In July 2018, Spark Therapeutics transitioned its ongoing, open-label Phase 1/2 trial of fidanacogene elaparvovec to Pfizer. [1] It is a subsidiary of Hoffmann-La Roche.. Each of our investigational research programs uses an adeno-associated viral (AAV) vector developed and manufactured by the Spark team and our collaborators. These programs include an innovative contracting model and an outcomes-based rebate arrangement with a short-term efficacy measure and a long-term durability measure.Spark Therapeutics’ clinical pipeline includes an ocular program consisting of its product candidate SPK-7001 targeting choroideremia and hemophilia programs consisting of SPK-9001 targeting hemophilia B and SPK-8011 targeting hemophilia A.SPK-7001 is currently in Phase 1 and 2 trials, and SPK-9001 and SPK-8011 are in Phase 1 and 2 trials.Spark Therapeutics also has several products in various stages of preclinical development. These cookies do not store any personal information.Any cookies that may not be particularly necessary for the website to function and is used specifically to collect user personal data via analytics, ads, other embedded contents are termed as non-necessary cookies. Each of Spark’s research programs uses an adeno-associated viral (AAV) vector.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. Pricing and Financials.
The company announced two novel payer programs in January 2018 to ensure eligible patients in the United States have access to Luxturna. Visual loss in children or infant can occur either at the stage of prenatal or postnatal stage. Spark Therapeutics is basically known for the development of … Spark Therapeutics’ Pipeline Overview Ron Philip, Chief Commercial Officer of Spark Therapeutics, discusses his company’s pipeline which is focused on a range of debilitating genetic diseases, including inherited retinal diseases (IRDs), liver-directed diseases such as hemophilia and lysosomal storage disorders and neurodegenerative diseases. The company is developing other liver-directed gene therapies, including SPK-GAA for Pompe disease, which is an inherited lysosomal storage disorder that leads to the accumulation of glycogen in cells. Reape is one of the senior Spark staffers hitting the exit in the aftermath of Roche’s $4.3 billion takeover. Spark Therapeutics, based in Philadelphia, Pennsylvania, is a fully integrated, commercial company committed to discovering, developing and delivering gene therapies for genetic diseases, including blindness, haemophilia, lysosomal storage disorders and neurodegenerative diseases.
60% of congenital blindness are contributed from prenatal stage and another 40% are contributed from inherited disease. At the forefront of gene therapy research for more than two decades, members of our scientific team are responsible for numerous development milestones, including the first clinical trials of adeno-associated viral (AAV) vectors in skeletal muscle tissue and the liver; the first clinical studies to evaluate AAV administration to the second eye; and the first gene therapy trial for a non-lethal disorder that included pediatric participants.We are committed to developing an approach to patient access tailored to the unique nature of an investigational, potentially one-time, life-altering gene therapy. Ron Philip, Chief Commercial Officer of Spark Therapeutics, discusses his company’s pipeline which is focused on a range of debilitating genetic diseases, including inherited retinal diseases (IRDs), liver-directed diseases such as hemophilia and lysosomal storage disorders and neurodegenerative diseases. This, in my opinion, validates the science and methodology behind Spark’s entire gene therapy pipeline. Spark Therapeutics, Inc. 3737 Market Street Philadelphia, PA 19104 Phone: 1-855-SPARKTX / +1 215-220-9300 © 2017–2019 Spark Therapeutics, Inc. P-RPE65-US-450002-7 Spark Therapeutics, Inc. is a developer of gene therapy treatments, which treat debilitating genetic diseases. The company has 3 gene therapy product candidates in clinical development: (i) SPK-8011, a candidate in the SPK-FVIII program for hemophilia A; (ii) SPK-8016, a product candidate for the hemophilia A inhibitor market; and (iii) SPK-7001, targeting choroideremia, or CHM. There are lots of causes that can promote congenital blindness but the most concern and highest cause of it is a genetic mutation. Spark retains global commercialization rights to its SPK-FVIII program includes both SPK-8011 and SPK-8016 for hemophilia A.SPK-3006 is an investigational gene therapy for the potential treatment of Pompe disease. Spark Therapeutics is developing potentially curative, one-time gene therapy products to transform the lives of patients and re-imagine the treatment of debilitating diseases. Nachrichten zur Aktie Spark Therapeutics Inc | A12HQS | ONCE | US84652J1034 The vectors used in our research programs have been engineered using Spark’s cutting-edge proprietary platform, selected through vigorous preclinical testing and validated in several clinical trials.State-of-the-art, in-house expertise in vector manufacturingWith adeno-associated viral (AAV) vector manufacturing capabilities in-house, investigational clinical-grade vectors developed and manufactured by our team have been delivered through six routes of administration to hundreds of patients in more than a dozen clinical trials.We work in a spirit of collaboration with investigators and regulators to develop rigorous and novel endpoints that measure the potential impact of our investigational, potentially one-time therapies on targeted diseases and to help shape high standards for future gene therapies.
147 Spark Therapeutics jobs available on Indeed.com. Our mission at Spark Therapeutics is to challenge the inevitability of genetic disease by discovering, developing and delivering potential treatments in ways unimaginable—until now. Contents. Each of our investigational research programs uses an adeno-associated viral (AAV) vector developed and manufactured by the Spark team and our collaborators.Spark Therapeutics is advancing an open-label, dose-escalating Phase 1/2 trial designed to assess the safety and preliminary efficacy of subretinal administration of investigational Spark Therapeutics is investigating a potential gene therapy for Stargardt disease, the most common form of inherited juvenile macular degeneration, which is caused by a mutation in Fidanacogene elaparvovec has received both breakthrough therapy and orphan product designations from the U.S. FDA. Out of these cookies, the cookies that are categorized as necessary are stored on your browser as they are essential for the working of basic functionalities of the website.