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  • Transforming Lives in a Single Dose

    Yannick, 13, was blind before receiving gene therapy, which restored much of his sight.

  • Leadership to Make Gene Therapy a Reality

    Dr. Katherine High has pioneered the development of gene therapy for two decades.

  • Capabilities to Re-Imagine What’s Possible

    Gene therapy is giving Michael, 67, new hope for a life free from hemophilia B.

  • Expanding Life Sciences in Philadelphia

    Spark to open permanent west Philadelphia headquarters.

About Spark Therapeutics

We are a leader in the field of gene therapy seeking to transform the lives of patients suffering from debilitating genetic diseases by developing one-time, life-altering treatments. Our initial focus is on treating rare diseases where no, or only palliative therapies, exist.

Spotlight

Spark initiates a Phase 1/2 clinical trial of SPK-CHM, its product candidate for the potential treatment of patients with choroideremia (CHM), a rare form of night blindness, expanding the company’s pipeline of potential treatments for rare, blinding conditions.

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