Spark Therapeutics Appoints Former Chief of U.S. Food and Drug Administration's Gene Therapy Branch as its Head of Regulatory Affairs

PHILADELPHIA, Nov. 25, 2014 /PRNewswire/ -- Spark Therapeutics, a late-stage gene therapy company developing treatments for debilitating, genetic diseases, announced today the appointment of Daniel M. Takefman, Ph.D., as head of regulatory affairs. Dr. Takefman previously served as chief of the gene therapy branch of the Center for Biologics Evaluation and Research (CBER) at the U.S. Food and Drug Administration (FDA), a position he held since 2006.

"We are honored that Dr. Takefman has made the difficult decision to leave FDA after 15 years of service to join Spark," said Katherine High, M.D., co-founder, president and chief scientific officer of Spark Therapeutics. "I have had the privilege of working with Dr. Takefman in a number of settings, including in his role as a reviewer at FDA, and I have always been impressed with his unwavering commitment to patient safety, his broad and deep knowledge of the gene therapy field and his balanced and practical approach to problem-solving. His experience will be invaluable to Spark as we advance our broad pipeline of gene therapy product candidates."

Dr. Takefman began his career at FDA as a Postdoctoral Fellow in 1999, and became a staff fellow the following year. He served as a microbiologist at FDA from 2001 to 2006 before being named chief of the gene therapy branch in 2006. As Chief, Dr. Takefman had oversight of the chemistry, manufacturing and control review process of numerous gene therapy and therapeutic cancer vaccine products.

"It was a privilege and an honor to have worked at FDA and have helped develop the approaches the agency is taking in its regulation of gene therapy," said Dr. Takefman. "I hope to further the field through my involvement with Spark and its various gene therapy programs. The team assembled at Spark across research, development and manufacturing provides a strong foundation for making gene therapy products a reality for patients. This is groundbreaking work and Spark is a leading company in the field."

Among his many accomplishments at FDA, Dr. Takefman oversaw the development of key regulatory documents, including:

• Draft Guidance for Industry: Design and Analysis of Shedding Studies for Virus or Bacteria-Based Gene Therapy and Oncolytic Products (2014);
• Draft Guidance for Industry: Determining the Need for and Content of Environmental Assessments for Gene Therapies, Vectored Vaccines and Related Recombinant Viral and Microbial Products (2014); and
• Guidance for Industry: Potency Measurements for Cell and Gene Therapy Products (2011).

Dr. Takefman has received numerous FDA honors and awards such as the FDA Commissioner's Special Citation (2013), FDA Technical Excellence Award (2013) and CBER Managerial Excellence Award (2009).

For more information on Spark and its pipeline of gene therapy candidates, including its Phase 3 program for a rare form of inherited retinal dystrophy, please visit www.sparktx.com.

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. Spark's initial focus is on treating orphan diseases where no, or only palliative therapies, exist. Spark's most advanced product candidate, SPK-RPE65, is in a fully-enrolled pivotal Phase 3 clinical trial for the treatment of a rare blinding condition. Spark plans to leverage its experience with SPK-RPE65 to address a broad spectrum of blinding conditions, and also has established a pipeline of gene therapy candidates to treat hematologic and neurodegenerative disorders. 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. To learn more, visit www.sparktx.com.

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SOURCE Spark Therapeutics

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