spark therapeutics pipeline

Other preclinical programs in our pipeline include investigational gene therapies for Stargardt disease and an additional Huntingtons disease candidate that we have in-licensed. To learn more visit www.sparktx.com/pipeline . It does not store any personal data. The cookie is used to store the user consent for the cookies in the category "Analytics". "Gene therapy helped these children see. For more information, visit www.sparktx.com, and follow us on Twitter and LinkedIn.. [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. Intent on expanding into gene therapy, Roche has agreed to acquire Spark Therapeutics for $4.8 billion cash, Spark said today, in a deal that would add the marketed Luxturna (voretigene neparvovec-rzyl) and a pipeline of other gene therapies to the biopharma giant's portfolio. 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 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 Companies like Spark Therapeutics. We currently have four programs in clinical trials. 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. We currently have four programs in clinical trials. It has a pipeline of product candidates targeting multiple rare blinding conditions, hematologic disorders and neurodegenerative diseases. This website uses cookies to improve your experience while you navigate through the website. Epilepsy is a major global medical problem and studies suggest that epilepsy fails to come quickly under control with medicines in about one-third of adults and approximately 20-25% of children. 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. 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. 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 . 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. You also have the option to opt-out of these cookies. Given the addressable patient population of 3,500 betwee [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. Fidanacogene elaparvovec is an adeno-associated viral vector which is designed to transfer a working copy of the Factor IX gene into the livers of patients who carry non-functioning copies. Our company was also named to Science Magazines Top Employer list in 2019, ranking in the top ten. "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. Our vision A world where no life is limited by genetic disease. 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. The initial construct for SPK-3006 was in-licensed from Genethon in 2017, and Spark retains global commercialization rights. [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 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. 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. "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. Spark Therapeutics, Inc. insights Based on 10 survey responses What people like Ability to learn new things Time and location flexibility Fair pay for job Areas for improvement Energizing work tasks Sense of belonging Overall satisfaction The best job I'll ever have Quality Control Analyst (Former Employee) - Philadelphia, PA - February 20, 2022 Horizon Therapeutics Personal Approach to Rare Diseases, Brief Overview of CLL Treatment Landscape. But opting out of some of these cookies may have an effect on your browsing experience. It has a pipeline of product candidates targeting multiple rare blinding conditions, hematologic disorders and neurodegenerative diseases. The Company provides therapy products to transform the lives of patients and re-imagine the treatment of debilitating . Their latest funding was raised on May 27, 2014 from a Series B round. The cookie is set by GDPR cookie consent to record the user consent for the cookies in the category "Functional". At Tmunity, we are implementing novel science into the clinic to find faster pathways to patients. In 2019, Spark received the Prix Galien USA Award for Best Biotechnology Product. Roche's Spark Therapeutics unit is offering up to $328.5 million in biobucks for CombiGene's gene therapy for epilepsy, which has a specific focus on drug-resistant forms | Roche's Spark . NeuExcell Therapeutics and Spark Therapeutics, a member of the Roche Group announced a gene therapy collaboration aimed at developing a safe and effective treatment for patients suffering from Huntington's Disease. Spark is a member of the Roche Group. Pfizer has announced it plans to initiate a Phase 3 lead-in study. 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. Fidanacogene elaparvovec has received both breakthrough therapy and orphan product designations from the U.S. FDA. 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. Enrollment of approximately 20 total study participants is ongoing. Drug resistant focal epilepsy occurs when a patients epileptic seizures are sometimes not controlled with antiseizure medications. We also use third-party cookies that help us analyze and understand how you use this website. Contact bizdev@sparktx.com with any inquiries or to submit a proposal. 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. Advertisement cookies are used to provide visitors with relevant ads and marketing campaigns. 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. Strong commitment to improve patient care. "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. 