JP2020531047A5 - - Google Patents
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- JP2020531047A5 JP2020531047A5 JP2020530730A JP2020530730A JP2020531047A5 JP 2020531047 A5 JP2020531047 A5 JP 2020531047A5 JP 2020530730 A JP2020530730 A JP 2020530730A JP 2020530730 A JP2020530730 A JP 2020530730A JP 2020531047 A5 JP2020531047 A5 JP 2020531047A5
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- 102000004169 proteins and genes Human genes 0.000 claims description 33
- 108090000623 proteins and genes Proteins 0.000 claims description 33
- 239000013598 vector Substances 0.000 claims description 28
- 102000040430 polynucleotide Human genes 0.000 claims description 21
- 108091033319 polynucleotide Proteins 0.000 claims description 21
- 239000002157 polynucleotide Substances 0.000 claims description 21
- 208000009415 Spinocerebellar Ataxias Diseases 0.000 claims description 20
- 102100031650 C-X-C chemokine receptor type 4 Human genes 0.000 claims description 13
- 101000922348 Homo sapiens C-X-C chemokine receptor type 4 Proteins 0.000 claims description 13
- 239000012634 fragment Substances 0.000 claims description 13
- 230000004845 protein aggregation Effects 0.000 claims description 13
- 102000014461 Ataxins Human genes 0.000 claims description 12
- 108010078286 Ataxins Proteins 0.000 claims description 12
- 206010008025 Cerebellar ataxia Diseases 0.000 claims description 12
- 208000023105 Huntington disease Diseases 0.000 claims description 12
- 201000004562 autosomal dominant cerebellar ataxia Diseases 0.000 claims description 12
- 230000002265 prevention Effects 0.000 claims description 10
- 208000012902 Nervous system disease Diseases 0.000 claims description 9
- 208000025966 Neurological disease Diseases 0.000 claims description 9
- 230000004913 activation Effects 0.000 claims description 9
- 230000001668 ameliorated effect Effects 0.000 claims description 9
- 230000004065 mitochondrial dysfunction Effects 0.000 claims description 9
- 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 description 8
- 208000024827 Alzheimer disease Diseases 0.000 claims description 8
- 208000018737 Parkinson disease Diseases 0.000 claims description 8
- 206010002026 amyotrophic lateral sclerosis Diseases 0.000 claims description 8
- 201000010099 disease Diseases 0.000 claims description 7
- 208000037265 diseases, disorders, signs and symptoms Diseases 0.000 claims description 7
- 241000702421 Dependoparvovirus Species 0.000 claims description 6
- 102000001708 Protein Isoforms Human genes 0.000 claims description 6
- 108010029485 Protein Isoforms Proteins 0.000 claims description 6
- 208000028389 Nerve injury Diseases 0.000 claims description 5
- 230000008764 nerve damage Effects 0.000 claims description 5
- 102000007372 Ataxin-1 Human genes 0.000 claims description 4
- 108010032963 Ataxin-1 Proteins 0.000 claims description 4
- 201000010374 Down Syndrome Diseases 0.000 claims description 4
- 208000024412 Friedreich ataxia Diseases 0.000 claims description 4
- 206010058799 Mitochondrial encephalomyopathy Diseases 0.000 claims description 4
- 208000024777 Prion disease Diseases 0.000 claims description 4
- 206010044688 Trisomy 21 Diseases 0.000 claims description 4
- 201000010802 Wolfram syndrome Diseases 0.000 claims description 4
- 150000001413 amino acids Chemical group 0.000 claims description 4
- 239000003610 charcoal Substances 0.000 claims description 4
- 208000006443 lactic acidosis Diseases 0.000 claims description 4
