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公开(公告)号:WO2019210097A1
公开(公告)日:2019-10-31
申请号:PCT/US2019/029199
申请日:2019-04-25
发明人: LEVY, Matthew , QUICK, Ryan , ERICKSON, Carl , RUSCONI, Christopher P. , HUTABARAT, Renta , MCLURE, Kevin G.
IPC分类号: A61K31/7115 , A61K31/712 , C12N15/115 , A61K48/00 , A61P27/02
摘要: This disclosure provides approaches and compositions for improving ocular therapeutics for pediatric and other applications. In general, the compositions and methods are designed to improve vitreous retention of ocular drugs, while simultaneously minimizing serum stability and associated systemic adverse events and retaining therapeutic efficacy. Also provided are aptamers with half-lives and potencies designed to maximize therapeutic efficacy and duration, while minimizing systemic side effects.
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公开(公告)号:WO2019222344A1
公开(公告)日:2019-11-21
申请号:PCT/US2019/032411
申请日:2019-05-15
发明人: ERICKSON, Carl , RUSCONI, Christopher P. , BHOWMICK, Arijit , LEVY, Matthew , WALKER, Matthew , MCLURE, Kevin G.
IPC分类号: C12N15/115 , A61K31/7088 , A61K31/7105 , A61K31/712
摘要: The application discloses methods and compositions for inhibiting functions associated with Interleukin-8 (IL8). The methods and compositions may involve the use of aptamers for binding to IL8 and preventing or reducing association of IL8 with CXCR1, CXCR2, or both. The methods and compositions may include one or more aptamers that bind to an N-terminal domain of IL8. The methods and compositions may include one or more aptamers that bind to a hydrophobic pocket of IL8. The methods and compositions may include one or more aptamers that bind to an N-loop of IL8. The methods and compositions may include one or more aptamers that bind to a GAG binding site of IL8. The application further provides anti-IL8 aptamers for the treatment of ocular diseases or disorders. In some cases, the anti-IL8 aptamers may have a stem-loop secondary structure.
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公开(公告)号:WO2017127762A1
公开(公告)日:2017-07-27
申请号:PCT/US2017/014459
申请日:2017-01-20
IPC分类号: C12N15/10 , C12N15/115
CPC分类号: C12N15/1048 , C12N15/111 , C12N2320/13
摘要: Methods are provided herein for generating and selecting aptamers. The aptamers can be suitable as therapeutic aptamers for the treatment of a disease or disorder. The methods provided may improve the efficiency and/or effectiveness of generating a therapeutic aptamer over traditional methods. The methods may generally involve selective pressure including competitive elution to generate aptamers that specifically bind to therapeutically-relevant epitopes and have a desired mechanism of action.
摘要翻译: 本文提供了用于产生和选择适体的方法。 适体可适合作为用于治疗疾病或病症的治疗适体。 所提供的方法可以提高产生治疗适体超过传统方法的效率和/或有效性。 所述方法通常可以包括选择性压力,包括竞争性洗脱以产生特异性结合治疗相关表位并具有期望的作用机制的适体。 p>
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公开(公告)号:WO2017127761A1
公开(公告)日:2017-07-27
申请号:PCT/US2017/014458
申请日:2017-01-20
IPC分类号: C12N15/115 , A61K31/7088
CPC分类号: C12N15/115 , A61K31/7105 , C12N2310/16
摘要: The application discloses methods and compositions for the inhibition of the alternative complement pathway. The methods and compositions involve the use of aptamers for inhibiting complement Factor D. The application further provides anti-Factor D aptamers for the treatment of dry age-related macular degeneration, geographic atrophy, wet age-related macular degeneration or Stargardt disease.
摘要翻译: 本申请公开了用于抑制替代补体途径的方法和组合物。 该方法和组合物涉及使用适体来抑制补体因子D.本申请还提供了用于治疗干性年龄相关性黄斑变性,地理萎缩,湿性年龄相关性黄斑变性或斯塔加特病的抗因子D适体。 p>
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公开(公告)号:WO2019210194A1
公开(公告)日:2019-10-31
申请号:PCT/US2019/029384
申请日:2019-04-26
IPC分类号: A61K47/60 , C12N15/115 , A61K31/712 , A61K31/7115 , A61P27/02
摘要: This disclosure provides PEGylated aptamers that contain PEG reagents with branching configurations useful for therapeutic applications, such as intraocular injection. This disclosure also provides methods for treating ocular disorders involving administering therapeutic PEGylated aptamers with branching configurations to a subject with an ocular disorder.
