Roche will gain a major position in gene therapy with its move to purchase Philadelphia-based Spark Therapeutics in what has been reported as a $4.8 billion deal. Though Spark is best known in the retina community for receiving FDA approval for Luxturna, the first approved gene therapy treatment for a genetic disease (a gene defect that causes Leber’s congenital amaurosis), the company has a strong pipeline of investigational drugs for conditions ranging from hemophilia to Huntington disease.

"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, CEO 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."

“Spark Therapeutics’ proven expertise in the entire gene therapy value chain may offer important new opportunities for the treatment of serious diseases,” said Roche Chief Executive Severin Schwan.