- 208000020431 spinal cord injury Diseases 0.000 claims description 4
- 201000003624 spinocerebellar ataxia type 1 Diseases 0.000 claims description 4
- 208000011580 syndromic disease Diseases 0.000 claims description 4
- 241001164825 Adeno-associated virus - 8 Species 0.000 claims description 3
- AYFVYJQAPQTCCC-UHFFFAOYSA-N Threonine Natural products CC(O)C(N)C(O)=O AYFVYJQAPQTCCC-UHFFFAOYSA-N 0.000 claims description 3
- 239000004473 Threonine Substances 0.000 claims description 3
- 239000013603 viral vector Substances 0.000 claims description 3
- 241001655883 Adeno-associated virus - 1 Species 0.000 claims description 2
- 241000702423 Adeno-associated virus - 2 Species 0.000 claims description 2
- 241000202702 Adeno-associated virus - 3 Species 0.000 claims description 2
- 241000580270 Adeno-associated virus - 4 Species 0.000 claims description 2
- 241001634120 Adeno-associated virus - 5 Species 0.000 claims description 2
- 241000972680 Adeno-associated virus - 6 Species 0.000 claims description 2
- 241001164823 Adeno-associated virus - 7 Species 0.000 claims description 2
- 206010003591 Ataxia Diseases 0.000 claims description 2
- COLNVLDHVKWLRT-QMMMGPOBSA-N L-phenylalanine Chemical compound OC(=O)[C@@H](N)CC1=CC=CC=C1 COLNVLDHVKWLRT-QMMMGPOBSA-N 0.000 claims description 2
- 241000713666 Lentivirus Species 0.000 claims description 2
- 241000125945 Protoparvovirus Species 0.000 claims description 2
- KZSNJWFQEVHDMF-UHFFFAOYSA-N Valine Natural products CC(C)C(N)C(O)=O KZSNJWFQEVHDMF-UHFFFAOYSA-N 0.000 claims description 2
- COLNVLDHVKWLRT-UHFFFAOYSA-N phenylalanine Natural products OC(=O)C(N)CC1=CC=CC=C1 COLNVLDHVKWLRT-UHFFFAOYSA-N 0.000 claims description 2
- 241000701161 unidentified adenovirus Species 0.000 claims description 2
- 241001529453 unidentified herpesvirus Species 0.000 claims description 2
- 239000008194 pharmaceutical composition Substances 0.000 claims 20
- 230000003247 decreasing effect Effects 0.000 claims 4
- 230000000694 effects Effects 0.000 claims 4
- 230000004898 mitochondrial function Effects 0.000 claims 4
- 230000004071 biological effect Effects 0.000 claims 3
- KZSNJWFQEVHDMF-BYPYZUCNSA-N L-valine Chemical compound CC(C)[C@H](N)C(O)=O KZSNJWFQEVHDMF-BYPYZUCNSA-N 0.000 claims 1
- 230000004952 protein activity Effects 0.000 claims 1
- 239000004474 valine Substances 0.000 claims 1
- 238000000034 method Methods 0.000 description 6
- 102100021528 BPI fold-containing family B member 4 Human genes 0.000 description 5
- 101000899066 Homo sapiens BPI fold-containing family B member 4 Proteins 0.000 description 5
- 208000027418 Wounds and injury Diseases 0.000 description 4
- 230000006378 damage Effects 0.000 description 4
- 208000014674 injury Diseases 0.000 description 4
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| EP17186528 | 2017-08-16 | ||
| EP17186528.0 | 2017-08-16 | ||
| PCT/EP2018/072184 WO2019034723A1 (en) | 2017-08-16 | 2018-08-16 | VTFT ISOFORM OF BPIFB4 PROTEIN INTENDED FOR USE IN NEURONAL DISEASES AND INJURIES |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| JP2020531047A JP2020531047A (ja) | 2020-11-05 |
| JP2020531047A5 true JP2020531047A5 (https=) | 2021-09-24 |
Family
ID=59713793
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2020530730A Pending JP2020531047A (ja) | 2017-08-16 | 2018-08-16 | 神経疾患及び神経損傷における使用のためのｂｐｉｆｂ４タンパク質のｖｔｆｔアイソフォーム |
Country Status (8)
| Country | Link |
|---|---|
| US (1) | US12311009B2 (https=) |
| EP (1) | EP3668891B1 (https=) |
| JP (1) | JP2020531047A (https=) |
| CN (1) | CN111094327A (https=) |
