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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

Key Statistics

Ownership: Public

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


Company News
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
The Children's Hospital of Philadelphia Participates In Spark Therapeutics (ONCE) Public Offering 12/16/2015 10:57:07 AM
Spark Therapeutics (ONCE) Announces December Conference Participation 12/1/2015 8:03:34 AM
Spark Therapeutics (ONCE) Announces Filing Of Registration Statement For Proposed Public Offering 11/30/2015 9:38:10 AM
Spark Therapeutics (ONCE) Announces Presentation Of Additional Phase 3 Data On SPK-RPE65 At American Academy of Ophthalmology 2015 Annual Meeting 11/16/2015 9:20:21 AM
Spark Therapeutics (ONCE) Announces SPK-TPP1, Its First Program Targeting Neurodegenerative Disease 11/12/2015 9:29:49 AM
Spark Therapeutics (ONCE) Expands Into Boston Following One of the Biggest IPOs of the Year 11/4/2015 2:32:56 PM