Spark Therapeutics, Inc. was a developer of gene therapy treatments, which treat debilitating genetic diseases. 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 AltschulerHiggins, Robert F., and Tina Liu. "Spark Therapeutics: Pioneering Gene Therapy." Harvard Business School Case 818-059, January 2018. in an effort to commercially develop treatments against haemophilia that High was working on at Children's Hospital of Philadelphia.
In January 2015, the company became a public company, trading under the ticker $ONCE via a $161 million initial public offering led by Chief Legal Officer Joseph La Barge.
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. 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. It now continues to operate as an independent subsidiary. Since the acquisition by Swiss pharma Roche, several key founding executives have departed, including scientist and co-founder Katherine High in February 2020, Chief Business/Legal Officer Joseph La Barge in December 2021, and co-founder and Chief Executive Officer Jeffrey Marrazzo in April 2022
On February 23, 2022, Marrazzo named big-Pharma veteran Ron Philip as his successor. Mr. Philip currently leads the organization.
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.
Fidanacogene elaparvovec Fidanacogene elaparvovec, previously known by its study ID number SPK-9001, 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. It received FDA approval in 2024.
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.
SPK-7001 SPK-7001 is an experimental drug under investigation for treatment of choroideremia, a genetic disorder that causes blindness.
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.
SPK-1001 SPK-1001 is an experimental drug under investigation for treatment of Batten disease, a fatal genetic nervous system disorder.
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