Spark Therapeutics: Difference between revisions
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===Voretigene neparvovec=== |
===Voretigene neparvovec=== |
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{{Main article|Voretigene neparvovec}} |
{{Main article|Voretigene neparvovec}} |
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Voretigene neparvovec, marketed under the tradename Luxturna, is a gene therapy |
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.<ref>{{cite news|url=https://www.fda.gov/NewsEvents/Newsroom/PressAnnouncements/ucm589467.htm|title=FDA approves novel gene therapy to treat patients with a rare form of inherited vision loss|last=|first=|date=19 Dec 2017|work=[[FDA]]|access-date=20 Dec 2017|archive-url=|archive-date=|dead-url=}}</ref> |
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===SPK-9001=== |
===SPK-9001=== |
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Revision as of 00:05, 21 December 2017
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| Nasdaq: ONCE | |
| Industry | Biotechnology, Pharmaceutical |
| Founded | 2013 in Philadelphia, Pennsylvania, United States of America |
| Founders | Jeffrey Marrazzo, Katherine High |
| Headquarters | , |
| Website | sparktx |
Spark Therapeutics is a startup pharmaceutical company that develops gene therapies.[1][2][3] 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.[4]
Clinical pipeline
Voretigene neparvovec
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.[5]
SPK-9001
SPK-9001 is an experimental drug under investigation for treatment of hemophilia B. Spark partnered with pharmaceutical giant Pfizer to develop SPK-9001.[2] SPK-9001 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.[6]
References
- ^ "Spark Therapeutics reports 3Q loss". Associated Press. 7 November 2017. Retrieved 16 November 2017.
- ^ a b 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.
{{cite news}}: Cite has empty unknown parameter:|dead-url=(help) - ^ 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.
External links
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