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. [17], SPK-3006 is an experimental drug under investigation for treatment of Pompe disease, a genetic disorder that leads to failure to correctly metabolize glycogen. Philadelphia, PA 19104 HELIXMITH Co., Ltd., Sarepta Therapeutics, Solid Biosciences Inc., Lexeo Therapeutics, Spark Therapeutics, Xalud Therapeutics, uniQure, Ultragenyx Pharmaceutical, Nanoscope Therapeutics . Spark Therapeutics was founded in March 2013 as a result of the technology and know-how developed at Childrens Hospital of Philadelphia (CHOP). 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. Press Releases. 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. Lost your password? Each of our investigational programs currently uses as adeno-associated viral (AAV) vector developed and manufactured by the Spark team and our collaborators. 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. The Company focuses on treating orphan diseases. Necessary cookies are absolutely essential for the website to function properly. Spark's initial focus is on treating orphan diseases where no, or only palliative therapies, exist. To learn more visit www.sparktx.com. The therapy transfers a working copy of the Factor VIII gene into patients who lack one. 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. Fidanacogene elaparvovec has received both breakthrough therapy and orphan product designations from the U.S. FDA. 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 . 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. We also use third-party cookies that help us analyze and understand how you use this website. The cookie is set by the GDPR Cookie Consent plugin and is used to store whether or not user has consented to the use of cookies. Spark Therapeutics is researching liver-directed therapies to address a range of diseases like lysosomal storage disorders such as Fabry disease. Spark Therapeutics is developing SPK-3006, an investigational gene therapy for the potential treatment of Pompe disease. And this one, for Spark Therapeutics, raised more cash", "Roche completes $4.3B purchase of Philadelphia gene therapy pioneer Spark Therapeutics", "Roche concludes acquisition of Spark Therapeutics, Inc. to strengthen presence in gene therapy", "Roche concludes acquisition of Spark Therapeutics, Inc. To strengthen presence in gene therapy", "Spark Co-Founder Katherine High Departs Company Ahead of Merger with Roche", "Spark Therapeutics Announces Departure of CEO and Founder Jeff Marrazzo; COO Ron Philip Named as Successor Spark Therapeutics", "FDA approves novel gene therapy to treat patients with a rare form of inherited vision loss", "Pfizer Initiates Pivotal Phase 3 Program for Investigational Hemophilia B Gene Therapy", "Spk-9001: Adeno-Associated Virus Mediated Gene Transfer for Hemophilia B Achieves Sustained Mean Factor IX Activity Levels of >30% without Immunosuppression", "Pfizer begins late-stage testing of Spark's hemophilia B gene therapy", "Spark's gene therapy data answer some burning questions and raise a few more", "Spark Therapeutics shares lose a third of their value as hemophilia gene therapy trial disappoints investors", "Spark's meteoric rise from hospital-funded spinout to $4.8 billion deal", https://en.wikipedia.org/w/index.php?title=Spark_Therapeutics&oldid=1102109041, This page was last edited on 3 August 2022, at 11:43. 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. 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. Out of these, 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. 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). Volastra Therapeutics Feb 2022 - Present 10 . Spark Therapeutics is developing SPK-8011, an investigational gene therapy for hemophilia A, or factor VIII deficiency. [1], Voretigene neparvovec, marketed under the tradename Luxturna, is a gene therapy approved by the Food and Drug Administration for treatment of Leber's congenital amaurosis, a rare genetic eye disease. Pipeline Prospector delivers free access to a database of drugs under clinical trials which made headlines done by Spark Therapeutics, Inc Website http://www.sparxbio.com Industries. The company now operates as a wholly owned subsidiary of Roche with a pipeline of gene . [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. Spark retains global commercialization rights to its SPK-FVIII program. 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. Other uncategorized cookies are those that are being analyzed and have not been classified into a category as yet. Corporate Governance. Such a high price tag, coupled with Luxturna being billed as a one-time treatment . 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. Indicated for a rare eye disease, Spark's therapy carries a list price of $425,000 per injection, or $850,000 for each patient. Spark is currently trading below the perceived value of SPK-RPE65 and the company. But opting out of some of these cookies may have an effect on your browsing experience. Our pipeline includes investigational next-generation therapies for a range of cancers including prostate, ovarian, pancreatic, lung, breast. 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. 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. 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). Pipeline. Develop connectors for ETL pipeline to Arcadia data lake . High, Jeffrey Marrazzo, and Steven Altschuler[2] in an effort to commercially develop treatments against haemophilia that High was working on at Children's Hospital of Philadelphia. 