|Traded as||NASDAQ: ONCE|
Russell 2000 Component
|Founded||2013Philadelphia, Pennsylvania, United States of Americain|
|Founders||Jeffrey Marrazzo, Katherine High|
Spark Therapeutics is a startup pharmaceutical company that develops gene therapies. It was founded in 2013 by Katherine High and Jeffrey Marrazzo in and effort to commercially develop treatments against haemophilia that High was working on at Children's Hospital of Philadelphia.
Products and pipeline
Voretigene neparvovec, marketed under the tradename Luxturna, is a gene therapy approved by the Food and Drug Administration for treatment of Leber's congenital amaurosis, a rare genetic eye disease.
Fidanacogene elaparvovec, previously known by its study ID number SPK-9001, is an experimental drug under investigation for treatment of hemophilia B. Spark partnered with pharmaceutical giant Pfizer to develop fidanacogene elaparvovec. Fidanacogene elaparvovec is an adeno-associated viral vector which is designed to transfer a working copy of the Factor IX gene into the livers of patients who carry non-functioning copies. In July, 2018 Pfizer said fidanacogene elaparvovec was entering late stage clinical trials.
SPK-8011 is an experimental drug under investigation for treatment of hemophilia A. It is entering phase III clinical trials in the United States. The therapy transfers a working copy of the Factor VIII gene into patients who lack one. In Phase II clinical trials 2 of 7 patients receiving the highest dose of the drug suffered immune responses. One patient had to be hospitalized. The reactions against the treatment were seen as a set back, though Spark suggested the responses could be controlled with steroids and promised to move forward with Phase III testing.
- "Spark Therapeutics reports 3Q loss". Associated Press. 7 November 2017. Retrieved 16 November 2017.
- George, John (7 November 2017). "Spark, Pfizer amend agreement for experimental hemophilia gene therapy". Philadelphia Business Journal. Retrieved 16 November 2017.
- Marchione, Marilynn (10 October 2017). "A gene therapy that could cure blindness is on the brink of getting approved". Business Insider. Retrieved 16 November 2017.
- Crow, David (19 October 2017). "Gene therapy helped these children see. Can it transform medicine?". Financial Times. Retrieved 16 November 2017.
- "FDA approves novel gene therapy to treat patients with a rare form of inherited vision loss". FDA. 19 Dec 2017. Retrieved 20 Dec 2017.
- "Pfizer Initiates Pivotal Phase 3 Program for Investigational Hemophilia B Gene Therapy" (Press release). Pfizer. 16 July 2018. Retrieved 2018-07-18.
- Lindsey, George (1 December 2016). "Spk-9001: Adeno-Associated Virus Mediated Gene Transfer for Hemophilia B Achieves Sustained Mean Factor IX Activity Levels of >30% without Immunosuppression". Blood. 128 (22): 3. Retrieved 16 November 2017.
- George, John (16 July 2018). "Pfizer begins late-stage testing of Spark's hemophilia B gene therapy". Philadelphia Business Journal. Retrieved 18 July 2018.
- Garde, Damian (7 August 2018). "Spark's gene therapy data answer some burning questions — and raise a few more". Stat. Retrieved 7 August 2018.
- Tirrell, Meg (7 August 2018). "Spark Therapeutics shares lose a third of their value as hemophilia gene therapy trial disappoints investors". CNBC. Retrieved 7 August 2018.
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