WASHINGTON, May 19, 2014 /PRNewswire/ -- Spark Therapeutics, a late-stage company developing gene-based medicines for a wide range of debilitating diseases, announced today that co-founder and scientific advisor Katherine High, M.D.; co-founder, president and CEO Jeffrey D. Marrazzo; and senior vice president of business development Carol Greve-Philips will each present at the American Society of Gene & Cell Therapy's (ASGCT) 2014 Annual Meeting.
Spark's team led the advancement of gene therapy research for nearly two decades—from designing and manufacturing vectors to conducting studies that have shown strong potential for safety and efficacy, building a recognized center of gene therapy innovation in research, translation and manufacturing. The company has a robust pipeline of clinical and preclinical gene therapy programs, and is currently advancing the only ongoing Phase 3 gene therapy study in the U.S. to address inherited retinal dystrophies caused by mutations of the RPE65 gene.
- Katherine High, M.D. will present "Gene Therapy for the Hemophilias," as part of the ASGCT Scientific Symposium 104: Gene & Cell Therapy of Genetic and Metabolic Diseases at 8:30 AM ET on Wednesday, May 21, 2014, in Washington 4 at the Marriott Wardman Park in Washington, DC. Kathy has dedicated her career to unraveling the molecular basis of hemophilia and her pioneering work to develop gene therapy as a treatment for the disease led to the advancement of new treatment modalities.
- Jeffrey D. Marrazzo will participate in a panel discussion that illuminates the path to translate groundbreaking scientific discoveries into effective treatments for patients. Topics for discussion include sources of funding to support translational research, clinical development needs, licensing products under development, and partnership considerations. The panel, "Perspective from Private Sector Leaders: What We Have and What We Need," will take place at 7:30 PM ET on Wednesday, May 21, 2014 in Delaware AB at the Marriott Wardman Park in Washington, DC.
- Carol Greve-Philips will present her talk, "Hemophilia: Building the Business Cases for Multiple Entrants," at 3:40 PM ET on Tuesday, May 20, 2014, in Washington 4 at the Marriott Wardman Park in Washington, DC. Carol previously served as vice president of corporate development at Genzyme and brings more than 15 years of perspective on the unique market dynamics associated with small patient populations.
About Spark Therapeutics
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. Spark's lead gene therapy candidate, for RPE65-related blindness, is currently in Phase 3 clinical trials with the potential to be the first approved gene therapy in the U.S., and the first treatment to address the significant unmet needs of patients living with blindness due to inherited retinal dystrophies.
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. In addition to the Phase 3 program in RPE65-related blindness, the company has a Phase 1/2 program in hemophilia B and preclinical programs to address neurodegenerative diseases and other inherited retinal dystrophies and hematologic disorders. Spark has rights to a proprietary manufacturing platform that has an unparalleled track record of success in supporting clinical studies across diverse therapeutic areas and routes of administration. The company's expertise across research, clinical, regulatory and manufacturing builds on a legacy of innovation and excellence in gene therapy established by Spark's team while at The Children's Hospital of Philadelphia Center for Cellular and Molecular Therapeutics. To learn more visit www.sparktx.com.
About the American Society of Gene & Cell Therapy (ASGCT)
The American Society of Gene & Cell Therapy (ASGCT) is a professional nonprofit medical and scientific organization dedicated to the understanding, development and application of genetic and cellular therapies and the promotion of professional and public education in the field. For more information on ASGCT, visit its website, www.asgct.org.
For information contact:
Feinstein Kean Healthcare
SOURCE Spark Therapeutics