Spark Therapeutics Launched with $50 Million in Financing to Advance Late- and Mid-Stage Gene Therapy Programs with Clinical Proof of Concept

PHILADELPHIA, Oct. 22, 2013 /PRNewswire/ -- Spark Therapeutics, a new, fully integrated company developing gene-based medicines for a wide range of debilitating diseases, announced today it has launched with a $50 million capital commitment from The Children's Hospital of Philadelphia (CHOP) to advance and commercialize multiple ongoing programs with clinical proof of concept.

Spark will assume control over two gene therapy clinical trials: a Phase 3 study for inherited blindness caused by mutations of the RPE65 gene and a Phase 1/2 study for hemophilia B. The company is also advancing toward the clinic with gene therapy programs to address neurodegenerative diseases and additional hematologic disorders and other forms of inherited blindness.

"The creation of Spark is the culmination of a decade-long commitment by CHOP and our founding team to drive the field of gene therapy forward during a time when many in the industry had moved away," said Jeffrey D. Marrazzo, co-founder, president and chief executive officer of Spark Therapeutics. "Their vision and long-term dedication have enabled us to effectively address many of the key challenges facing the field and to emerge with one of the industry's most robust clinical-stage gene therapy pipelines; as well as exclusive rights to commercialize a proprietary manufacturing platform, supply from a world-class manufacturing facility and a founding team with a proven track record of executing safe and effective gene therapy trials for nearly two decades. We are working with great urgency and care to deliver gene therapy products with the potential to transform the lives of those affected by severe genetic diseases."

Spark builds on the work of CHOP's Center for Cellular and Molecular Therapeutics (CCMT), established in 2004 as a world-class center for gene therapy translational research and manufacturing. Many of the CCMT's leaders will assume management roles within Spark or engage with the company as scientific advisors, including Katherine A. High, M.D, a gene therapy pioneer who has served as the director of the CCMT since its inception.

"Gene-based medicines are among the most complex therapeutics ever developed," said Dr. High. "We at CCMT have persevered through more than a decade of scientific and clinical development and are now closer than ever to realizing the ambitious vision of one-time, potentially curative therapies to address serious genetic conditions. The team at Spark has incredible goals for the treatment of diseases including hemophilia B and inherited blindness, and we look forward to working with them to deliver groundbreaking new treatments to patients in need."

Spark has entered into agreements with multiple academic institutions to assemble the technology, programs and capabilities needed to deliver its pioneering gene therapy products. Notably, Spark has exclusive rights to commercialize CHOP's proprietary manufacturing technology and will use clinical-grade gene therapy vectors produced by the CCMT's state of the art good manufacturing practices (cGMP) clinical facility.

Pioneers in AAV delivery   
Over the past two decades, the Spark leadership team has developed unrivaled expertise in the design, manufacturing and delivery of gene therapies using adeno-associated virus (AAV) vectors. AAV has been demonstrated in clinical studies to be a safe and effective vehicle for the delivery of genetic material into targeted cells and provides unique advantages over alternative delivery approaches. The Spark team was among the first to demonstrate human clinical proof of concept in two distinct organ systems—the eye and the liver—establishing a strong foundation for the company's current programs, and has clinical experience in 15 studies across diverse genetic and non-genetic diseases and five distinct routes of administration. 

Spark's most advanced clinical program is a Phase 3 study to address blindness caused by mutations in the RPE65 gene. There is currently no pharmacologic treatment for this form of inherited retinal degeneration, which ultimately causes irreversible blindness.

The open-label, randomized, controlled study builds on an earlier clinical study in which 12 patients with RPE65-related blindness demonstrated notable improvement in visual function, moving in some cases from being profoundly blind to being able to recognize faces and ambulate independently. All school-age patients enrolled in the trial were able to transfer from Braille classrooms to sighted classrooms. 

In hemophilia B, the company's Phase 1/2 study is enrolling up to 15 subjects who will receive a one-time dose through an intravenous infusion of the gene responsible for expressing factor IX. The open-label, dose escalation study is designed to assess safety and effectiveness of gene therapy in inducing the body to create native factor IX, as measured in their blood. If successful, gene therapy in hemophilia B would eliminate or reduce the need for regular infusions of clotting factor, enabling physicians to provide comprehensive, accessible treatment to all hemophilia B patients, potentially including those with inhibiting antibodies.

This study builds on the Spark team's previous groundbreaking work in advancing the first two gene therapy clinical studies in hemophilia B, the second of which achieved the highest levels of factor IX expression of any gene therapy trial to date.

Leading expertise  
Spark's founding team includes gene therapy scientists and experts in clinical development and manufacturing who have been at the forefront of the effort to advance gene therapy as a viable treatment paradigm for debilitating, incurable diseases. Spark's team has more than 120 years of combined experience addressing the clinical, regulatory and manufacturing issues related to gene-based medicines.   

• Jean Bennett, M.D., Ph.D., scientific co-founder and advisor, has more than 20 years' experience researching inherited retinal conditions and led the first team to demonstrate preclinical proof of principle in ocular gene therapy.
• Beverly Davidson, Ph.D., scientific co-founder and advisor, has worked for nearly 20 years in developing gene therapies to address genetic diseases that cause central nervous system dysfunction.
• Bernd Hauck, Ph.D., an advisor on clinical vector operations, has served as the assistant director of production at the CCMT vector manufacturing facility, establishing standard operating procedures for the AAV vector manufacturing process.
• Katherine A. High, M.D., scientific co-founder and advisor, is a Howard Hughes Medical Institute investigator, director of the CCMT, was elected a fellow of the American Association for the Advancement of Science based on "distinguished contributions in the field of human gene therapy," and is a former president of the American Society of Gene & Cell Therapy (ASGCT).
• Jeffrey D. Marrazzo, M.B.A., M.P.A., co-founder, president and CEO, is an experienced entrepreneur dedicated to curing disease through genomic medicine and has held leadership roles at several successful molecular and cellular medicine companies including Generation Health, MolecularHealth, Inc., and Tengion.
• Guang Qu, Ph.D., M.S., vice president of process development, has over 15 years' working experience in the gene therapy field, including six years in the biotechnology industry directing process development at Avigen, Inc.
• Junwei Sun, M.S., an advisor on R&D operations, has served as the administrative director for the CCMT since its inception.
• Jennifer Wellman, vice president of regulatory affairs, has 13 years of R&D and regulatory experience specifically related to AAV-mediated gene therapy and has spent the past eight years at the CCMT where she directed regulatory interactions for the Center's gene therapy studies. She previously worked at Avigen, Inc. 
• J. Fraser Wright, Ph.D., scientific co-founder and chief technology advisor, is a leading expert in clinical vector production and characterization and has directed the CCMT's vector manufacturing since its establishment. He has overseen investigational product chemistry, manufacturing and controls for gene therapy vectors administered to more than 100 human subjects in 12 clinical studies.

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. The 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. Our robust pipeline includes a Phase 3 program in blindness due to mutations in the RPE65 gene and a Phase 1/2 program in hemophilia B, as well as preclinical programs to address neurodegenerative diseases and other retinal degenerative diseases 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.

Media Inquiries:  
Jessica Rowlands 
202-729-4089 
[email protected]  

SOURCE Spark Therapeutics