ASH 2013: Spark Therapeutics Co-Founder and Gene Therapy Pioneer Dr. Katherine A. High Outlines Challenges and Promises for Gene Therapy

PHILADELPHIA, Dec. 9, 2013 /PRNewswire/ -- Spark Therapeutics, a fully integrated, late-stage gene therapy company, today announced that its co-founder and scientific advisor, Dr. Katherine A. High, accepted the E. Donnall Thomas Lecture Prize and presented a lecture on her career's work to develop novel genetic therapies at the 55^th American Society of Hematology (ASH) Annual Meeting and Exposition in New Orleans. Named for the late Nobel Prize laureate and past ASH president, E. Donnall Thomas, M.D., the lectureship and prize recognizes pioneering research achievements in hematology that have propelled the field forward.

As a leader in the development of gene therapy, Dr. High has dedicated her career to bringing curative treatments to patients with debilitating, genetic disorders. Her work has ranged from basic biochemical investigations of clotting factors, virology of the AAV viruses as relevant to vector design, dissection of the human immune response to vector and transgene proteins, development of predictive large animal models, and careful and highly instructive clinical trials. Dr. High's more than two decades of investigation regarding the promise of gene therapy has culminated in groundbreaking, positive results in clinical studies of patients with hemophilia B and inherited blindness.

As part of her presentation to the scientific community at ASH, Dr. High outlined new opportunities for gene therapy, including advancements of gene therapy as an effective treatment modality. These include highlights from Dr. High's recent research which found that humoral immunity to adeno-associated virus (AAV) vectors, a barrier to effective gene expression, can be overcome by using capsid decoys that "distract" neutralizing antibodies so the gene therapy vector can effectively correct the defect gene—potentially expanding the number of patients who could benefit from treatment with gene therapy. The findings are summarized in a paper titled, "Overcoming Preexisting Humoral Immunity to AAV Using Capsid Decoys," and published in Science Translational Medicine in July 2013. 

"No one is more deserving of this recognition than Kathy. A relentless pioneer of gene therapy as a superior alternative to current treatment modalities for many diseases, Kathy has continued to contribute key insights to the field and advance gene-based medicines," said Jeffrey D. Marrazzo, president, CEO and co-founder, Spark Therapeutics. "Her ongoing work is poised to resolve the last remaining hurdles to gene therapy which have challenged the scientific community for decades, giving us a real opportunity to deliver potentially curative, one-time therapy products with the ability to transform patients' lives."

Dr. High currently advises on a variety of gene therapy programs at Spark Therapeutics:

• A Phase 1/2 program in hemophilia B. 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 around the globe.
• A Phase 3 clinical program in blindness due to mutations in the RPE65 gene (RPE65 blindness). This study builds on an earlier clinical study in which 12 patients demonstrated notable improvement in functional vision, moving in some cases from being blind to being able to recognize faces and ambulate independently. All school age patients enrolled in the trial were transferred from Braille classrooms to sighted classrooms.  
• Preclinical programs to address neurodegenerative diseases, and other retinal degenerative diseases and hematologic disorders

For more information on Dr. High's work with Spark Therapeutics, please visit www.sparktx.com or follow on Twitter at www.twitter.com/spark_tx.

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 RPE65 blindness 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.

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SOURCE Spark Therapeutics