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. Its pipeline includes a product candidate targeting choroideremia (CHM), which is in a Phase I/II clinical . Spark Therapeutics is funded by 7 investors. This website uses cookies to improve your experience while you navigate through the website. In July 2018, Spark Therapeutics transitioned its ongoing, open-label Phase 1/2 trial of fidanacogene elaparvovec to Pfizer. But opting out of some of these cookies may affect your browsing experience. Glunomab / Glunozumab monoclonal antibody, a groundbreaking mechanism of action targeting the blood-brain barrier: In the pathophysiology of neurological diseases such as stroke, multiple sclerosis, Parkinson's disease as well as many other neurodegenerative disorders, one protease called tissue plasminogen activator (tPA) is triggering off . Stock Information. Spark Therapeutics is registered under the ticker NASDAQ:ONCE . 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. In December 2019, the company was acquired by Hoffmann-La Roche for $4.3 billion. Funds will be used to advance the company's deep pipeline of gene therapy programs including development of its lead Phase 3 program to address RPE65-related retinal dystrophies, as well as support the company's growth over the next three years. This cookie is set by GDPR Cookie Consent plugin. This website uses cookies and similar technologies to optimize and improve the experience on our site (. 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. Spark Therapeutics, Inc. . 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. 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. Spark Therapeutics is working to address a range of debilitating genetic diseases. We are a vertically integrated, clinical stage gene therapy company with six ongoing clinical programs and a broad pipeline of preclinical and research . Spark Therapeutics, Inc. Biotechnology Research Philadelphia, PA 62,450 followers We don't follow footsteps. Contents 1 History 2 Products and pipeline 2.1 Voretigene neparvovec 2.2 Fidanacogene elaparvovec 2.3 SPK-8011 2.4 SPK-7001 2.5 SPK-3006 2.6 SPK-1001 3 References 4 External links 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. [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. Our mission at Spark Therapeutics is to challenge the inevitability of genetic disease by discovering, developing and delivering potential treatments in ways unimaginableuntil now. 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. We are also developing SPK-3006, previously SPK-GAA, an investigational gene therapy for the potential treatment of Pompe disease. 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. 99 Hayden Avenue Suite 120, Building E Lexington, MA 02421 These cookies do not store any personal information. 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 have built a fully integrated company, combining our proprietary adeno-associated viral (AAV) gene therapy platform with excellence in R&D, manufacturing and . Analytical cookies are used to understand how visitors interact with the website. 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. How it navigates the commercial landscape will be eyed by a slate of biotechs hoping to bring other one-time gene treatments to market. This website uses cookies to improve your experience while you navigate through the website. 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. "With our combination of industry-leading gene therapy expertise, deep pipeline, and strong clinical results to-date, we've been able to attract a diverse set of blue-chip investors, giving us capital to maintain our position as a leader in the gene therapy field," said Jeffrey D. Marrazzo, co-founder and CEO of Spark Therapeutics. SPK-9001, a lead product candidate in the SPK-FIX program for hemophilia B, is being developed in partnership with Pfizer. Meet the Spark corporate management team About Compliance and Ethics Industry recognitions Gene therapy clinical trial pipeline constitutes 250+ key companies continuously working towards developing 300+ gene therapies, analyzes DelveInsight . [15][16], SPK-7001 is an experimental drug under investigation for treatment of choroideremia, a genetic disorder that causes blindness. Stargardt Disease [13] In July 2018, fidanacogene elaparvovec entered late stage clinical trials. 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. Grants and support are subject to review and approval. 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. 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. 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. The Company focuses on treating orphan diseases. Necessary cookies are absolutely essential for the website to function properly. CHOP's participation in this round brings its total equity investment in Spark to more than $30 million. 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. See what kind of people work at Spark Therapeutics, career paths working at Spark Therapeutics, company culture, salaries, employee political affiliation, and more. 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. These cookies will be stored in your browser only with your consent. 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. Annual Reports. Spark made history in 2017 when it won FDA approval to market . Spark Therapeutics had about . Mr. Philip currently leads the organization. Under the terms of the licensing agreement, Novartis will pay Spark Therapeutics $105 million in cash as an upfront fee. [17], SPK-1001 is an experimental drug under investigation for treatment of Batten disease, a fatal genetic, nervous system disorder. 32 open jobs for Pipeline engineer.

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