Spinal muscular atrophy (SMA) is an inherited neuromuscular disorder characterized by the increasing weakness and atrophy in muscles affecting movement, speaking, swallowing and breathing. SMA is caused by missing or mutated SMN1 gene encoding SMN protein which is necessary to maintain the survival and proper function of motor neurons. SMA is a rare disease, yet it affects one out of 6,000 to 10,000 children and is the leading cause of infant mortality.

脊髓性肌萎缩症 (SMA) 是一种遗传性神经肌肉疾病，其特征是肌肉逐渐无力和萎缩，影响运动、说话、吞咽和呼吸。 SMA 是由编码 SMN 蛋白的 SMN1 基因缺失或突变引起的，SMN 蛋白是维持运动神经元存活和正常功能所必需的。 SMA 是一种罕见疾病，但发病率在6000分之一至1万分之一，并且是婴儿死亡的主要原因。

We are investigating a rAAV-based gene therapy for pediatric SMA patients. With a novel promoter design, we are able to develop a gene therapy for treatment of SMA with improved safety profile and efficacy.

我们正在研究针对小儿 SMA 患者的基于 rAAV 的基因疗法。 通过创新设计，我们能够开发出一种安全性和有效性更高的 SMA 基因疗法。

Parkinson’s disease (PD) is the second most prevalent neurodegenerative disease affecting 2-3% of the population over the age of 65 worldwide. PD is characterized by the accumulation of Lewy bodies and the progressive dopaminergic neuronal cell death, which caused tremor, stiffness, slowness of movement and impaired balance and coordination. Apart from the idiopathic cases, the association of monogenic mutations in various genes suggests the causative role of the genetic risk factors in PD.

帕金森病 (PD) 是第二大流行的神经退行性疾病，影响全球 2-3% 的 65 岁以上人口。 PD 的特点是路易体的积累和进行性多巴胺能神经元细胞死亡，导致震颤、僵硬、运动缓慢以及平衡和协调受损。 除了特发性病例外，各种基因中单基因突变的关联表明遗传风险因素在 PD 中的致病作用。

We are developing gene therapy treatment to supply an engineered and more stabilized version of the functional protein which is deficient in the sub-population of PD patients.

我们正在开发基因疗法，以提供一种经过工程改造且更稳定的功能性蛋白质，这种蛋白质在 PD 患者亚群中是缺乏的。

Wet Age-related macular degeneration (wAMD) is a leading cause of vision loss in patients over 60 years of age. It is caused by abnormal blood vessels grown in choroid and penetrate into retina, which may lead to swelling and damage of the macula. About 200,000 new cases of wet AMD are diagnosed each year in North America alone, with a global prevalence of approximate 20 million. The current standard of care is frequent injections of anti-vascular endothelial growth factor (VEGF) protein into the eye. wAMD is not a genetic disorder, but gene therapy may help with a one-time intervention.

湿性年龄相关性黄斑变性 (wAMD) 是 60 岁以上患者视力丧失的主要原因。 它是由于脉络膜异常血管生长并渗入视网膜，可能导致黄斑部肿胀和损伤。 仅在北美，每年就诊断出约 200,000 例新的湿性 AMD 病例，全球患病率约为 2000 万。 当前的护理标准是经常向眼内注射抗血管内皮生长因子 (VEGF) 蛋白。 wAMD 不是遗传病，但基因疗法可能有助于一次性的干预。

The AAV-based gene therapy we are developing delivers a gene encoding a novel therapeutic fusion protein that targets multiple distinct pathways leading to wAMD.

我们正在开发的基于 AAV 的基因疗法提供了一种编码新型治疗性融合蛋白的基因，该融合蛋白靶向导致 wAMD 的多种不同途径。

Bietti crystalline dystrophy (BCD) is a rare-inherited disease caused by mutations in the CYP4V2 gene and characterized by the presence of multiple shimmering yellow-white deposits in the posterior pole of the retina in association with atrophy of the retinal pigment epithelium (RPE) and chorioretinal atrophy. The estimated prevalence of BCD is 1 in 67,000 individuals, affecting 21,000 patients in China and about 5000 in USA. No current treatment is available.
结晶样视网膜变性(BCD) 是一种罕见的遗传性疾病，由 CYP4V2 基因突变引起，其特征是视网膜后极存在多个闪烁的黄白色沉积物，伴有视网膜色素上皮细胞 (RPE) 萎缩 和脉络膜视网膜萎缩。 据估计，BCD 的患病率为每 67,000 人中就有 1 人，影响中国的 21,000 名患者和美国的约 5000 名患者。 目前没有可用的治疗方法。

We are developing an AAV-based gene therapy with our AI-backed proprietary platform to harmony each component including capsid, promoter, gene of interest, etc.

我们正在开发一种基于 AAV 的基因疗法，使用我们的 AI 支持的专有平台来协调每个组件，包括衣壳、启动子、基因等。

Hemophilia A is a rare bleeding disorder caused by insufficient coagulation Factor VIII in blood. The deleterious mutations in F8 gene result in deficient Factor VIII. Hemophilia A affects about 1 in 5,000 male births. For most hemophilia A patients, the current treatment is prophylactic infusions two or three times every week.

血友病 A 是一种罕见的出血性疾病，由血液中凝血因子 VIII 不足引起。 F8 基因的有害突变导致凝血因子 VIII 缺陷。 血友病 A 影响大约五千分之一的男性新生儿。 对于大多数血友病 A 患者，目前的治疗方法是每周预防性输注凝血因子 2 至 3 次。

We exploit rAAV vectors to deliver a modified F8 gene to express functional Factor VIII. Through our proprietary vector design, our single-dose gene therapy is aimed to provide durable benefits to severe hemophilia A patients.

我们利用 rAAV 载体传递修饰的 F8 基因来表达功能性凝血因子 VIII。 通过我们专有的载体设计，我们的单剂量基因疗法旨在为重度血友病 A 患者提供持久的益处。

PKU is an inborn error of metabolism caused by defective phenylalanine hydroxylase (PAH) which catalyzes the phenylalanine to tyrosine. Accumulated phenylalanine can cause impaired cognitive development. PKU affects about 1 in 12,000 births.

PKU 是由苯丙氨酸羟化酶 (PAH) 缺陷引起的先天性代谢错误，该酶将苯丙氨酸催化为酪氨酸。 累积的苯丙氨酸会导致认知发育受损。 PKU 在新生儿中的发病率大约为12000分之1。

We are developing a liver-directed gene therapy for PKU by using rAAV to deliver an optimized PAH gene expression cassette.

我们正在通过使用 rAAV 提供优化的 PAH 基因表达来开发针对 PKU 的肝脏定向基因疗法。