Roche has entered into a definitive merger agreement to acquire Spark Therapeutics a in a cash transaction valued at approximately $4.3 billion. Spark Therapeutics, based in Philadelphia, PA, discovers, develops and delivers gene therapies for genetic diseases, including blindness, hemophilia, lysosomal storage disorders and neurodegenerative diseases.

Spark’s lead clinical asset is SPK-8011, a novel gene therapy for the treatment of hemophilia A, which is expected to start Phase III in 2019. Spark also has SPK-8016 in a phase 1/2 trial aimed at addressing the hemophilia A inhibitor population. Spark Therapeutics was the first company to receive FDA approval for a gene therapy for a genetic disease in 2017. LUXTURNA is a one-time gene therapy indicated for the treatment of biallelic RPE65 mutation-associated retinal dystrophy.

Spark Therapeutics’ additional clinical assets include: SPK-9001, an investigational gene therapy for the potential treatment of hemophilia B in Phase III and SPK-7001 for choroideremia in Phase 1/2. The company is also developing SPK-3006 for Pompe disease and SPK-1001 for CLN2 disease (a form of Batten disease) which are expected to be ready for clinical development in 2019, as well as additional preclinical programs for Huntington’s disease and Stargardt disease.

Severin Schwan, chief executive officer of Roche, said, “Spark Therapeutics’ proven expertise in the entire gene therapy value chain may offer important new opportunities for the treatment of serious diseases. In particular, Spark Therapeutics’ hemophilia A program could become a new therapeutic option for people living with this disease. We are also excited to continue the investments in Spark Therapeutics’ broad product portfolio and commitment to Philadelphia as a center of excellence.” Spark Therapeutics’ will continue its operations in Philadelphia as an independent company within the Roche Group. 

“As the only biotechnology company that has successfully commercialized a gene therapy for a genetic disease in the US, we have built unmatched competencies in the discovery, development and delivery of genetic medicines. But the needs of patients and families living with genetic diseases are immediate and their needs vast,” said Jeffrey D. Marrazzo, chief executive officer of Spark Therapeutics. “With its worldwide reach and extensive resources, Roche will help us accelerate the development of more gene therapies for more patients for more diseases and further expedite our vision of a world where no life is limited by genetic disease.”