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.has received both breakthrough therapy and orphan product designations from the U.S. FDA. In July 2018, Spark Therapeutics transitioned its ongoing, open-label Phase 1/2 trial of fidanacogene elaparvovec to Pfizer. Pfizer has announced it plans to initiate a Phase 3 lead-in study. As part of the collaboration, Pfizer assumes sole responsibility for all subsequent pivotal studies, all regulatory activities, manufacturing and potential global commercialization of any products resulting from the hemophilia B gene therapy program.

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-fviiiprogram 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.，一种用于潜在治疗Pompe病的研究基因治疗。Pompe疾病是致命溶酶体储存障碍和神经肌肉疾病的常杂化，具有由编码酸性α-葡糖苷酶（GaA）的基因中的功能突变失去导致的多器官表现。初始构造SPK-3006.2017年的Genethon获得了许可，Spark保留了全球商业化权利。