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公开(公告)号:WO2019022986A1
公开(公告)日:2019-01-31
申请号:PCT/US2018/042317
申请日:2018-07-16
IPC分类号: C12N15/115 , A61K31/7088 , A61P25/02
CPC分类号: A61P25/02 , A61K31/7088 , C12N15/115 , C12N2310/16 , C12N2310/317 , C12N2310/351 , C12N2310/322 , C12N2310/3533 , C12N2310/321 , C12N2310/3521 , A61K2300/00
摘要: The application discloses methods and compositions for the inhibition of the alternative complement pathway. The methods and compositions generally involve the use of aptamers for inhibiting complement Factor D. The application further provides anti-Factor D aptamers for the treatment of dry age-related macular degeneration, geographic atrophy, wet age-related macular degeneration, Stargardt disease and other ocular disorders or diseases. In some cases, anti-fD aptamer consensus sequences are provided.
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公开(公告)号:WO2017139417A1
公开(公告)日:2017-08-17
申请号:PCT/US2017/017066
申请日:2017-02-08
IPC分类号: C12N15/115 , A61K31/7088
CPC分类号: A61K31/7088 , A61K9/0048 , A61K31/7105 , A61K31/711 , A61K45/06 , A61K47/549 , C12N15/115 , C12N2310/16 , C12N2310/317 , C12N2310/351 , C12N2310/3519 , C12N2320/51 , A61K2300/00 , C12N2310/321 , C12N2310/3521 , C12N2310/322 , C12N2310/3533
摘要: Provided herein are compositions and methods for the treatment of retinal diseases. The compositions and methods include a therapeutic agent conjugated to a vitreous component binding moiety. The vitreous component binding moiety may be an aptamer or a small molecule that binds to a structural component of the vitreous humor (e.g., hyaluronic acid, collagen or vitronectin).
摘要翻译: 本文提供了用于治疗视网膜疾病的组合物和方法。 组合物和方法包括与玻璃体组分结合部分缀合的治疗剂。 玻璃质组分结合部分可以是结合玻璃体液的结构成分(例如透明质酸,胶原或玻连蛋白)的适体或小分子。 p>
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公开(公告)号:WO2018148333A1
公开(公告)日:2018-08-16
申请号:PCT/US2018/017296
申请日:2018-02-07
IPC分类号: C12N15/115 , A61K48/00 , A61P27/02
CPC分类号: A61P27/02 , A61K31/7088 , C12N15/115 , C12N2310/16 , C12N2310/317 , C12N2310/335 , C12N2310/51 , C12N2310/321 , C12N2310/3521 , C12N2310/322 , C12N2310/3533
摘要: Provided herein are compositions and methods for the treatment of retinal diseases. The compositions and methods include a therapeutic agent conjugated to a vitreous component binding moiety. The vitreous component binding moiety may be an aptamer or a small molecule that binds to a structural component of the vitreous humor (e.g., hyaluronic acid, collagen or vitronectin).
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公开(公告)号:WO2018136831A1
公开(公告)日:2018-07-26
申请号:PCT/US2018/014579
申请日:2018-01-19
IPC分类号: C12N15/115 , A61K31/7088
CPC分类号: C12N15/115 , A61K31/7088 , C12N2310/317 , C12N2310/321 , C12N2310/322 , C12N2310/3517 , C12N2310/3521 , C12N2310/3533
摘要: The application discloses methods and compositions for the inhibition of the alternative complement pathway. The methods and compositions involve the use of aptamers for inhibiting complement Factor D. The application further provides anti-Factor D aptamers for the treatment of dry age-related macular degeneration, geographic atrophy, wet age-related macular degeneration or Stargardt disease. In some cases, the compositions include aptamers having a pseudoknot secondary structure for the inhibition of fD.
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公开(公告)号:WO2018136827A1
公开(公告)日:2018-07-26
申请号:PCT/US2018/014573
申请日:2018-01-19
IPC分类号: C12N15/115 , A61K31/7088
摘要: The application discloses methods and compositions for the inhibition of the alternative complement pathway. The methods and compositions involve the use of aptamers for inhibiting complement Factor D. The application further provides anti-Factor D aptamers for the treatment of dry age-related macular degeneration, geographic atrophy, wet age-related macular degeneration or Stargardt disease. In some cases, stem-loop aptamers are provided for the inhibition of Factor D.
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