JP2015529685A5 - - Google Patents
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- JP2015529685A5 JP2015529685A5 JP2015532145A JP2015532145A JP2015529685A5 JP 2015529685 A5 JP2015529685 A5 JP 2015529685A5 JP 2015532145 A JP2015532145 A JP 2015532145A JP 2015532145 A JP2015532145 A JP 2015532145A JP 2015529685 A5 JP2015529685 A5 JP 2015529685A5
- Authority
- JP
- Japan
- Prior art keywords
- pharmaceutical composition
- composition according
- nucleic acid
- compound
- cytoplasmic
- 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.)
- Pending
Links
- 239000008194 pharmaceutical composition Substances 0.000 claims 20
- 102000039446 nucleic acids Human genes 0.000 claims 9
- 108020004707 nucleic acids Proteins 0.000 claims 9
- 150000007523 nucleic acids Chemical class 0.000 claims 9
- 150000001875 compounds Chemical class 0.000 claims 8
- 210000002161 motor neuron Anatomy 0.000 claims 6
- 230000001086 cytosolic effect Effects 0.000 claims 5
- 239000008187 granular material Substances 0.000 claims 5
- 239000013598 vector Substances 0.000 claims 4
- 102000001398 Granzyme Human genes 0.000 claims 3
- 108060005986 Granzyme Proteins 0.000 claims 3
- 102000043129 MHC class I family Human genes 0.000 claims 3
- 108091054437 MHC class I family Proteins 0.000 claims 3
- 102000004503 Perforin Human genes 0.000 claims 3
- 108010056995 Perforin Proteins 0.000 claims 3
- 206010002026 amyotrophic lateral sclerosis Diseases 0.000 claims 3
- 231100000331 toxic Toxicity 0.000 claims 3
- 230000002588 toxic effect Effects 0.000 claims 3
- 206010003694 Atrophy Diseases 0.000 claims 2
- KHGNFPUMBJSZSM-UHFFFAOYSA-N Perforine Natural products COC1=C2CCC(O)C(CCC(C)(C)O)(OC)C2=NC2=C1C=CO2 KHGNFPUMBJSZSM-UHFFFAOYSA-N 0.000 claims 2
- 210000001130 astrocyte Anatomy 0.000 claims 2
- 230000037444 atrophy Effects 0.000 claims 2
- 210000005056 cell body Anatomy 0.000 claims 2
- 239000003814 drug Substances 0.000 claims 2
- 229940079593 drug Drugs 0.000 claims 2
- 230000000694 effects Effects 0.000 claims 2
- 210000002241 neurite Anatomy 0.000 claims 2
- 229930192851 perforin Natural products 0.000 claims 2
- 102000004196 processed proteins & peptides Human genes 0.000 claims 2
- 108090000765 processed proteins & peptides Proteins 0.000 claims 2
- 230000001629 suppression Effects 0.000 claims 2
- 231100000419 toxicity Toxicity 0.000 claims 2
- 230000001988 toxicity Effects 0.000 claims 2
- 239000013603 viral vector Substances 0.000 claims 2
- 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 1
- 241000702421 Dependoparvovirus Species 0.000 claims 1
- 108700018351 Major Histocompatibility Complex Proteins 0.000 claims 1
- 201000008257 amyotrophic lateral sclerosis type 1 Diseases 0.000 claims 1
- 230000001580 bacterial effect Effects 0.000 claims 1
- 230000003247 decreasing effect Effects 0.000 claims 1
- 208000019929 sporadic amyotrophic lateral sclerosis Diseases 0.000 claims 1
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201261702030P | 2012-09-17 | 2012-09-17 | |
| US61/702,030 | 2012-09-17 | ||
| PCT/US2013/060153 WO2014043696A2 (en) | 2012-09-17 | 2013-09-17 | Compositions and methods for treating amyotrophic lateral sclerosis |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| JP2015529685A JP2015529685A (ja) | 2015-10-08 |
| JP2015529685A5 true JP2015529685A5 (OSRAM) | 2017-01-12 |
Family
ID=50278875
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2015532145A Pending JP2015529685A (ja) | 2012-09-17 | 2013-09-17 | 筋萎縮性側索硬化症の処置のための組成物および方法 |
Country Status (6)
| Country | Link |
|---|---|
| US (1) | US9539307B2 (OSRAM) |
| EP (1) | EP2895606A4 (OSRAM) |
| JP (1) | JP2015529685A (OSRAM) |
| AU (1) | AU2013315007A1 (OSRAM) |
| CA (1) | CA2885216A1 (OSRAM) |
| WO (1) | WO2014043696A2 (OSRAM) |
Families Citing this family (16)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US9597379B1 (en) | 2010-02-09 | 2017-03-21 | David Gordon Bermudes | Protease inhibitor combination with therapeutic proteins including antibodies |
| US8524220B1 (en) | 2010-02-09 | 2013-09-03 | David Gordon Bermudes | Protease inhibitor: protease sensitivity expression system composition and methods improving the therapeutic activity and specificity of proteins delivered by bacteria |
