In July 2018, Spark Therapeutics transitioned its ongoing, open-label Phase 1/2 trial of fidanacogene elaparvovec to Pfizer. Visual loss in children or infant can occur either at the stage of prenatal or postnatal stage. 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 Initiates Phase 1/2 Clinical Trial of SPK-CHM for Choroideremia, Expanding its Pipeline of Potential Treatments for Rare, Blinding Conditions The news that Roche has acquired Spark Therapeutics helped shares of the latter skyrocket 120.1%. The company has received an orphan drug designation for its SPK-TPP1 candidate for treating CLN2 disease in the United States.Additionally, Spark Therapeutics entered into a licensing and commercialization agreement with Novartis in January 2018 for the development and commercialization of the investigational drug voretigene neparvovec outside the United States.In the next article, we’ll take a look at the financial performance of Spark Therapeutics. 60% of congenital blindness are contributed from prenatal stage and another 40% are contributed from inherited disease. Spark Therapeutics Expands Leadership Team to Support Development and Commercialization of its Late-Stage Gene Therapy Pipeline. 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.
147 Spark Therapeutics jobs available on Indeed.com. Luxturna has also received an orphan product designation, and on approval, it received a rare pediatric disease priority review voucher.To build and promote access to Luxturna, Spark Therapeutics is supporting product use through small, targeted commercial and medical affairs groups in the United States. 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. This, in my opinion, validates the science and methodology behind Spark’s entire gene therapy pipeline. Find out more about how we strive to … We are providing education and other resources to promote a shift from clinical to genetic-based disease classification, and advocating for patient access to care and incentives for innovation.Spark Therapeutics is working to address a range of debilitating genetic diseases, including inherited retinal diseases (IRDs), liver-directed diseases such as hemophilia and lysosomal storage disorders and neurodegenerative diseases. There are lots of causes that can promote congenital blindness but the most concern and highest cause of it is a genetic mutation. 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. But opting out of some of these cookies may have an effect on your browsing experience.This website uses cookies and similar technologies to optimize and improve the experience on our site ( It is mandatory to procure user consent prior to running these cookies on your website.Liver Delivery / Hemophilia and Lysosomal Storage DisordersCentral Nervous System Delivery / Neurodegenerative DiseasesThis website uses cookies to improve your experience while you navigate through the website. It’s also developing candidates for neurodegenerative diseases such as Batten disease and Huntington’s disease. Reape is one of the senior Spark staffers hitting the exit in the aftermath of Roche’s $4.3 billion takeover. 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. 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. Nachrichten zur Aktie Spark Therapeutics Inc | A12HQS | ONCE | US84652J1034
The initial construct for SPK-3006 was in-licensed from Genethon in 2017, and Spark retains global commercialization rights. 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 However, most of the congenital blindness cases show that it can be avoidable or preventable with early treatment. Hemophilia A is a serious and rare inherited hematologic disorder, characterized by mutations in the F8 gene, which lead to deficient blood coagulation and an increased risk of bleeding or hemorrhaging. Spark Therapeutics’ chief medical officer Kathy Reape is set to leave the biotech. 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.