spark therapeutics choroideremia

Nightstar's US competitor Spark Therapeutics is in Phase I/II trials for their gene therapy SPK-CHM for choroideremia. It is also. Spark Therapeutics has launched a new resource website for those with IRD's (Inherited Retinal Disorders), including CHM. Based in Philadelphia, Pennsylvania, Spark Therapeutics develops gene therapy products for individuals who suffer from debilitating genetic disorders. The announcement of the initiation of clinical trials is a pivotal point in the CRF's history, and we are proud to have provided funding to Penn for the pre-clinical work that laid the foundation for this achievement. 3737 Market Street Can it transform medicine? We are proud members of the global IRD community, dedicated to developing patient-focused initiatives, from clinical research and educational programs, to programs designed to increase access to genetic screening for people with IRDs. The same clinical study teams that have progressed the companys lead therapeutic, SPK-RPE65, to Phase III, will now be leveraged to conduct the SPK-CHM Phase I/II study. 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. On October 6, friends, families and allies of the Foundation Fighting Blindness (FFB), a nonprofit organization focused on research for preventing and treating blindness caused by inherited retinal diseases filled Philadelphias Independence Hall (famously where the United States Declaration of Independence and the United States Constitution were adopted) to participate in FFBs annual Philadelphia VisionWalk. Chm Associated Choroideremia, supplied by Spark Therapeutics, used in various techniques. "We are thrilled at the potential for Spark to deliver a treatment to patients with CHM to stop progressive vision loss and prevent blindness. spark therapeutics products. Choroideremia Therapies covered in the report include: SPK-7001 (AAV2-hCHM) BIIB111 (AAV2-REP1) 4D-110 And many more.. SPK-7001 is an experimental drug under investigation for treatment of choroideremia, a genetic disorder that causes blindness. Among other invited guests from the FFB, Gordon Gund and his wife Lulie, founders of FFB, along with Tom Scott, the films director, spoke on an interactive panel following the screening. 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's headquarters are in 3737 Market St, Philadelphia, Pennsylvania, 19104, United States What is Spark Therapeutics's phone number? People with conditions like ours talk of trying to build a visual scrapbook, placing our visual memories in our brain, Eric said. Kirby Professor of Ophthalmology at the Perelman School of Medicine, University of Pennsylvania (Penn), have previously demonstrated the ability to restore REP-1 protein production, intracellular trafficking and retinal structure in models of CHM. Cision Distribution 888-776-0942 Spark Therapeutics, Inc. announced it has initiated enrollment of a Phase 1/2 clinical trial of its product candidate, SPK-CHM, for patients with choroideremia . Learn more about our expanded genetic testing panel and terms and conditions of the program. Our mission at Spark Therapeutics is to challenge the inevitability of genetic disease by discovering, developing and delivering potential treatments in ways unimaginableuntil now. 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. The first symptom of this condition is usually an impairment of night vision (night blindness), which can occur in early childhood. 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. Choroidermia is a very rare genetic condition that is characterized by progressive vision loss, predominantly found in men. [17], "Spark Therapeutics, Inc. 2018 Form 10-K Annual Report". The company estimates that CHM affects approximately 12,500 males in the US and five of the major European markets. Spark Therapeutics, Inc. Mr. Philip currently leads the organization. Choroideremia Therapies covered in the report include: SPK-7001 (AAV2-hCHM) BIIB111 (AAV2-REP1) 4D-110 And many more. Bringing the hope of gene therapy research to those living with declining eyesight, blindness, and other vision problems caused by inherited retinal diseases. Additionally, we work with advocacy groups from around the world to help connect patients and families to education, research, support services and each other. 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. Spark's integrated gene therapy platform builds on two decades of research, development and manufacturing at The Children's Hospital of Philadelphia, including human trials conducted across diverse therapeutic areas and routes of administration. It has a pipeline of product candidates targeting multiple rare blinding conditions, hematologic disorders and neurodegenerative diseases. Spark Therapeutics 4D Molecular Therapeutics (4DMT) And many others. SPK-9001, a lead product candidate in the SPK-FIX program for hemophilia B, is being developed in partnership with Pfizer. 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. He has been at it ever since. It was such a relief that there are people for me to talk with.. [17] SPK-3006 [ edit] SPK-3006 is an experimental drug under investigation for treatment of Pompe disease, a genetic disorder that leads to failure to correctly metabolize glycogen. The Company focuses on treating orphan diseases. Spark Therapeutics, Inc. is a developer of gene therapy treatments, which treat debilitating genetic diseases. This website uses cookies and similar technologies to optimize and improve the experience on our site (, LEARN MORE ABOUT OUR APPROACH TO GENE THERAPY RESEARCH. In February 2019, the business announced it would acquire gene therapy company, Spark Therapeutics, for $4.3 billion ($114.50 per share) adding Spark's gene therapy portfolio to its previous acquired assets. Joining the local Philadelphia community, Team Spark participated in one walk to benefit research and support for retinal degenerative diseases and one walk to support the local hemophilia community. in an inter-subject group dose escalation in individuals with choroideremia, based on a comprehensive clinical monitoring plan. Necessary cookies are absolutely essential for the website to function properly. Since 2015, Spark has been working to make the critical diagnostic step of genetic testing more accessible to those living with an IRD. Contact Data Investor Contact: Ryan Asay Ryan.asay@sparktx.com (215) 239-6424 Media Contact: Monique da Silva Monique.dasilva@sparktx.com (215) 282-7470 Gene therapy research is an investigational approach to treat or prevent genetic disease. Eric, living with choroideremia Your inherited retinal disease patient advocacy team Amy Fisher Contact us at patients@sparktx.com Resources for inherited retinal diseases Spark's newest resource Eye Want 2 Know aims to equip those living with an IRD with the knowledge and resources they need to get started with genetic testing. [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. A business executive turned artist, Eric was living on Cape Cod in the early 2000s when he began to train in the style of Monet and Degas in 19th-century France. 