| US8771669B1 (en) | 2010-02-09 | 2014-07-08 | David Gordon Bermudes | Immunization and/or treatment of parasites and infectious agents by live bacteria |
| US9737592B1 (en) | 2014-02-14 | 2017-08-22 | David Gordon Bermudes | Topical and orally administered protease inhibitors and bacterial vectors for the treatment of disorders and methods of treatment |
| KR102584655B1 (ko) | 2014-11-14 | 2023-10-06 | 보이저 테라퓨틱스, 인크. | 조절성 폴리뉴클레오티드 |
| MX2017006216A (es) | 2014-11-14 | 2018-08-29 | Voyager Therapeutics Inc | Composiciones y métodos para tratar la esclerosis lateral amiotrófica (ela). |
| CA2975101A1 (en) * | 2015-01-26 | 2016-08-04 | The Research Institute At Nationwide Children's Hospital | Compounds, compositions, and methods for using hla-f |
| US10676723B2 (en) | 2015-05-11 | 2020-06-09 | David Gordon Bermudes | Chimeric protein toxins for expression by therapeutic bacteria |
| EP3436572A4 (en) * | 2016-03-31 | 2019-11-20 | Lion TCR Pte. Ltd. | EXOGENOUS VIRUS-SPECIFIC T-CELL RECEPTOR (TCR) EXPRESSIVE UNACTIVATED T-CELLS |
| RU2758488C2 (ru) | 2016-05-18 | 2021-10-28 | Вояджер Терапьютикс, Инк. | Модулирующие полинуклеотиды |
| US11180535B1 (en) | 2016-12-07 | 2021-11-23 | David Gordon Bermudes | Saccharide binding, tumor penetration, and cytotoxic antitumor chimeric peptides from therapeutic bacteria |
| US11129906B1 (en) | 2016-12-07 | 2021-09-28 | David Gordon Bermudes | Chimeric protein toxins for expression by therapeutic bacteria |
| AU2018261790B2 (en) | 2017-05-05 | 2024-10-03 | Voyager Therapeutics, Inc. | Compositions and methods of treating amyotrophic lateral sclerosis (ALS) |
| US11434502B2 (en) | 2017-10-16 | 2022-09-06 | Voyager Therapeutics, Inc. | Treatment of amyotrophic lateral sclerosis (ALS) |
| EP4454654A3 (en) | 2017-10-16 | 2025-02-19 | Voyager Therapeutics, Inc. | Treatment of amyotrophic lateral sclerosis (als) |
| US12285437B2 (en) | 2019-10-30 | 2025-04-29 | The Research Foundation For The State University Of New York | Reversing the undesirable pH-profile of doxorubicin via activation of a disubstituted maleamic acid prodrug at tumor acidity |
Family Cites Families (38)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US5173414A (en) | 1990-10-30 | 1992-12-22 | Applied Immune Sciences, Inc. | Production of recombinant adeno-associated virus vectors |
| EP0733103B1 (en) | 1993-11-09 | 2004-03-03 | Targeted Genetics Corporation | Generation of high titers of recombinant aav vectors |
| ATE272123T1 (de) | 1993-11-09 | 2004-08-15 | Ohio Med College | Stabile zellinie, die in der lage ist, das replikationsgen des adenoassoziertenvirus zu exprimieren |
| US5658785A (en) | 1994-06-06 | 1997-08-19 | Children's Hospital, Inc. | Adeno-associated virus materials and methods |
| US5856152A (en) | 1994-10-28 | 1999-01-05 | The Trustees Of The University Of Pennsylvania | Hybrid adenovirus-AAV vector and methods of use therefor |
| EP0796339A1 (en) | 1994-12-06 | 1997-09-24 | Targeted Genetics Corporation | Packaging cell lines for generation of high titers of recombinant aav vectors |
| FR2737730B1 (fr) | 1995-08-10 | 1997-09-05 | Pasteur Merieux Serums Vacc | Procede de purification de virus par chromatographie |
| JPH11511326A (ja) | 1995-08-30 | 1999-10-05 | ジエンザイム コーポレイション | アデノウィルスおよびａａｖの精製 |
| US6632670B1 (en) | 1995-09-08 | 2003-10-14 | Genzyme Corporation | AAV vectors for gene therapy |
| US5910434A (en) | 1995-12-15 | 1999-06-08 | Systemix, Inc. | Method for obtaining retroviral packaging cell lines producing high transducing efficiency retroviral supernatant |
| ATE465267T1 (de) | 1996-09-06 | 2010-05-15 | Univ Pennsylvania | Rekombinante aav zur herstellung eines medikaments für die gentherapie von muskelzellen |
| FR2753379B1 (fr) | 1996-09-13 | 1998-10-30 | Rhone Poulenc Rorer Sa | Methode de traitement de la sclerose laterale amyotrophique |
| CN1181298A (zh) | 1996-10-28 | 1998-05-13 | 赵幼仪 | 开关型程控快速活扳手 |
| US6566118B1 (en) | 1997-09-05 | 2003-05-20 | Targeted Genetics Corporation | Methods for generating high titer helper-free preparations of released recombinant AAV vectors |
