ATE387217T1 - Mannose-6-phosphat rezeptor vermittelter gentransfer zu muskelzellen - Google Patents
Mannose-6-phosphat rezeptor vermittelter gentransfer zu muskelzellenInfo
- Publication number
- ATE387217T1 ATE387217T1 AT03077205T AT03077205T ATE387217T1 AT E387217 T1 ATE387217 T1 AT E387217T1 AT 03077205 T AT03077205 T AT 03077205T AT 03077205 T AT03077205 T AT 03077205T AT E387217 T1 ATE387217 T1 AT E387217T1
- Authority
- AT
- Austria
- Prior art keywords
- mannose
- muscle cells
- phosphate receptor
- gene transfer
- mediated gene
- Prior art date
Links
- 210000000663 muscle cell Anatomy 0.000 title abstract 3
- NBSCHQHZLSJFNQ-QTVWNMPRSA-N D-Mannose-6-phosphate Chemical compound OC1O[C@H](COP(O)(O)=O)[C@@H](O)[C@H](O)[C@@H]1O NBSCHQHZLSJFNQ-QTVWNMPRSA-N 0.000 title 1
- 108090000623 proteins and genes Proteins 0.000 title 1
- 206010013801 Duchenne Muscular Dystrophy Diseases 0.000 abstract 2
- 230000000692 anti-sense effect Effects 0.000 abstract 2
- 229930182470 glycoside Natural products 0.000 abstract 2
- 150000002338 glycosides Chemical class 0.000 abstract 2
- 102000001039 Dystrophin Human genes 0.000 abstract 1
- 108010069091 Dystrophin Proteins 0.000 abstract 1
- 108010031792 IGF Type 2 Receptor Proteins 0.000 abstract 1
- 102000019218 Mannose-6-phosphate receptors Human genes 0.000 abstract 1
- 108091034117 Oligonucleotide Proteins 0.000 abstract 1
- 239000000074 antisense oligonucleotide Substances 0.000 abstract 1
- 238000012230 antisense oligonucleotides Methods 0.000 abstract 1
- 230000015572 biosynthetic process Effects 0.000 abstract 1
- 210000004027 cell Anatomy 0.000 abstract 1
- 150000001875 compounds Chemical class 0.000 abstract 1
- 238000001727 in vivo Methods 0.000 abstract 1
- 239000003446 ligand Substances 0.000 abstract 1
- 238000003786 synthesis reaction Methods 0.000 abstract 1
Classifications
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K47/00—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
- A61K47/50—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
- A61K47/51—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
- A61K47/54—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 organic compound
Landscapes
- Health & Medical Sciences (AREA)
- Epidemiology (AREA)
- Animal Behavior & Ethology (AREA)
- Chemical & Material Sciences (AREA)
- Medicinal Chemistry (AREA)
- Pharmacology & Pharmacy (AREA)
- Engineering & Computer Science (AREA)
- Life Sciences & Earth Sciences (AREA)
- Bioinformatics & Cheminformatics (AREA)
- General Health & Medical Sciences (AREA)
- Public Health (AREA)
- Veterinary Medicine (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Polysaccharides And Polysaccharide Derivatives (AREA)
Applications Claiming Priority (1)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| EP03077205A EP1495769B1 (de) | 2003-07-11 | 2003-07-11 | Mannose-6-Phosphat Rezeptor vermittelter Gentransfer zu Muskelzellen |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| ATE387217T1 true ATE387217T1 (de) | 2008-03-15 |
Family
ID=33442814
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| AT03077205T ATE387217T1 (de) | 2003-07-11 | 2003-07-11 | Mannose-6-phosphat rezeptor vermittelter gentransfer zu muskelzellen |
Country Status (5)
| Country | Link |
|---|---|
| EP (1) | EP1495769B1 (de) |
| AT (1) | ATE387217T1 (de) |
| DE (1) | DE60319354T2 (de) |
| DK (1) | DK1495769T3 (de) |
| ES (1) | ES2302898T3 (de) |
Families Citing this family (27)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| EP2500430B1 (de) | 2004-06-28 | 2017-03-08 | The University Of Western Australia | Antisense-Oligonukleotide zur Induktion von Exon-Skipping sowie Verfahren zur Verwendung davon |
| GB0524987D0 (en) | 2005-12-08 | 2006-01-18 | Ge Healthcare Ltd | Novel imaging agents for fibrosis |
| WO2007123391A1 (en) | 2006-04-20 | 2007-11-01 | Academisch Ziekenhuis Leiden | Therapeutic intervention in a genetic disease in an individual by modifying expression of an aberrantly expressed gene. |
| EP1857548A1 (de) | 2006-05-19 | 2007-11-21 | Academisch Ziekenhuis Leiden | Mittel und Verfahren zur Induktion von Exon-skipping |
