Figure 2: Pipeline (source: corporate presentation) We also use third-party cookies that help us analyze and understand how you use this website. These cookies do not store any personal information. [3], In January 2015, the company became a public company, trading under the ticker $ONCE via a $161 million initial public offering[4] led by Chief Legal Officer Joseph La Barge.[5]. 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 ABCA4 gene. Analytical cookies are used to understand how visitors interact with the website. Environmental, Social and Governance (ESG), HVAC (Heating, Ventilation and Air-Conditioning), Machine Tools, Metalworking and Metallurgy, Aboriginal, First Nations & Native American, corporate headquarters and manufacturing facility. Spark Therapeutics is currently meeting with insurers and the FDA to discuss how this therapy will be priced, though many analysts believe the drug will be priced anywhere from $400,000-500,000 per treatment (per eye), for a total cost of $800,000-$1,000,000. Spark Therapeutics was founded in March 2013 as a result of the technology and know-how developed at Childrens Hospital of Philadelphia (CHOP). The cookie is used to store the user consent for the cookies in the category "Other. 32 open jobs for Pipeline engineer. 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. 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. Spark Therapeutics had about . You will receive a link to create a new password via email. philadelphia, dec. 8, 2014 /prnewswire/ -- spark therapeutics, a late-stage gene therapy company developing treatments for debilitating genetic diseases, announced today that it has entered. We use cookies on our website to give you the most relevant experience by remembering your preferences and repeat visits. Our mission at Spark Therapeutics is to challenge the inevitability of genetic disease by discovering, developing and delivering potential treatments in ways unimaginableuntil now. The cookies is used to store the user consent for the cookies in the category "Necessary". 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. For more information, visit. Spark Therapeutics is a gene therapy company. For information about Spark Therapeutics products, visit www.sparktx.com/products. There are lots of opportunities within the company Aug 20, 2022 - Quality Control Analyst in Philadelphia, PA Recommend CEO Approval Business Outlook Pros The culture is the highlight of the company. SEC Filings. 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. 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. ", "Spark nails a $161M IPO to fund its 'breakthrough' gene therapy", "Shake Shack wasn't the day's only gonzo IPO. Spark Therapeutics will consider funding requests for the disease states we investigate. SPK-RPE65's positive phase 3 top-line results brought Spark Therapeutics much closer to the commercial stage. 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. 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. Overview. 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. See what kind of people work at Spark Therapeutics, career paths working at Spark Therapeutics, company culture, salaries, employee political affiliation, and more. Spark Therapeutics, a fully integrated company, strives to challenge the inevitability of genetic disease by working to discover, develop and deliver gene therapies that addressinherited retinal diseases (IRDs), neurodegenerative diseases, as well as diseases that can be addressed by targeting the liver. Volastra Therapeutics Feb 2022 - Present 10 . This category only includes cookies that ensures basic functionalities and security features of the website. 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 Worlds 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. Under the terms of the licensing agreement, Novartis will pay Spark Therapeutics $105 million in cash as an upfront fee. Spark Therapeutics SPK-8011 AAV-vector gene therapy Hemophilia A Ph I/II plan to initiate a Phase 3 run-in study in Q4 2018 Bioverativ BIVV003 Gene-edited cell therapy Sickle cell disease Pre-Ph I Received IND approval in May Pipeline. The vectors used in our research programs have been engineered using Sparks cutting-edge proprietary platform, selected through vigorous preclinical testing and validated in several clinical trials. It has a pipeline of product candidates targeting multiple rare blinding conditions, hematologic disorders and neurodegenerative diseases. He also spearheaded Spark's $4.8 billion acquisition. Spark Therapeutics, Inc. [13] In July 2018, fidanacogene elaparvovec entered late stage clinical trials. Spark retains global commercialization rights to its SPK-FVIII program. "Our goal from day one of CHOP's spin-out of Spark Therapeutics was to more effectively advance a deep clinical pipeline of gene therapy products with the potential to benefit patients. Analyst Coverage. "The Spark team has significant experience across the core capabilities required to develop gene therapy products as evidenced by the quality of the company's pipeline and programs. Phone: 1-855-SPARKTX / +1 215-220-9300. [14], SPK-8011 is an experimental drug under investigation for treatment of Haemophilia A. In July 2018, Spark Therapeutics transitioned its ongoing, open-label Phase 1/2 trial of fidanacogene elaparvovec to Pfizer. These cookies help provide information on metrics the number of visitors, bounce rate, traffic source, etc. [6][7] It now continues to operate as an independent subsidiary. 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. For more information, . HELIXMITH Co., Ltd., Sarepta Therapeutics, Solid Biosciences Inc., Lexeo Therapeutics, Spark Therapeutics, Xalud Therapeutics, uniQure, Ultragenyx Pharmaceutical, Nanoscope Therapeutics . Contact bizdev@sparktx.com with any inquiries or to submit a proposal. This website uses cookies to improve your experience while you navigate through the website. 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. 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. 