3737 Market Street SPK-CHM builds on the experience and technology utilized in the clinical development of Spark's lead Phase 3 program, SPK-RPE65, including the same vector, target cells, and route of administration, as well as the same manufacturing process. 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, Biotechnology companies of the United States, Pharmaceutical companies of the United States, Pharmaceutical companies established in 2013, Biotechnology companies established in 2013, Health care companies based in Pennsylvania, American subsidiaries of foreign companies, Short description is different from Wikidata, Official website different in Wikidata and Wikipedia, Creative Commons Attribution-ShareAlike License 3.0, This page was last edited on 3 August 2022, at 11:43. The pipeline also includes SPK-7001 in an ongoing Phase 1/2 clinical trial for choroideremia. Phone: 1-855-SPARKTX / +1 215-220-9300, Spark Therapeutics at the Bulletin Building in Philadelphia, Spark Therapeutics Announces Departure of CEO and Founder Jeff Marrazzo; COO Ron Philip Named as Successor, Reflecting on my time at Spark: Jeffs message to Team Spark, Spark Therapeutics to Invest $575M in New 500k Square Foot State-of-the-Art Gene Therapy Innovation Center on Drexels University City Campus. from 8 AM - 9 PM ET. It is caused by a mutation in the CHM gene in which it does not produce the gene REP-1, located on the X chromosome. It is mandatory to procure user consent prior to running these cookies on your website. Find out more about how we strive to turn genes into medicines for patients with inherited diseases, including inherited retinal diseases (IRDs), liver-directed diseases such as hemophilia, and neurodegenerative diseases. Learn more about the benefits of testing, our work with the community and the Payor dialogues are under way and a BLA submission is planned for 2016. It is great to have Spark Therapeutics lead this effort and we pledge our continued support as the clinical trials proceed. This category only includes cookies that ensures basic functionalities and security features of the website. Among Spark's top drug hopefuls is SPK-8011, for haemophilia A, expected to start Phase 3 trials in 2019. Roche is acquiring Spark Therapeutics in a multi-billion dollar deal that underscores big pharma's growing appetite for new technologies like gene therapy. With SPK-CHM, Spark is leveraging the experience and technology utilized in the development of its lead Phase 3 program, SPK-RPE65, including the same vector, target cells and route of administration, as well as the same manufacturing process. The pathology is understood to arise from mutations in the CHM gene leading to a defective or absent Rab escort protein-1 (REP-1). Please add some widgets here! [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. Expanding upon an earlier collaboration around SPK-RPE65, in December of 2014 Spark and Penn, through Penn's technology commercialization organization, the Penn Center for Innovation (PCI), entered into an exclusive license agreement to certain Penn-owned intellectual property rights, including assets related to the choroideremia program. At Spark Therapeutics, we don't follow footsteps. flies on dogs' ears home remedies; who has authority over vehicle violations. This website uses cookies to improve your experience while you navigate through the website. For more information on Spark and its pipeline of gene therapy candidates, including its Phase 3 program for rare blinding conditions, please visit www.sparktx.com/pipeline. Spark Therapeutics, Inc. is a gene therapy company. Spark Therapeutics's phone number is (215) 220-9300 What is Spark Therapeutics's stock symbol? "Choroideremia is a rare, blinding condition which affects males most severely at middle age, a critical time personally and professionally," said Jeffrey D. Marrazzo, co-founder and chief executive officer of Spark. The documentary has been shown at film festivals across the country and has earned festival honors at numerous events. We are contributing to the development of gene therapies to treat and cure inherited retinal diseases, such as choroideremia and Stargardt disease. Bioz Stars score: 86/100, based on 1 PubMed citations. The study, an open-label dose escalating safety and preliminary efficacy trial, aims to enrol up to 10 subjects and to begin dosing in the first quarter of FY2015. But opting out of some of these cookies may have an effect on your browsing experience. The disease is characterized by deletions or mutations in the CHM gene, resulting in defective, or absent, Rab escort protein-1 (REP-1). "The SPK-CHM program, for the first time, creates the potential for patients to use their vision for longer and see more things.". Sungazing Praksa. . Ceased Application number GBGB1103062.4A Other languages English (en) Erics art has taken him places near and far from months-long plein-air immersions in the French countryside, to afternoons with his easel in City Park, in his native New Orleans. Collaborator (none) 15 . State-of-the-art, in-house expertise in vector manufacturing, Innovative scientific and regulatory strategies, Strong commitment to improve patient care, This website uses cookies and similar technologies to optimize and improve the experience on our site (. Detailed Description: The primary objective is to evaluate the safety and tolerability of subretinal administration of AAV2-hCHM, in an inter-subject group dose escalation in individuals with choroideremia, based on a comprehensive clinical monitoring plan. Growing up in New Orleans in the 1960s and 70s, Eric, himself, did not meet another person with choroideremia until he was 40 years old, in 1998. France and UK ) and Japan ) choroideremia ( or CHM ), which is in a Phase III trials! Of Spark Therapeutics is proud to be a part of the website clinical monitoring plan are define! Build a visual scrapbook, placing our visual memories in our brain, Eric said and investigating its lead candidate! 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