Our Scientific Platform and Programs

Fidanacogene Elaparvovec., previouslySPK-9001., is an investigational bio-engineered AAV vector utilizing a high-activityF9.transgene for hemophilia B, or factor IX deficiency. Hemophilia B is a serious and rare inherited hematologic disorder, characterized by mutations in theF9.基因导致血液凝血的缺乏和出血风险增加或出血。

Fidanacogene Elaparvovec从美国FDA获得突破治疗和孤儿产品名称。2018年7月，Spark raybetapp下载Therapeutics将其持续的，开放标签阶段1/2的Fidanacogene Elaparvovec试验转变为辉瑞。辉瑞公司宣布计划启动3阶段的进入研究。作为合作的一部分，PFizer对所有随后的关键研究，所有监管活动，制造和潜在全球全球商业化的唯一责任均为血友病B基因治疗计划引起的任何产品。

raybetapp下载Spark Therapeutics正在发展SPK-8011，对血友病A或因子VIII缺乏的调查基因治疗。血友病A是一种严重和罕见的遗传性血液学疾病，其特征在于突变F8.基因导致血液凝血的缺乏和出血风险增加或出血。火花retains global commercialization rights to itsSPK-FVIII.program includes bothSPK-8011和SPK-8016对于血友病A.

raybetapp下载Spark Therapeutics正在发展SPK-8016, a novel, internally developed investigational gene therapy for hemophilia A, or Factor VIII deficiency, to address the inhibitor market using a gene-based approach. Hemophilia A is a serious and rare inherited hematologic disorder, characterized by mutations in theF8.基因导致血液凝血的缺乏和出血风险增加或出血。Inhibitors occur in as many as 30 percent of people with severe or moderately severe hemophilia A. Spark retains global commercialization rights toSPK-8016。

raybetapp下载Spark Therapeutics正在发展SPK-3006.一个临床实验的基因治疗潜能l treatment of Pompe disease. Pompe disease is an oftentimes fatal lysosomal storage disorder and neuromuscular disease, with systemic, multi-organ manifestations resulting from loss of function mutations in the gene encoding acid alpha-glucosidase (GAA). The initial construct forSPK-3006.2017年的Genethon获得了许可，Spark保留了全球商业化权利。

raybetapp下载火花疗法正在开发一个investigational gene therapy for the treatment of hereditary angioedema (HAE), a rare genetic condition that causes severe swelling in various parts of the body such as the extremities, intestinal tract and airway. Spark plans to pursue the use of liver-directed AAV therapy with C1-Inhibitor replenishment for the treatment of HAE.