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. Spark recently announced plans to open its corporate headquarters and manufacturing facility. Spark Therapeutics is developing SPK-8011, an investigational gene therapy for hemophilia A, or factor VIII deficiency. 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. Corporate Governance. Hemophilia B is a serious and rare inherited hematologic disorder, characterized by mutations in the F9 gene, which lead to deficient blood coagulation and an increased risk of bleeding or hemorrhaging. Spark Therapeutics initiated the ongoing Phase 1/2 clinical trial of fidanacogene elaparvovec that was transitioned to Pfizer in July 2018. Spark Therapeutics is developing an investigational gene therapy for the treatment of Huntingtons disease, a fatal genetic disorder that causes the progressive breakdown of nerve cells in the brain. But opting out of some of these cookies may have an effect on your browsing experience. Other uncategorized cookies are those that are being analyzed and have not been classified into a category as yet. These cookies do not store any personal information. Company's longtime business and pipeline leader Ron Philip takes helm as CEO. The therapy transfers a working copy of the Factor VIII gene into patients who lack one. Spark Therapeutics, Inc. 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. [10], 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. "Gene therapy helped these children see. Spark Therapeutics is developing 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. Spark is currently trading below the perceived value of SPK-RPE65 and the company. Brookside Capital and Sofinnova Investments are the most recent investors. Its pipeline includes a product candidate targeting choroideremia (CHM), which is in a Phase I/II clinical . About. Media Inquiries: Jessica Rowlands202-729-4089[emailprotected], Cision Distribution 888-776-0942 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). Additionally, the company has clinical and preclinical programs in other inherited retinal dystrophies and hematological disorders, and a proprietary manufacturing platform that has successfully supported human gene therapy trials across diverse therapeutic areas and routes of administration. 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. The Children's Hospital of Philadelphia (CHOP), a co-founder of the company, also participated significantly in the round. Spark Therapeutics, Inc. Biotechnology Research Philadelphia, PA 62,450 followers We don't follow footsteps. . 99 Hayden Avenue Suite 120, Building E Lexington, MA 02421 It is entering phase III clinical trials in the United States. Such a high price tag, coupled with Luxturna being billed as a one-time treatment . Additionally, we are continuing to build strategic partnerships to help accelerate our progress for patients. The reactions against the treatment were seen as a set-back, though Spark suggested that the responses could be controlled with steroids, and promised to move forward with Phase III testing. Develop connectors for ETL pipeline to Arcadia data lake . Philadelphia, PA 19104 This website uses cookies and similar technologies to optimize and improve the experience on our site (. But opting out of some of these cookies may affect your browsing experience. State-of-the-art, in-house expertise in vector manufacturing. Other preclinical programs in our pipeline include investigational gene therapies for Stargardt disease and an additional Huntingtons disease candidate that we have in-licensed. . [8] Since the acquisition by Swiss pharma Roche, several key founding executives have departed, including scientist and co-founder Katherine High in February 2020,[9] Chief Business/Legal Officer Joseph La Barge in December 2021, and co-founder and Chief Executive Officer Jeffrey Marrazzo in April 2022 [10], On February 23, 2022, Marrazzo named big-Pharma veteran Ron Philip as his successor. CHOP previously committed $50 million in funding as part of the launch of Spark Therapeutics in October 2013, investing $10 million in a Series A financing. We are advancing research programs against cell targets in the retina, liver and central nervous system using adeno-associated viral (AAV) vectors. At Tmunity, we are implementing novel science into the clinic to find faster pathways to patients. We continue to advance our portfolio of investigational gene therapies for hemophilia A, or factor VIII deficiency. Browse 12 Jobs at Spark Therapeutics. 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. You also have the option to opt-out of these cookies. Website http://www.sparxbio.com Industries. Spark Therapeutics is registered under the ticker NASDAQ:ONCE . 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. CHOP's participation in this round brings its total equity investment in Spark to more than $30 million. You also have the option to opt-out of these cookies. Our Pipeline Our Leads Folder: Investors and Media. Innovative scientific and regulatory strategies. One patient had to be hospitalized. Press Releases. Spark Therapeutics is researching liver-directed therapies to address a range of diseases like lysosomal storage disorders such as Fabry disease. In 2019, Spark received the Prix Galien USA Award for Best Biotechnology Product. This website uses cookies and similar technologies to optimize and improve the experience on our site (. 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 successful completion of a $72.8 million Series B financing led by Sofinnova Ventures. Spark Therapeutics: Pioneering Gene Therapy. For more information, visit www.sparktx.com, and follow us on Twitter and LinkedIn.. 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. 