Spark_Therapeutics

Spark Therapeutics

Spark Therapeutics

American pharmaceutical company

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

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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, trading under the ticker $ONCE via a $161 million initial public offering[4] led by Chief Legal Officer Joseph La Barge.[5]

In December 2017, the U.S. Food and Drug Administration approved Luxturna (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] The company is currently developing several gene therapies to target a suite of diseases, including Haemophilia A and B, and several central nervous system diseases.

In December 2019, the company was acquired by Hoffmann-La Roche for $4.3 billion.[6][7] It now continues to operate as an independent subsidiary. [8] Since the acquisition by Swiss pharma Roche, several key founding executives have departed, including scientist and co-founder Katherine High in February 2020,[9] Chief Business/Legal Officer Joseph La Barge in December 2021, and co-founder and Chief Executive Officer Jeffrey Marrazzo in April 2022 [10]

On February 23, 2022, Marrazzo named big-Pharma veteran Ron Philip as his successor. Mr. Philip currently leads the organization.[10]

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.[11]

Fidanacogene elaparvovec

Fidanacogene elaparvovec, previously known by its study ID number SPK-9001,[12] 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.[13] In July 2018, fidanacogene elaparvovec entered late stage clinical trials.[14]

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.[15][16]

SPK-7001

SPK-7001 is an experimental drug under investigation for treatment of choroideremia, a genetic disorder that causes blindness.[17]

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.[17]

SPK-1001

SPK-1001 is an experimental drug under investigation for treatment of Batten disease, a fatal genetic, nervous system disorder.[17]

References

  1. [1]
    "Spark Therapeutics, Inc. 2018 Form 10-K Annual Report". U.S. Securities and Exchange Commission.
  2. [2]
    Higgins, Robert F., and Tina Liu. "Spark Therapeutics: Pioneering Gene Therapy." Harvard Business School Case 818-059, January 2018.
  3. [3]
    Crow, David (19 October 2017). "Gene therapy helped these children see. Can it transform medicine?". Financial Times.
  4. [4]
    "Spark nails a $161M IPO to fund its 'breakthrough' gene therapy". 30 January 2015.
  5. [5]
    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.
  6. [6]
    "Roche completes $4.3B purchase of Philadelphia gene therapy pioneer Spark Therapeutics". American City Business Journals. 17 December 2019.
  7. [7]
    "Roche concludes acquisition of Spark Therapeutics, Inc. to strengthen presence in gene therapy" (Press release). Hoffmann-La Roche. 17 December 2019.
  8. [8]
    "Roche concludes acquisition of Spark Therapeutics, Inc. To strengthen presence in gene therapy".
  9. [9]
    "Spark Co-Founder Katherine High Departs Company Ahead of Merger with Roche". BioSpace. Retrieved 2020-03-03.
  10. [10]
    "Spark Therapeutics Announces Departure of CEO and Founder Jeff Marrazzo; COO Ron Philip Named as Successor – Spark Therapeutics".
  11. [11]
    "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.
  12. [12]
    "Pfizer Initiates Pivotal Phase 3 Program for Investigational Hemophilia B Gene Therapy" (Press release). Pfizer. 16 July 2018.
  13. [13]
    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.
  14. [14]
    George, John (16 July 2018). "Pfizer begins late-stage testing of Spark's hemophilia B gene therapy". American City Business Journals.
  15. [15]
    Garde, Damian (7 August 2018). "Spark's gene therapy data answer some burning questions — and raise a few more". Stat.
  16. [16]
    Tirrell, Meg (7 August 2018). "Spark Therapeutics shares lose a third of their value as hemophilia gene therapy trial disappoints investors". CNBC.
  17. [17]
    Morrison, Chris (5 March 2019). "Spark's meteoric rise from hospital-funded spinout to $4.8 billion deal". Nature Biotechnology.
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