Optimization of an Optogenetic-Based Vision Therapy for Retinitis Pigmentosa

描述（由申请人提供）：Eos Neuroscience，Inc.，与其学术伙伴合作，正在开发一种结合基因治疗和光学工程技术的新技术，以恢复因视网膜色素变性或视网膜相关黄斑变性等感光细胞相关疾病而失明的个人的功能性视力。我们相信，这项技术将广泛适用于美国和世界各地的普通大众，他们患有这些使人衰弱和致盲的疾病。简而言之，Eos Neuroscience，Inc.正在创造一种技术，可以在光感受器死亡或不再起作用后恢复视网膜剩余细胞的光敏性，从而恢复视网膜对光刺激的反应能力。为此，我们创建了一个 使用腺相关病毒（AAV）的递送机制，其被证明在将基因递送到细胞中方面是安全和有效的。我们将利用这种机制将光敏蛋白表达到视网膜的备用功能细胞中。此外，我们正在完成剩余的必要的安全性和有效性研究，以获得临床前准备。在我们的第一阶段资助/运营期间，我们完成了在视网膜变性小鼠模型中评估生理和行为有效性指标的研究。在这里，我们将尝试通过采用更敏感的视蛋白来进一步改进我们的治疗载体，并通过生理和行为测量来评估这些视蛋白的功效。 公共卫生相关性：视网膜色素变性（RP）和年龄相关性黄斑变性（AMD）等感光性疾病是失明的主要原因，影响全球约1500万人。目前的疗法针对单一遗传缺陷或使用电刺激来恢复视觉功能-这两种疗法都不能广泛应用于感光细胞疾病。为此，Eos Neuroscience，Inc.已经建立了一种使用遗传表达的光敏蛋白来恢复患有光感受器变性的患者的光敏性的技术，该技术可以广泛且准确地应用于这些疾病的治疗。

DESCRIPTION (provided by applicant): Eos Neuroscience, Inc., in collaboration with its academic partners, is developing a novel technology combining gene therapeutics and optical engineering techniques to restore functional vision to individuals that are blind from photoreceptor related diseases such as Retinitis Pigmentosa or Age-Related Macular Degeneration. We believe that this technology will be widely applicable to the general public, in the United States and worldwide, which suffers from these debilitating and blinding diseases. In brief, Eos Neuroscience, Inc. is creating a technology that will restore photosensitivity in the remaining cells of the retina after the photoreceptors have died or are no longer functional, thus restoring the ability of the retina to respond to light stimulation. To this end, we have created a delivery mechanism using an adeno-associated virus (AAV) that is proven safe and effective at delivering genes into cells. We will use this mechanism to express light-sensitive proteins into the spared, functioning cells of the retina. Additionally, we are completing the remaining required safety and efficacy studies necessary to gain preclinical readiness. In the course of our Phase I funding/operating period, we completed studies to evaluate both physiological and behavioral efficacy measures in murine models of retinal degeneration. Here, we will attempt to further improve our therapeutic vector by employing more sensitive opsins and evaluate the efficacy of these opsins through physiological and behavioral measures. PUBLIC HEALTH RELEVANCE: Photoreceptor diseases such as retinitis pigmentosa (RP) and age-related macular degeneration (AMD) are leading causes of blindness, affecting approximately 15 million people worldwide. Current therapies are targeting single genetic defects or are using electrical stimulation to restore visual function - neither of which is capabl of being a treatment that can be applied broadly to photoreceptor disease. To this end, Eos Neuroscience, Inc. has established a technology using genetically expressed light sensitive proteins to restore light sensitivity in patients suffering from photoreceptor degeneration, a technology that can be applied broadly and accurately for the treatment of these diseases.