| WO1999011764A2 (en) | 1997-09-05 | 1999-03-11 | Targeted Genetics Corporation | Methods for generating high titer helper-free preparations of recombinant aav vectors |
| US6258595B1 (en) | 1999-03-18 | 2001-07-10 | The Trustees Of The University Of Pennsylvania | Compositions and methods for helper-free production of recombinant adeno-associated viruses |
| CA2406743A1 (en) | 2000-04-28 | 2001-11-08 | The Trustees Of The University Of Pennsylvania | Recombinant aav vectors with aav5 capsids and aav5 vectors pseudotyped in heterologous capsids |
| BR0113099A (pt) | 2000-08-07 | 2004-07-06 | Genset Sa | Gene e proteìna relacionados com esquizofrenia |
| US6905827B2 (en) | 2001-06-08 | 2005-06-14 | Expression Diagnostics, Inc. | Methods and compositions for diagnosing or monitoring auto immune and chronic inflammatory diseases |
| US20050019915A1 (en) | 2001-06-21 | 2005-01-27 | Bennett C. Frank | Antisense modulation of superoxide dismutase 1, soluble expression |
| US9233131B2 (en) | 2003-06-30 | 2016-01-12 | The Regents Of The University Of California | Mutant adeno-associated virus virions and methods of use thereof |
| US9441244B2 (en) | 2003-06-30 | 2016-09-13 | The Regents Of The University Of California | Mutant adeno-associated virus virions and methods of use thereof |
| WO2005096781A2 (en) | 2004-04-06 | 2005-10-20 | University Of Massachusetts | Methods and compositions for treating gain-of-function disorders using rna interference |
| WO2006066203A2 (en) | 2004-12-16 | 2006-06-22 | Alsgen, Llc | Small interfering rna (sirna) molecules for modulating superoxide dismutase (sod) |
| DE102006019480A1 (de) * | 2006-04-26 | 2007-10-31 | Sirs-Lab Gmbh | Verfahren zur in-vitro Überwachung postoperativer Veränderungen nach Lebertransplantation |
| EP2019143A1 (en) | 2007-07-23 | 2009-01-28 | Genethon | CNS gene delivery using peripheral administration of AAV vectors |
| EP2058401A1 (en) | 2007-10-05 | 2009-05-13 | Genethon | Widespread gene delivery to motor neurons using peripheral injection of AAV vectors |
| EP3643782A1 (en) | 2008-02-11 | 2020-04-29 | Phio Pharmaceuticals Corp. | Modified rnai polynucleotides and uses thereof |
| WO2009114724A2 (en) | 2008-03-12 | 2009-09-17 | Intradigm Corporation | COMPOSITIONS COMPRISING NUCLEAR FACTOR-KAPPA B (NF-ĸB) SIRNA AND METHODS OF USE |
| WO2010071832A1 (en) | 2008-12-19 | 2010-06-24 | Nationwide Children's Hospital | Delivery of polynucleotides across the blood brain barrier using recombinant aav9 |
| EP3514232A1 (en) | 2010-04-23 | 2019-07-24 | University of Massachusetts | Cns targeting aav vectors and methods of use thereof |
| KR101235256B1 (ko) * | 2010-09-13 | 2013-02-21 | 서울대학교산학협력단 | ｍｉＲＮＡ를 타겟으로 한 신경퇴행성 질환 치료 |
| US9109037B2 (en) | 2010-10-26 | 2015-08-18 | University Of Massachusetts | Anti-SOD1 antibodies and uses thereof |
| US20130344081A1 (en) | 2011-03-07 | 2013-12-26 | The University Of Tokushima | Method of treating amyotrophic lateral sclerosis |
| WO2013078316A1 (en) | 2011-11-23 | 2013-05-30 | Nationwide Children's Hospital, Inc. | Recombinant adeno-associated virus delivery of alpha-sarcoglycan polynucleotides |
| BR112014020325A2 (pt) | 2012-02-17 | 2017-08-08 | Childrens Hospital Philadelphia | composições de vetor do aav e métodos para a transferência de gene para as células, órgãos e tecidos |
| CN104685674B (zh) | 2012-09-28 | 2019-03-26 | 卡博特公司 | 包含高表面积含碳材料的活性材料组合物 |
| BR122019025681B1 (pt) | 2012-11-01 | 2023-04-18 | Factor Bioscience Inc | Método para inserir uma sequência de ácido nucleico em uma localização segura de um genoma de uma célula |
-
2013
- 2013-09-17 EP EP13837504.3A patent/EP2895606A4/en not_active Withdrawn
- 2013-09-17 JP JP2015532145A patent/JP2015529685A/ja active Pending
- 2013-09-17 AU AU2013315007A patent/AU2013315007A1/en not_active Abandoned
- 2013-09-17 CA CA2885216A patent/CA2885216A1/en not_active Abandoned
- 2013-09-17 US US14/428,765 patent/US9539307B2/en active Active
- 2013-09-17 WO PCT/US2013/060153 patent/WO2014043696A2/en not_active Ceased
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