| WO2008018795A1 (en) | 2006-08-11 | 2008-02-14 | Prosensa Technologies B.V. | Methods and means for treating dna repeat instability associated genetic disorders |
| NZ582521A (en) | 2007-07-12 | 2011-09-30 | Prosensa Technologies Bv | A conjugate comprising the amino acid sequence LGAQSNF for targeting compounds to muscle tissue |
| WO2009008725A2 (en) | 2007-07-12 | 2009-01-15 | Prosensa Technologies B.V. | Molecules for targeting compounds to various selected organs, tissues or tumor cells |
| USRE48468E1 (en) | 2007-10-26 | 2021-03-16 | Biomarin Technologies B.V. | Means and methods for counteracting muscle disorders |
| WO2009054725A2 (en) | 2007-10-26 | 2009-04-30 | Academisch Ziekenhuis Leiden | Means and methods for counteracting muscle disorders |
| NZ587178A (en) | 2008-02-08 | 2011-11-25 | Prosensa Holding Bv | Methods and means for treating dna repeat instability associated genetic disorders |
| EP2119783A1 (de) | 2008-05-14 | 2009-11-18 | Prosensa Technologies B.V. | Verfahren für effizientes Exon (44)-Skipping bei Duchenne-Muskeldystrophie und entsprechende Mittel |
| KR20250016146A (ko) | 2008-10-24 | 2025-02-03 | 사렙타 쎄러퓨틱스 인코퍼레이티드 | Dmd를 위한 다중 엑손 스키핑 조성물 |
| AU2010239779A1 (en) | 2009-04-24 | 2011-11-17 | Prosensa Technologies B.V. | Oligonucleotide comprising an inosine for treating DMD |
| EP2448600B1 (de) | 2009-07-03 | 2016-03-30 | INSERM - Institut National de la Santé et de la Recherche Médicale | Auf den kationunabhängigen mannose 6-phosphat-rezeptor gerichtete verbindungen |
| KR102239374B1 (ko) | 2009-11-12 | 2021-04-14 | 더 유니버시티 오브 웨스턴 오스트레일리아 | 안티센스 분자 및 이를 이용한 질환 치료방법 |
| CA2785451C (en) | 2009-12-24 | 2019-01-22 | Prosensa Technologies B.V. | Molecule for treating an inflammatory disorder |
| EP4043039A1 (de) | 2012-01-27 | 2022-08-17 | BioMarin Technologies B.V. | Rna-modulierende oligonukleotide mit verbesserten eigenschaften zur behandlung der duchenne- und becker-muskeldystrophie |
| CN104918639B (zh) * | 2012-10-22 | 2018-01-26 | 萨拜格Rfa公司 | 用于将治疗剂递送到活细胞和细胞核中的系统 |
| HK1214297A1 (zh) | 2012-11-15 | 2016-07-22 | Roche Innovation Center Copenhagen A/S | 寡核苷酸缀合物 |
| WO2014118272A1 (en) | 2013-01-30 | 2014-08-07 | Santaris Pharma A/S | Antimir-122 oligonucleotide carbohydrate conjugates |
| RU2018108405A (ru) | 2013-01-30 | 2019-02-26 | Ф. Хоффманн-Ля Рош Аг | Конъюгаты углевода и lna-олигонуклеотида |
| CN105378081B (zh) | 2013-03-14 | 2019-06-14 | 萨勒普塔医疗公司 | 用于治疗肌营养不良的外显子跳跃组合物 |
| EP3662912B1 (de) | 2013-03-15 | 2025-12-03 | Sarepta Therapeutics, Inc. | Verbesserte eteplirsen dosierungen zur behandlung von duchenne muskeldystrophie |
| MX391977B (es) | 2013-06-27 | 2025-03-21 | Roche Innovation Ct Copenhagen As | Oligómeros antisentido y conjugados con diana en la proteína-convertasa subtilisina/kexina tipo 9(pcsk9). |
| GB201408623D0 (en) | 2014-05-15 | 2014-07-02 | Santaris Pharma As | Oligomers and oligomer conjugates |
| CN114717235A (zh) | 2016-03-14 | 2022-07-08 | 豪夫迈·罗氏有限公司 | 用于减少pd-l1表达的寡核苷酸 |
| JP7503072B2 (ja) | 2019-02-26 | 2024-06-19 | ロシュ イノベーション センター コペンハーゲン エーエス | オリゴヌクレオチドの製剤化方法 |
Family Cites Families (1)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US6172208B1 (en) * | 1992-07-06 | 2001-01-09 | Genzyme Corporation | Oligonucleotides modified with conjugate groups |
-
2003
- 2003-07-11 AT AT03077205T patent/ATE387217T1/de not_active IP Right Cessation
- 2003-07-11 DE DE60319354T patent/DE60319354T2/de not_active Expired - Lifetime
- 2003-07-11 EP EP03077205A patent/EP1495769B1/de not_active Expired - Lifetime
- 2003-07-11 ES ES03077205T patent/ES2302898T3/es not_active Expired - Lifetime
- 2003-07-11 DK DK03077205T patent/DK1495769T3/da active
Also Published As
| Publication number | Publication date |
|---|---|
| DE60319354D1 (de) | 2008-04-10 |
| DE60319354T2 (de) | 2009-03-26 |
| EP1495769B1 (de) | 2008-02-27 |
| DK1495769T3 (da) | 2008-06-23 |
| ES2302898T3 (es) | 2008-08-01 |
| EP1495769A1 (de) | 2005-01-12 |
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Legal Events
| Date | Code | Title | Description |
|---|---|---|---|
| RER | Ceased as to paragraph 5 lit. 3 law introducing patent treaties |