Spark Therapeutics, Inc. is 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.
 | |
| Company type | Subsidiary of Hoffmann-La Roche |
|---|---|
| Industry | Biotechnology Pharmaceutical |
| Founded | 2013 |
| Founders | Katherine A. High Jeffrey Marrazzo Jean Bennett J Fraser Wright Beverly Davidson Jennifer Wellman |
| Headquarters | Philadelphia, Pennsylvania |
| Revenue |  $64 million (2018) |
| -$78 million (2018) | |
| Total assets | $814 million (2018) |
| Total equity |  $496 million (2018) |
Number of employees | 368 (2019) |
| Parent | Hoffmann-La Roche |
| Website | sparktx |
| Footnotes / references [1] | |
History
editThe 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
editVoretigene neparvovec
editVoretigene 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
editFidanacogene 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
editSPK-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
editSPK-7001 is an experimental drug under investigation for treatment of choroideremia, a genetic disorder that causes blindness.[17]
SPK-3006
editSPK-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
editSPK-1001 is an experimental drug under investigation for treatment of Batten disease, a fatal genetic, nervous system disorder.[17]
References
edit- ^ a b c "Spark Therapeutics, Inc. 2018 Form 10-K Annual Report". U.S. Securities and Exchange Commission.
- ^ Higgins, Robert F., and Tina Liu. "Spark Therapeutics: Pioneering Gene Therapy." Harvard Business School Case 818-059, January 2018.
- ^ Crow, David (19 October 2017). "Gene therapy helped these children see. Can it transform medicine?". Financial Times.
- ^ "Spark nails a $161M IPO to fund its 'breakthrough' gene therapy". 30 January 2015.
- ^ 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.
- ^ "Roche completes $4.3B purchase of Philadelphia gene therapy pioneer Spark Therapeutics". American City Business Journals. 17 December 2019.
- ^ "Roche concludes acquisition of Spark Therapeutics, Inc. to strengthen presence in gene therapy" (Press release). Hoffmann-La Roche. 17 December 2019.
- ^ "Roche concludes acquisition of Spark Therapeutics, Inc. To strengthen presence in gene therapy".
- ^ "Spark Co-Founder Katherine High Departs Company Ahead of Merger with Roche". BioSpace. Retrieved 2020-03-03.
- ^ a b "Spark Therapeutics Announces Departure of CEO and Founder Jeff Marrazzo; COO Ron Philip Named as Successor – Spark Therapeutics".
- ^ "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.
- ^ "Pfizer Initiates Pivotal Phase 3 Program for Investigational Hemophilia B Gene Therapy" (Press release). Pfizer. 16 July 2018.
- ^ 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.
- ^ "U.S. FDA Approves Pfizer's Beqvez (fidanacogene elaparvovec-dzkt), a One-Time Gene Therapy for Adults with Hemophilia B" (Press release). Pfizer. 26 April 2024. Archived from the original on 29 April 2024. Retrieved 29 April 2024 – via Business Wire.
- ^ Garde, Damian (7 August 2018). "Spark's gene therapy data answer some burning questions — and raise a few more". Stat.
- ^ Tirrell, Meg (7 August 2018). "Spark Therapeutics shares lose a third of their value as hemophilia gene therapy trial disappoints investors". CNBC.
- ^ a b c Morrison, Chris (5 March 2019). "Spark's meteoric rise from hospital-funded spinout to $4.8 billion deal". Nature Biotechnology.