05-16 | Mayo Foundation For Medical Education And Research | Treating proteinopathies |
Family Cites Families (8)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US20030143668A1 (en) | 2001-06-18 | 2003-07-31 | National Institute Of Advanced Industrial | Guanosine triphosphate-binding protein coupled receptors |
| US20040102608A1 (en) * | 2002-05-13 | 2004-05-27 | Cornell Research Foundation, Inc. | Multiblock copolymers having improved mechanical properties |
| EP1739167B1 (en) | 2004-04-19 | 2015-08-12 | DENKA SEIKEN Co., Ltd. | Method of producing virus |
| EP1997877B1 (en) * | 2006-03-17 | 2014-03-05 | Sanyo Chemical Industries, Ltd. | Cell culture substrate |
| WO2010115033A2 (en) | 2009-04-02 | 2010-10-07 | Regents Of The University Of Minnesota | Nucleotide repeat expansion-associated polypeptides and uses thereof |
| PL2571510T3 (pl) | 2010-05-21 | 2019-03-29 | Xl-Protein Gmbh | Biosyntetyczne polipeptydy prolinowo/alaninowe o strukturze kłębka statystycznego i ich zastosowania |
| RU2020127664A (ru) * | 2012-10-15 | 2020-09-17 | Ионис Фармасьютикалз, Инк. | Композиции для модуляции экспрессии гена c9orf72 |
| EP3588091A1 (en) * | 2013-01-22 | 2020-01-01 | Deutsches Zentrum für Neurodegenerative Erkrankungen e.V. (DZNE) | Dipeptide-repeat proteins as therapeutic target in neurodegenerative diseases with hexanucleotide repeat expansion |
-
2014
- 2014-01-22 EP EP19180100.0A patent/EP3588091A1/en active Pending
- 2014-01-22 WO PCT/EP2014/051204 patent/WO2014114660A1/en active Application Filing
- 2014-01-22 SI SI201431324T patent/SI2948777T1/sl unknown
- 2014-01-22 HU HUE14701089A patent/HUE046434T2/hu unknown
- 2014-01-22 EP EP14701089.6A patent/EP2948777B1/en active Active
- 2014-01-22 LT LTEP14701089.6T patent/LT2948777T/lt unknown
- 2014-01-22 PL PL14701089T patent/PL2948777T3/pl unknown
- 2014-01-22 ES ES14701089T patent/ES2744804T3/es active Active
- 2014-01-22 US US14/762,623 patent/US10066007B2/en active Active
- 2014-01-22 DK DK14701089.6T patent/DK2948777T3/da active
- 2014-01-22 RS RSP20191145 patent/RS59283B1/sr unknown
- 2014-01-22 PT PT14701089T patent/PT2948777T/pt unknown
-
2019
- 2019-09-05 CY CY20191100935T patent/CY1122079T1/el unknown
- 2019-09-05 HR HRP20191608 patent/HRP20191608T1/hr unknown
Also Published As
| Publication number | Publication date |
|---|---|
| WO2014114660A1 (en) | 2014-07-31 |
| ES2744804T3 (es) | 2020-02-26 |
| DK2948777T3 (da) | 2019-09-23 |
| EP2948777B1 (en) | 2019-06-26 |
| RS59283B1 (sr) | 2019-10-31 |
| EP2948777A1 (en) | 2015-12-02 |
| US20150361166A1 (en) | 2015-12-17 |
| US10066007B2 (en) | 2018-09-04 |
| HUE046434T2 (hu) | 2020-03-30 |
| PL2948777T3 (pl) | 2019-12-31 |
| EP3588091A1 (en) | 2020-01-01 |
| SI2948777T1 (sl) | 2019-11-29 |
| PT2948777T (pt) | 2019-09-26 |
| LT2948777T (lt) | 2019-09-25 |
| CY1122079T1 (el) | 2020-11-25 |
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