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Company Profile


Spark Therapeutics 

3737 Market Street
Suite 1300
Philadelphia  Pennsylvania  10104  U.S.A.
Phone: 888-772-7560 Fax: n/a


Spark Therapeutics is developing curative, one-time gene therapy products with the potential 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 United States, and the first approved treatment to address the significant unmet needs of patients living with blindness due to inherited retinal dystrophies.

Additionally, the company has clinical and preclinical programs in other inherited retinal dystrophies and hematological disorders, and a proprietary manufacturing platform that has successfully supported human gene therapy trials across diverse therapeutic areas and routes of administration. 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.

Spark is based in Philadelphia, PA and was spun out of The Children's Hospital of Philadelphia in October 2013. In May 2014, Spark announced the completion of a $72.8 million Series B financing led by Sofinnova Ventures, bringing the company's total funding raised to $82.8 million to-date. To learn more about Spark visit




Founders: Jeffrey Marrazzo, J. Fraser Wright and Katherine High

CEO: Jeffrey Marrazzo

CTO: J. Fraser Wright

CSO (Scientific): Katherine High


Please click here for Spark Therapeutics job opportunities.


Please click here for clinical trial information.


Key Statistics

Ownership: Public

Web Site: Spark Therapeutics
Employees: n/a
Symbol: ONCE


Company News
Spark Therapeutics (ONCE): The Next Big Thing In Gene Therapy? 5/26/2017 5:49:40 AM
Massachusetts' Selecta (SELB) Wrangles $890 Million+ R&D Deal Out of Spark Therapeutics (ONCE) 12/5/2016 5:44:25 AM
Biogen (BIIB) Taps Former Spark Therapeutics (ONCE) Exec and a Finance Vet for Hemophilia Spin-off Company Bioverativ 11/15/2016 6:24:42 AM
Spark Therapeutics (ONCE) Touts Long-Term Positive Phase III Gene Therapy Data 8/11/2016 6:45:21 AM
Shire (SHPG) Kills Baxalta (BXLT)'s Hemophilia B Program; Clears Path for BioMarin (BMRN), Spark Therapeutics (ONCE) and uniQure (QURE) 8/3/2016 7:26:00 AM
FDA Calls Pfizer (PFE), Spark Therapeutics (ONCE)'s Hemophilia B Drug a Breakthrough 7/22/2016 7:09:00 AM
Spark Therapeutics (ONCE), Pfizer (PFE)'s Hemophilia B Gene Therapy Candidate Shows Promise in Early Studies 5/19/2016 6:35:37 AM
Spark Therapeutics (ONCE) Snaps Up Genable Tech in $15 Million Cash-Stock Deal 3/7/2016 7:34:07 AM
Spark Therapeutics (ONCE)' Ambitious Goals Include 10 Gene Therapy Programs by 2018 1/13/2016 6:45:52 AM
The Children's Hospital of Philadelphia Participates In Spark Therapeutics (ONCE) Public Offering 12/16/2015 11:10:13 AM