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Spark Therapeutics

Spark Therapeutics, Inc. was a developer of gene therapy treatments, which treat debilitating genetic diseases.[1] It was founded in 2013 and has been a subsidiary of Hoffmann-La Roche since 2020.

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

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 a gene therapy for the treatment of hemophilia B. It was developed by Spark 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] It received FDA approval in 2024.[14]

SPK-8011

SPK-8011 (Dirloctogene samoparvovec) 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]

External links

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. David Crow. Gene therapy helped these children see. Can it transform medicine? Financial Times, 19 October 2017^
  4. Spark nails a $161M IPO to fund its 'breakthrough' gene therapy 30 January 2015^
  5. John George. Shake Shack wasn't the day's only gonzo IPO. And this one, for Spark Therapeutics, raised more cash. American City Business Journals, January 30, 2015^
  6. Roche completes $4.3B purchase of Philadelphia gene therapy pioneer Spark Therapeutics American City Business Journals, 17 December 2019^
  7. Roche concludes acquisition of Spark Therapeutics, Inc. to strengthen presence in gene therapy Hoffmann-La Roche, 17 December 2019^
  8. Roche concludes acquisition of Spark Therapeutics, Inc. To strengthen presence in gene therapy^
  9. Spark Co-Founder Katherine High Departs Company Ahead of Merger with Roche BioSpace, 21 February 2020, retrieved 2020-03-03^
  10. Spark Therapeutics Announces Departure of CEO and Founder Jeff Marrazzo; COO Ron Philip Named as Successor – Spark Therapeutics^
  11. FDA approves novel gene therapy to treat patients with a rare form of inherited vision loss Food and Drug Administration, 19 December 2017^
  12. Pfizer Initiates Pivotal Phase 3 Program for Investigational Hemophilia B Gene Therapy Pfizer, 16 July 2018^
  13. George Lindsey. Spk-9001: Adeno-Associated Virus Mediated Gene Transfer for Hemophilia B Achieves Sustained Mean Factor IX Activity Levels of >30% without Immunosuppression Blood, 1 December 2016^
  14. U.S. FDA Approves Pfizer's Beqvez (fidanacogene elaparvovec-dzkt), a One-Time Gene Therapy for Adults with Hemophilia B Pfizer, 26 April 2024, retrieved 29 April 2024^
  15. Damian Garde. Spark's gene therapy data answer some burning questions — and raise a few more Stat, 7 August 2018^
  16. Meg Tirrell. Spark Therapeutics shares lose a third of their value as hemophilia gene therapy trial disappoints investors CNBC, 7 August 2018^
  17. Chris Morrison. Spark's meteoric rise from hospital-funded spinout to $4.8 billion deal Nature Biotechnology, 5 March 2019^