About. Along with our ongoing research across therapeutic areas, we are continuing to conduct research related to the immune system, targeted gene therapy deliveries, and enabling technologies to optimize delivery and potentially expand access for eligible patient populations. Sparx has a rich development pipeline for treatment of cancer and CNS diseases, leveraging its proprietary and proven antibody and chemistry platforms. In this series, we'll take a look at six. The CHOP Foundation will collect about $430 million of that total for its Spark shares a huge return for the hospital's $33 million investment since 2013. The cookie is used to store the user consent for the cookies in the category "Analytics". Spark Therapeutics is developing SPK-8011, an investigational gene therapy for hemophilia A, or factor VIII deficiency. Website http://www.sparxbio.com Industries. Spark also has a deep pipeline of programs behind the RPE65 program in other inherited retinal dystrophies, hematological disorders, and other rare diseases. 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. . These cookies will be stored in your browser only with your consent. Spark Therapeutics has raised a total of $122.8M in funding over 2 rounds. The Children's Hospital of Philadelphia (CHOP), a co-founder of the company, also participated significantly in the round. 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. Spark went public in 2015 raising $161 million in one of the year's largest initial public stock offerings. Spark Therapeutics: Pioneering Gene Therapy. To learn more visit www.sparktx.com. Advertisement cookies are used to provide visitors with relevant ads and marketing campaigns. This cookie is set by GDPR Cookie Consent plugin. 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. Back. For Swedish gene therapy company CombiGene, whose candidate CG01 is being developed in collaboration with Spark Therapeutics, bluebird's approval is a barometer of the interest in the gene therapy field in general. Each of Sparks research programs uses an adeno-associated viral (AAV) vector. First participant dosed in the RESOLUTE SM trial, a Phase 1/2 dose-escalation study of SPK-3006. Brookside Capital and Sofinnova Investments are the most recent investors. from 8 AM - 9 PM ET. 3737 Market Street Spark Therapeutics is developing curative, one-time gene therapy products with the potential to transform the lives of patients and re-imagine the treatment of debilitating diseases. Browse 12 Jobs at Spark Therapeutics. spark's robust pipeline includes spk-rpe65, a fully enrolled, pivotal phase 3 program in blindness due to mutations in the rpe65 gene, spk-chm for the treatment of choroideremia, and spk-fix, a program for the potential treatment of hemophilia b through a global collaboration with pfizer inc., as well as preclinical programs to address Spark Therapeutics has built a leading integrated gene therapy platform as we strive to turn genes into medicines for patients with inherited diseases, including inherited retinal diseases (IRDs), liver-directed diseases including hemophilia and lysosomal storage disorders such as Pompe and Fabry, and neurodegenerative diseases. This program is currently in Phase 3 and builds on an earlier clinical study in which 12 patients with RPE65-related blindness demonstrated significant improvement, moving in some cases from being profoundly blind to being able to function in sighted classrooms, recognize faces, and walk independently. With 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. About Spark Therapeutics Spark is a gene therapy leader seeking to transform the lives of patients suffering from debilitating genetic diseases by developing one-time, life-altering treatments. Enrollment of approximately 20 total study participants is ongoing. Spark made history in 2017 when it won FDA approval to market . 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. Performance cookies are used to understand and analyze the key performance indexes of the website which helps in delivering a better user experience for the visitors. Pfizer has announced it plans to initiate a Phase 3 lead-in study. This cookie is set by GDPR Cookie Consent plugin. This website uses cookies to improve your experience while you navigate through the website. This website uses cookies and similar technologies to optimize and improve the experience on our site (. ", "Spark nails a $161M IPO to fund its 'breakthrough' gene therapy", "Shake Shack wasn't the day's only gonzo IPO. Events & Presentations. PHILADELPHIA, PA, USA I February 01, 2021 I Spark Therapeutics, a member of the Roche Group (SIX: RO, ROG; OTCQX: RHHBY) and a fully integrated, commercial gene therapy company dedicated to challenging the inevitability of genetic . To learn more visit www.sparktx.com. We engineer investigational gene therapy vectors using a cutting-edge, proprietary adeno-associated viral (AAV) vector platform, developed through vigorous preclinical and clinical testing. 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). Legal Name Spark Therapeutics, Inc. Stock Symbol NASDAQ:ONCE Company Type For Profit Contact Email info@sparktx.com Phone Number +1 215-220-9300 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. Ron Philip, Chief Commercial Officer of Spark Therapeutics, discusses his companys 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. PHILADELPHIA, Jan. 20, 2015 /PRNewswire/ -- Spark Therapeutics, a late-stage gene therapy company developing treatments for debilitating, genetic diseases, announced today it has initiated. SPK-RPE65's positive phase 3 top-line results brought Spark Therapeutics much closer to the commercial stage. philadelphia, jan. 9, 2014 /prnewswire/ -- spark therapeutics, a late-stage, fully integrated company developing gene-based medicines for a wide range of debilitating diseases, announced today. Spark Therapeutics also supports research that advances medical and scientific knowledge in the company's therapies and fields of interest. 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. 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. Spark Therapeutics is developing SPK-8011, an investigational gene therapy for hemophilia A, or factor VIII deficiency. Phone: 1-855-SPARKTX / +1 215-220-9300. We currently have four programs in clinical trials. SPK-8011 is an investigational gene therapy for hemophilia A, or factor VIII deficiency. Our vision A world where no life is limited by genetic disease. PHILADELPHIA, May 27, 2014 /PRNewswire/ -- Spark Therapeutics, a late-stage company developing gene-based medicines for a wide range of rare, debilitating diseases, today announced the. Spark Therapeutics, Inc. is a developer of gene therapy treatments, which treat debilitating genetic diseases. If you have an opportunity for collaboration or an interesting technology, or if you have an asset in one of our areas of interest, we would like to hear from you. Spark Therapeutics will consider funding requests for the disease states we investigate. Learn more about our platform below. 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. [1] The company is currently developing several gene therapies to target a suite of diseases, including Haemophilia A and B, and several central nervous system diseases. When typing in this field, a list of search results will appear and be automatically updated as you type. At Spark, a member of the Roche Group, we see the path to a world where no life is limited by genetic disease. The pipeline also includes SPK-7001 in an ongoing Phase 1/2 clinical trial for choroideremia. Spark Therapeutics is funded by 7 investors. 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. Company's longtime business and pipeline leader Ron Philip takes helm as CEO. Spark Therapeutics, Inc. Biotechnology Research Philadelphia, PA 62,450 followers We don't follow footsteps. 32 open jobs for Pipeline engineer. Overview. Spark Therapeutics is developing SPK-3006, an investigational gene therapy for the potential treatment of Pompe disease. It does not store any personal data. Usa Award for Best Biotechnology product ) vectors the experience on our website function Research programs against cell targets in the category `` Functional '' thrilled to lead this brings! 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