| AU (1) | AU2018317807A1 (https=) |
| CA (1) | CA3109159A1 (https=) |
| ES (1) | ES2970198T3 (https=) |
| WO (1) | WO2019034723A1 (https=) |
Families Citing this family (1)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| EP4434534A1 (en) | 2023-03-22 | 2024-09-25 | ADvantage Therapeutics, Inc. | Klotho mrna |
Family Cites Families (39)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| GB9506197D0 (en) * | 1995-03-27 | 1995-05-17 | Hoffmann La Roche | Inhibition of tau-tau association. |
| AU2144999A (en) * | 1998-01-13 | 1999-08-02 | Claude Gravel | Viral vectors encoding neurofilament heavy proteins and their use |
| WO1999036560A2 (en) * | 1998-01-13 | 1999-07-22 | Julien Jean Pierre | Viral vectors encoding neurofilament light proteins and their use |
| US6472378B2 (en) * | 1998-08-31 | 2002-10-29 | Pro-Neuron, Inc. | Compositions and methods for treatment of mitochondrial diseases |
| AU2002313992B2 (en) * | 1998-08-31 | 2006-09-21 | Pro-Neuron, Inc. | Compositions and Methods for Treatment of Mitochondrial Diseases |
| AU6234700A (en) * | 1999-07-27 | 2001-02-13 | Abgenix, Inc. | Methods and compositions for inhibiting polypeptide accumulation associated with neurological disorders |
| US6924357B2 (en) * | 2000-04-18 | 2005-08-02 | Merck Patent Gmbh | Lipid binding protein 4 |
| US6515197B1 (en) * | 2000-08-24 | 2003-02-04 | Cedars-Sinai Medical Center | Transgenic mouse expressing a polynucleotide encoding a human ataxin-2 polypeptide |
| GB0100119D0 (en) * | 2001-01-03 | 2001-02-14 | Univ Aberdeen | Materials and methods relating to protein aggregation in neurodegenerative disease |
| JP2004532006A (ja) * | 2001-02-08 | 2004-10-21 | インサイト・ゲノミックス・インコーポレイテッド | 細胞内シグナル伝達分子 |
| WO2002090504A2 (en) * | 2001-05-03 | 2002-11-14 | Curagen Corporation | Novel antibodies that bind to antigenic polypeptides, nucleic acids encoding the antigens, and methods of use |
| WO2003004660A1 (en) * | 2001-07-03 | 2003-01-16 | Institut National De La Sante Et De La Recherche Medicale (Inserm) | Methods of administering vectors to synaptically connected neurons |
| AU2002359284A1 (en) | 2001-12-17 | 2003-06-30 | The Trustees Of The University Of Pennsylvania | Adeno-associated virus (aav) serotype 9 sequences, vectors containing same, and uses therefor |
| NZ533833A (en) * | 2001-12-21 | 2008-01-31 | Salk Inst For Biological Studi | Targeted retrograde gene delivery to motor neurons |
| US20040092715A1 (en) * | 2002-02-07 | 2004-05-13 | Li Ding | Intracellular signaling molecules |
| EP1489423A1 (en) * | 2003-06-20 | 2004-12-22 | Universite Louis Pasteur | Use of mitochondrial uncoupling proteins for diagnostic, prevention and treatment of diseases involving neuromuscular affection |
| WO2005095610A1 (en) * | 2004-03-31 | 2005-10-13 | Agt Biosciences Limited | Nucleic acid expressed in the hypothalamus or muscle tissue and uses thereof in diagnoses |
| WO2006022619A2 (en) * | 2004-07-22 | 2006-03-02 | Sequenom, Inc. | Methods for identifying risk of type ii diabetes and treatments thereof |
| CA2584986A1 (en) * | 2004-10-22 | 2006-05-04 | Neurologix, Inc. | Use of apoptosis inhibiting compounds in degenerative neurological disorders |
| PL1879623T3 (pl) * | 2005-05-02 | 2013-03-29 | Genzyme Corp | Terapia genowa zaburzeń rdzenia kręgowego |
| KR20080080109A (ko) * | 2005-11-04 | 2008-09-02 | 바이오겐 아이덱 엠에이 인코포레이티드 | 도파민성 뉴런의 신경돌기 성장 및 생존의 촉진 방법 |
| PL2066791T3 (pl) * | 2006-10-03 | 2013-02-28 | Genzyme Corp | Terapia Genowa do leczenia stwardnienia zanikowego bocznego i innych zaburzeń rdzenia kręgowego |
