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Targeting SDF-1 for effective wet AMD treatment

靶向 SDF-1 进行有效的湿性 AMD 治疗

基本信息

批准号：
9408583
负责人：
Qiang Gong
金额：
$ 25.01万
依托单位：
APTITUDE MEDICAL SYSTEMS, INC.
依托单位国家：
美国
项目类别：
财政年份：
2017
资助国家：
美国
起止时间：
2017-09-30 至 2018-09-29
项目状态：
已结题

来源：
https://reporter.nih.gov/project-details/9408583
关键词：
Accounting Adverse effects Affinity Age related macular degeneration Animal Model Animal Testing Aptitude Avastin Binding Blindness Chemicals Choroidal Neovascularization Chronic Clinic Combined Modality Therapy Complement Complex DNA Development Disease Drops Elderly Formulation Future Goals Half-Life Head Immunologics Injection of therapeutic agent KDR gene Label Laser injury Lasers Lead Legal Licensing Lucentis Maintenance Market Research Mediating Methods Modeling Modification Molecular Target Monoclonal Antibodies Mus Nature New Agents Nucleotides Oligonucleotides Ophthalmology Pathologic Processes Patients Performance Pharmaceutical Preparations Phase Platelet-Derived Growth Factor Play Prevalence Procedures RNA Recruitment Activity Role Safety Signal Transduction Small Business Innovation Research Grant Stromal Cell-Derived Factor 1 Testing Therapeutic Tissues Treatment Factor VEGFA gene Validation Vascular Endothelial Growth Factors Vision alternative treatment angiogenesis aptamer base chemical synthesis cost efficacy testing experience high standard improved in vivo intravitreal injection mouse model new technology novel nuclease ocular neovascularization particle pre-clinical pre-clinical research preclinical study precursor cell receptor binding resistance mechanism standard care standard of care

项目摘要

PROJECT ABSTRACT Wet Age-related Macular Degeneration (AMD) is the leading cause of new blindness among people who are 55 or older. The current standard of care for wet AMD is anti-Vascular Endothelial Growth Factor (VEGF) agents, such as Lucentis, Eylea, and Avastin (used off-label). These anti-VEGF agents inhibit VEGF-mediated angiogenesis, and effectively stop or even reverse the vision loss for most patients. Despite being the best treatment currently available, it has two critical shortcomings: 1) limited near-term vision gain and poor long- term vision maintenance; (2) major cost and treatment burden. First, despite continued treatment, only ~1/3 of the patient gain >3 lines of vision over the first 12 months; after 3-4 years, most patients start losing vision again and eventually dropping to pre-treatment levels. Second, patients must return to the clinic every 1 to 2 months for intravitreal injections – a specialized, uncomfortable and costly procedure that represents significant treatment burden. The purpose of this SBIR is to develop highly stable aptamers against SDF-1, which may serve as an orthogonal treatment for wet AMD. SDF-1 has long been known to play an important role in choroidal neovascularization (CNV), the landmark pathological process of wet AMD. The Aptitude team has accumulated extensive experience in aptamer discovery. We have previously developed the Particle Display method that significantly improves the aptamer performance. Moreover, we have made further improvement to directly screen for fully modified aptamers that enables longer duration of efficacy. Our expertise in aptamer discovery is complemented by our collaborators’ expertise in wet AMD preclinical research. Our collaborator is among the first to demonstrate the role of SDF-1 in CNV, and possess in-depth expertise in relevant animal models. If successful, this project has the potential of bringing more efficacious and affordable treatment to wet AMD patients.

项目摘要