Spark Therapeutics

Spark Therapeutics, Inc. is a developer of gene therapy treatments, which treat debilitating genetic diseases.[1] It is a subsidiary of Hoffmann-La Roche.

Spark Therapeutics, Inc.
Subsidiary of Hoffmann-La Roche
IndustryBiotechnology
Pharmaceutical
Founded2013 (2013)
FoundersKatherine A. High
Jeffrey Marrazzo
Jean Bennett
J Fraser Wright
Beverly Davidson
Jennifer Wellman
HeadquartersPhiladelphia, Pennsylvania
Revenue $64 million (2018)
-$78 million (2018)
Total assets $814 million (2018)
Total equity $496 million (2018)
Number of employees
368 (2019)
ParentHoffmann-La Roche
Websitesparktx.com
Footnotes / references
[1]

History

The company was founded in 2013 by Katherine A. High, Jeffrey Marrazzo, and Steven Altschuler[2] in an effort to commercially develop treatments against haemophilia that High was working on at Children's Hospital of Philadelphia.[3]

In January 2015, the company became a public company via an initial public offering.[4]

In December 2017, the U.S. Food and Drug Administration approved LUXTURNATM (voretigene neparvovec-rzyl) for the treatment of patients with viable retinal cells and confirmed biallelic RPE65 mutation-associated retinal dystrophy, a genetic blinding condition caused by mutations in the RPE65 gene.[1]

In December 2019, the company was acquired by Hoffmann-La Roche for $4.3 billion.[5][6]

In February 2020, Spark co-founder Katherine A. High stepped down from the company after the acquisition by Swiss pharma Roche.[7]

Products and pipeline

The company has 3 gene therapy product candidates in clinical development: (i) SPK-8011, a candidate in the SPK-FVIII program for hemophilia A; (ii) SPK-8016, a product candidate for the hemophilia A inhibitor market; and (iii) SPK-7001, targeting choroideremia, or CHM. SPK-9001, a lead product candidate in the SPK-FIX program for hemophilia B, is being developed in partnership with Pfizer.[1]

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.[8]

Fidanacogene elaparvovec

Fidanacogene elaparvovec, previously known by its study ID number SPK-9001,[9] is an experimental drug under investigation for treatment of hemophilia B in partnership with Pfizer. 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.[10] In July 2018, fidanacogene elaparvovec entered late stage clinical trials.[11]

SPK-8011

SPK-8011 is an experimental drug under investigation for treatment of Haemophilia 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 that the responses could be controlled with steroids, and promised to move forward with Phase III testing.[12][13]

SPK-7001

SPK-7001 is an experimental drug under investigation for treatment of choroideremia, a genetic disorder that causes blindness.[14]

SPK-3006

SPK-3006 is an experimental drug under investigation for treatment of Pompe disease, a genetic disorder that leads to failure to correctly metabolize glycogen.[14]

SPK-1001

SPK-1001 is an experimental drug under investigation for treatment of Batten disease, a fatal genetic, nervous system disorder.[14]

gollark: We were looking at replacing it with superconductors, but the liquid helium looked like it would have been expensive.
gollark: My house uses aluminum power cabling, not foolish aluminium.
gollark: You may be overestimating the carrying capacity of pigeons.
gollark: You *can*, at great cost, get 1TB SD cards. Pigeons could carry those more easily than flash sticks.
gollark: So flash.

References
  1. "Spark Therapeutics, Inc. 2018 Form 10-K Annual Report". U.S. Securities and Exchange Commission.
  2. Higgins, Robert F., and Tina Liu. "Spark Therapeutics: Pioneering Gene Therapy." Harvard Business School Case 818-059, January 2018.
  3. Crow, David (19 October 2017). "Gene therapy helped these children see. Can it transform medicine?". Financial Times.
  4. George, John (January 30, 2015). "Shake Shack wasn't the day's only gonzo IPO. And this one, for Spark Therapeutics, raised more cash". American City Business Journals.
  5. "Roche completes $4.3B purchase of Philadelphia gene therapy pioneer Spark Therapeutics". American City Business Journals. 17 December 2019.
  6. "Roche concludes acquisition of Spark Therapeutics, Inc. to strengthen presence in gene therapy" (Press release). Hoffmann-La Roche. 17 December 2019.
  7. "Spark Co-Founder Katherine High Departs Company Ahead of Merger with Roche". BioSpace. Retrieved 2020-03-03.
  8. "FDA approves novel gene therapy to treat patients with a rare form of inherited vision loss" (Press release). Food and Drug Administration. 19 December 2017.
  9. "Pfizer Initiates Pivotal Phase 3 Program for Investigational Hemophilia B Gene Therapy" (Press release). Pfizer. 16 July 2018.
  10. 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. doi:10.1182/blood.V128.22.3.3.
  11. George, John (16 July 2018). "Pfizer begins late-stage testing of Spark's hemophilia B gene therapy". American City Business Journals.
  12. Garde, Damian (7 August 2018). "Spark's gene therapy data answer some burning questions — and raise a few more". Stat.
  13. Tirrell, Meg (7 August 2018). "Spark Therapeutics shares lose a third of their value as hemophilia gene therapy trial disappoints investors". CNBC.
  14. Morrison, Chris (5 March 2019). "Spark's meteoric rise from hospital-funded spinout to $4.8 billion deal". Nature Biotechnology.
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