| EP2150117A4 (en) * | 2007-05-02 | 2011-10-05 | Mclean Hospital Corp | METHODS AND COMPOSITIONS FOR MITOCHONDRIA REPLACEMENT THERAPY |
| WO2009020624A1 (en) * | 2007-08-08 | 2009-02-12 | The Board Of Trustees Of The University Of Alabama For And On Behalf Of The University Of Alabama | Regulators of protein misfolding and neuroprotection and methods of use |
| MX2010010495A (es) * | 2008-04-03 | 2010-10-15 | Hoffmann La Roche | Uso de variantes del factor de crecimiento similar a la insulina (igf-i) pegiladas para el tratamiento de trastornos neuromusculares. |
| WO2010071832A1 (en) | 2008-12-19 | 2010-06-24 | Nationwide Children's Hospital | Delivery of polynucleotides across the blood brain barrier using recombinant aav9 |
| WO2010088729A1 (en) * | 2009-02-04 | 2010-08-12 | University Of Tasmania Through The Menzies Research Institute | Compositions and uses therefor |
| JP5879256B2 (ja) * | 2009-05-02 | 2016-03-08 | ジェンザイム・コーポレーション | 神経変性障害のための遺伝子治療 |
| CA2800375C (en) * | 2010-05-27 | 2021-03-09 | Atyr Pharma, Inc. | Innovative discovery of therapeutic, diagnostic, and antibody compositions related to protein fragments of glutaminyl-trna synthetases |
| US20140147434A1 (en) * | 2011-05-06 | 2014-05-29 | Ecole Polytechnique Federale De Lausanne (Epfl) | NCoR1 is a Physiological Modulator of Muscle Mass and Oxidative Function |
| US10815285B2 (en) * | 2011-07-01 | 2020-10-27 | University Of South Florida | Recombinant adeno-associated virus-mediated expression of fractalkine for treatment of neuroinflammatory and neurodegenerative diseases |
| JP6253596B2 (ja) * | 2012-02-16 | 2017-12-27 | ザ ペン ステイト リサーチ ファンデーション | アシル補酵素ａ：リゾカルジオリピン・アシル基転移酵素１（ａｌｃａｔ１）の発現、機能または活性の阻害物質を同定する方法 |
| EP2895621B1 (en) * | 2012-09-14 | 2020-10-21 | Population Bio, Inc. | Methods and compositions for diagnosing, prognosing, and treating neurological conditions |
| EP2749569A1 (en) * | 2012-12-28 | 2014-07-02 | Annibale Alessandro Puca | Variant of BPIFB4 protein |
| WO2014161095A1 (en) * | 2013-04-04 | 2014-10-09 | Crp40 Inc. | Crp40 fragments for the treatment of neurological disorders |
| AU2014329606B2 (en) * | 2013-10-01 | 2020-02-06 | Dana-Farber Cancer Institute, Inc. | Methods for the identification, assessment, prevention, and treatment of neurological disorders and diseases using Fndc5 |
| US20170129930A1 (en) * | 2014-06-19 | 2017-05-11 | Board Of Trustees Of The University Of Arkansas | Compositions and methods for modulating neuronal degeneration |
| US11027024B2 (en) * | 2015-05-29 | 2021-06-08 | University Of Iowa Research Foundation | Methods of delivery of transgenes for treating brain diseases |
| CA2973891A1 (en) * | 2016-07-21 | 2018-01-21 | Stealth Biotherapeutics Corp | Therapeutic compositions including modified creatine compounds and uses thereof to treat and prevent mitochondrial diseases and conditions |
-
2018
- 2018-08-16 EP EP18756412.5A patent/EP3668891B1/en active Active
- 2018-08-16 US US16/639,185 patent/US12311009B2/en active Active
- 2018-08-16 AU AU2018317807A patent/AU2018317807A1/en not_active Abandoned
- 2018-08-16 JP JP2020530730A patent/JP2020531047A/ja active Pending
- 2018-08-16 ES ES18756412T patent/ES2970198T3/es active Active
- 2018-08-16 WO PCT/EP2018/072184 patent/WO2019034723A1/en not_active Ceased
- 2018-08-16 CA CA3109159A patent/CA3109159A1/en active Pending
- 2018-08-16 CN CN201880052353.2A patent/CN111094327A/zh active Pending
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