Istituto di Bioscienze e Biorisorse, National Research Council (CNR) , Napoli , Italy.
Institut des Biomolécules Max Mousseron (IBMM), UMR 5247 CNRS, ENSCM, Université de Montpellier , Montpellier , France.
Expert Opin Ther Pat. 2019 Oct;29(10):749-752. doi: 10.1080/13543776.2019.1661991. Epub 2019 Sep 1.
: Age-related macular degeneration (AMD) is the leading cause of central vision loss in developed countries. Effective therapy for AMD is currently limited to the treatment of the patient with intravitreal injections of anti-VEGF drugs. : A method for the management of age-related macular degeneration (AMD) is claimed. It consists of the administration of pure botulinum-toxin or a serogroup of recombinant botulinum-toxins or their peptide fragments or neurotoxin associated with accessory proteins in extra-ocular regions of the eye. The toxin modifies the retinal pigment epithelium, maintaining its structure and function, and delaying the various stages of the macular degeneration. : The botulinum-toxin (BT) is administrated as extra-ocular infusions avoiding the risk of direct intraocular injection and the complications associated with the patients. The possibility to administer BT with accessory proteins (hemagglutinin, monoclonal antibody (anti-VEGF)), makes the novel system interesting for developing combined approaches for AMD treatment. The use of BT (alone or conjugated to other agents) as a method for treating or preventing AMD requires necessarily a patient case report study, as well as the or data, for supporting all the claims of the present patent.
年龄相关性黄斑变性（AMD）是发达国家导致中心视力丧失的主要原因。目前，AMD 的有效治疗方法仅限于玻璃体内注射抗血管内皮生长因子（VEGF）药物。本专利要求提供一种年龄相关性黄斑变性（AMD）的治疗方法。该方法包括在眼部的眼外区域给予纯肉毒毒素或重组肉毒毒素血清型或其肽片段或与辅助蛋白相关的神经毒素。该毒素可修饰视网膜色素上皮，维持其结构和功能，并延缓黄斑变性的各个阶段。肉毒毒素（BT）作为眼外滴注给药，避免了直接眼内注射的风险和与患者相关的并发症。BT 与辅助蛋白（血凝素、单克隆抗体（抗 VEGF））联合给药，使得这种新型系统对于开发 AMD 治疗的联合方法具有吸引力。BT（单独使用或与其他药物结合使用）作为治疗或预防 AMD 的方法的使用需要对患者进行病例报告研究，以及支持本专利所有权利要求的临床或 数据。
