Optimization of an Optogenetic-Based Vision Therapy for Retinitis Pigmentosa

描述(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疗法和光学工程技术的新技术，以恢复因视网膜色素变性或老年性黄斑变性等与光感受器相关的疾病而失明的人的功能视力。我们相信，这项技术将广泛适用于患有这些衰弱和致盲疾病的美国和世界各地的普通公众。简而言之，Eos NeuroScience，Inc.正在创造一种技术，在感光器死亡或不再起作用后，将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)的传递机制，已被证明在将基因传递到细胞方面是安全和有效的。我们将使用这一机制将光敏蛋白表达到备用的、功能正常的视网膜细胞中。此外，我们正在完成其余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金/运营期间，我们完成了评估视网膜变性小鼠模型的生理和行为疗效测量的研究。在这里，我们将尝试通过使用更敏感的opsins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opsins的疗效。 公共卫生相关性：视网膜色素变性(RP)和老年性黄斑变性(AMD)等感光性疾病是导致失明的主要原因，全球约有1500万人受到影响。目前的治疗方法针对的是单一的基因缺陷或使用电刺激来恢复视觉功能--这两种方法都不能广泛应用于光感受器疾病的治疗。为此，Eos NeuroScience，Inc.建立了一项技术，使用基因表达的光敏蛋白来恢复光感受器变性患者的光敏感性，这项技术可以广泛而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.