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). "2022 is set to be a turning point in the U.S., as biosimilars expand into new therapeutic areas and sites of care, and reimbursement models continue to evolve," Hunter said." 6. By: Peter Ciszewski| Published on: Mar 3, 2019. This website uses cookies to improve your experience while you navigate through the website. PHILADELPHIA, Feb. 23, 2022 (GLOBE NEWSWIRE) -- Spark Therapeutics, a member of the Roche Group (SIX: RO, ROG; OTCQX . These cookies track visitors across websites and collect information to provide customized ads. Spark Therapeutics is developing SPK-3006, an investigational gene therapy for the potential treatment of Pompe disease. SPARK THERAPEUTICS INC : Company profile, business summary, shareholders, managers, financial ratings, industry, sector and market information | Nasdaq: | Nasdaq Drug resistant focal epilepsy occurs when a patients epileptic seizures are sometimes not controlled with antiseizure medications. The initial construct for SPK-3006 was in-licensed from Genethon in 2017, and Spark retains global commercialization rights. Huntingtons disease is characterized by motor, cognitive and behavioral symptoms which usually appear between the ages of 30 to 50, and worsen over a 10- to 25-year period. Our viral vectors are designed to encapsulate genetic material for the potential treatment of genetic diseases. Horizon Therapeutics Personal Approach to Rare Diseases, Brief Overview of CLL Treatment Landscape. Athena DB; Development in Spark running on AWS; Limited use of Groovy, maven, gradle The Company focuses on treating orphan diseases. We invite you to explore our pipeline*. Spark's initial focus is on treating orphan diseases where no, or only palliative therapies, exist. [11], Fidanacogene elaparvovec, previously known by its study ID number SPK-9001,[12] is an experimental drug under investigation for treatment of hemophilia B in partnership with Pfizer. 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. [1] It is a subsidiary of Hoffmann-La Roche . "The funding will support the expansion of our team and ongoing development of our pipeline as we build the infrastructure needed for a first-in-class, FDA-approved gene therapy.". Functional cookies help to perform certain functionalities like sharing the content of the website on social media platforms, collect feedbacks, and other third-party features. Spark Therapeutics, a fully integrated company, strives to challenge the inevitability of genetic disease by discovering, developing, and delivering gene therapies that address inherited retinal diseases (IRDs), neurodegenerative diseases, as well as diseases that can be addressed by targeting the liver. Please enter your username or email address. We currently have four programs in clinical trials. 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, Inc. This cookie is set by GDPR Cookie Consent plugin. Our mission Challenge the inevitability of genetic disease by discovering, developing and delivering treatments in ways unimaginable - until now. Spark Therapeutics is working to address a range of debilitating genetic diseases. Spark's lead program addresses a specific form of inherited retinal dystrophy caused by mutations in the RPE65 gene. 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. It is mandatory to procure user consent prior to running these cookies on your website. Spark Therapeutics also supports research that advances medical and scientific knowledge in the company's therapies and fields of interest. [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. Roche is acquiring Spark Therapeutics in a multi-billion dollar deal that underscores big pharma's growing appetite for new technologies like gene therapy. Spark went public in 2015 raising $161 million in one of the year's largest initial public stock offerings. Gene therapy clinical trial pipeline constitutes 250+ key companies continuously working towards developing 300+ gene therapies, analyzes DelveInsight . Each of our investigational programs currently uses as adeno-associated viral (AAV) vector developed and manufactured by the Spark team and our collaborators. Members of our founding scientific team have been at the forefront of gene therapy research for more than two decades. Pfizer has announced it plans to initiate a Phase 3 lead-in study. 3737 Market Street Annual Reports. To learn more visit www.sparktx.com. At Spark, a member of the Roche Group, we see the path to a world where no life is limited by genetic disease. The company was founded in 2013 by Katherine A. Our company was also named to Science Magazines Top Employer list in 2019, ranking in the top ten. 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. The cookie is used to store the user consent for the cookies in the category "Analytics". Spark Therapeutics, Inc. focuses on the development of gene therapy products. Spark Therapeutics, Inc. is a developer of gene therapy treatments, which treat debilitating genetic diseases. Our vision is a world where no life is limited by genetic disease. 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. NeuExcell's pipeline covers major neurodegenerative diseases such as Stroke, Huntington's Disease, Amyotrophic Lateral . Spark's founding team includes scientists who led the movement to develop gene therapy as a new treatment paradigm, establishing clinical proof of concept in the eye and liver and contributing key insights to the field that have resulted in a resurgence of industry interest in gene-based medicines. Spark Therapeutics will enroll up to five additional participants in the current Phase 1/2 clinical trial who will receive SPK-9001 manufactured using an enhanced process to test its . It does not store any personal data. These cookies will be stored in your browser only with your consent. Stock Information. We also use third-party cookies that help us analyze and understand how you use this website. Hemophilia A Drug Pipeline Market Research Report 2021 Featuring Spark Therapeutics, Sigilon Therapeutics, ASC Therapeutics, Pfizer, Sanofi Genzyme, Novo Nordisk - ResearchAndMarkets.com Posted . Similar companies and competitors in the areas of Biotech, Life Sciences, Drug Development, Singe Dose Gene Therapy, Gene Therapy, Biologics, DNA and more.
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