Spark Therapeutics, a fully integrated company, strives to challenge the inevitability of genetic disease by working to discover, develop and deliver gene therapies that address inherited retinal diseases (IRDs), neurodegenerative diseases, as well as diseases that can be addressed by targeting the liver.
At Spark Therapeutics, a fully integrated, commercial company committed to discovering, developing and delivering gene therapies, we challenge the inevitability of genetic diseases, including blindness, hemophilia, lysosomal storage disorders and neurodegenerative diseases. We aim to reawaken healthy biologic processes through the potential one-time administration of gene therapies and spark a transformation for people affected by rare genetic diseases where no, or only palliative, therapies exist. Our vision is a world where no life is limited by genetic disease.
Our approach to gene therapy is to investigate treatments that target an inherited disease at its root by augmenting, replacing or suppressing the function of a mutated gene. We engineer investigational gene therapy vectors using a cutting-edge, proprietary adeno-associated viral (AAV) vector platform, developed through vigorous preclinical and clinical testing. Our validated gene therapy platform has delivered human proof-of-concept data in two target tissues and secured breakthrough therapy designations in the retina and liver.
We are the only biotechnology company that has successfully commercialized a gene therapy for a genetic disease in the U.S., bringing a one-time treatment to market. We have created unique competencies in the discovery, development and delivery of genetic medicines which are unmatched across the value chain, including target selection and AAV vector optimization, commercial and scalable AAV manufacturing, regulatory innovation and precedent-setting approvals and gene therapy market development and access.
Spark Therapeutics was founded in March 2013 as a result of the technology and know-how developed at Children’s Hospital of Philadelphia (CHOP). Members of our founding scientific team have been at the forefront of gene therapy research for more than two decades. They are responsible for numerous development milestones, including the first clinical trials of AAV vectors in skeletal muscle tissue and the liver, the first clinical studies to evaluate AAV administration to the second eye, the first gene therapy trial for a nonlethal disorder that included pediatric participants, and the first approved gene therapy for a genetic disease in the U.S.
The Spark Therapeutics team includes more than 450 professionals with deep experience in research and development activities, manufacturing and commercializing complex and novel biotechnology products. We are headquartered in Philadelphia, where our state-of-the-art current good manufacturing practices (cGMP) manufacturing facility, the only AAV commercial manufacturing facility for an FDA approved gene therapy for a genetic disease, is located.
In 2019, Spark received the Prix Galien USA Award for Best Biotechnology Product. Our company was also named to Science Magazine’s Top Employer list in 2019, ranking in the top ten. We received recognition from MIT Technology Review as a “50 Smartest Companies” and to Bloomberg Businessweek as one of their “50 Companies to Watch.” We have also been recognized as one of the World’s Most Innovative Companies by Fast Company magazine, and as one of the “Best Places to Work” for three years in a row (2017-2019) by the Philadelphia Business Journal.
In December 2019, Spark Therapeutics was acquired by the Roche Group and will maintain its headquarters in Philadelphia.
For information about Spark Therapeutics products, visit www.sparktx.com/products.
Our Clinical Programs
We are putting our unique competencies to use to evaluate and select a portfolio of potential gene therapies targeting three target tissues – the retina, liver and CNS – across multiple therapeutic areas by moving investigational assets into the clinic to optimize our success at developing and delivering medicines to patients with unmet medical needs.
We continue to advance our portfolio of investigational gene therapies for hemophilia A, or factor VIII deficiency. We have initiated the Phase 3 run-in study for SPK-8011 for the hemophilia A non-inhibitor patient population. The Phase 1/2 dose-finding study for SPK-8016 for the hemophilia A inhibitor patient population will initially evaluate safety, efficacy and tolerability in non-inhibitor patients with clinically severe hemophilia A. Hemophilia A is a serious and rare inherited hematologic disorder, characterized by mutations in the coagulation factor VIII, or F8 gene, which lead to deficient blood coagulation and an increased risk of bleeding or hemorrhaging. Spark Therapeutics retains global commercialization rights to its SPK-8011 and SPK-8016 programs for hemophilia A.
Fidanacogene elaparvovec, previously SPK-9001, is an investigational bio-engineered AAV vector utilizing a high-activity F9 transgene for hemophilia B, or factor IX deficiency. Hemophilia B is a serious and rare inherited hematologic disorder, characterized by mutations in the coagulation factor IX, or F9 gene, which lead to deficient blood coagulation and an increased risk of bleeding or hemorrhaging. Fidanacogene elaparvovec has received both breakthrough therapy and orphan product designations from the U.S. FDA. Spark Therapeutics initiated the ongoing Phase 1/2 clinical trial of fidanacogene elaparvovec that was transitioned to Pfizer in July 2018. At this time, Pfizer is conducting the Phase 3 trial. 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.
We are also developing SPK-3006, previously SPK-GAA, an investigational gene therapy for the potential 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 for SPK-3006 was in-licensed from Genethon in 2017, and Spark retains global commercialization rights.
SPK-1001 is an investigational central nervous system (CNS)-directed AAV gene therapy that has demonstrated preclinical proof-of-concept in a naturally occurring model of tripeptidyl peptidase 1 (TPP1) enzyme deficiency, or CLN2 (a form of Batten disease). Batten disease is a fatal neurological disorder that begins in early childhood and is characterized by mutations of the TPP1 gene. We have obtained orphan drug designation from the U.S. FDA for SPK-1001 for the treatment of CLN2 disease caused by TPP1 deficiency and Spark retains global rights.
Other preclinical programs in our pipeline include investigational gene therapies for Stargardt disease and an additional Huntington’s disease candidate that we have in-licensed.
*Last updated: December 2019