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Gene Therapy Company Acquired in $4.3B Deal

Retinal gene therapy

(National Human Genome Research Institute, NIH)

25 Feb. 2019. Spark Therapeutics, a biotechnology company that pioneered gene therapies, is being acquired by drug maker Roche Group in a deal valued at $4.3 billion. Philadelphia-based Spark Therapeutics is a 6 year-old enterprise spun-off from Children’s Hospital of Philadelphia and University of Pennsylvania.

The company develops treatments for inherited diseases that transfer healthy genes into patients to replace their mutated or damaged versions. These transfers are made with engineered adeno-associated viruses, benign microbes designed to deliver genetic material. Among Spark’s founders is Katherine High, a hematologist at Children’s Hospital and UPenn who pioneered adeno-associated virus delivery of gene therapies, and is now president and R&D director of the company.

Spark’s lead product is Luxturna, known generically as voretigene neparvovec, approved by the Food and Drug Administration to treat biallelic RPE65 mutation-associated retinal dystrophy, which results from a mutation in both alternative forms of the RPE65 gene, expressed in the retina. The mutations prevent correct encoding of a protein that converts light entering the eye into electrical signals transmitted to the brain, making sight possible. Without this protein functioning properly, visual cycles are disrupted and impaired leading to blindness. From 1,000 to 2,000 individuals in the U.S. have this condition. Luxturna was the first gene therapy approved by FDA, in December 2017.

The company is also developing a gene therapy for hemophilia-B, a disorder where the blood does not coagulate, due to a missing protein that mixes with blood platelets. About 8 in 10 people with hemophilia have type A of the disease, where the protein clotting factor 8 is missing. In type B, clotting factor 9 is missing. Factor 9 is made in the liver, and circulates dormant in the blood stream until a blood vessel is damaged, when it is activated by other proteins that set in motion the processes to coagulate into a clot. The treatment, code-named SPK-9001, is in late-stage clinical trials.

In addition, Spark is developing therapies for hemophilia A, also in clinical trials, as well as the rare inherited disorders Pompe, Batten, Huntington, and Stargardt disease. Genentech, a biopharmaceutical subsidiary company of Roche, now offers a hemophilia-A drug, Hemlibra, which as the industry newsletter Endpoints News points out, puts Roche in a leading position for hemophilia therapies.

Under the agreement, Roche is acquiring Spark Therapeutics shares at $114.50, about 2.2 times the share price at its close on Friday. With the absorption of a $500 million negative cash balance, the deal is valued at $4.3 billion. Spark is expected to continue its independent operations under the Roche umbrella, in much the same way as Genentech.

While the cash outlay by Roche for Spark is significant, the deal is considerably smaller than similar recent acquisitions. As reported by Science & Enterprise, Novartis paid $8.7 billion for the gene-therapy biotechnology company AveXis in April 2018, and Sanofi acquired Bioverativ, a developer of biologics to treat hemophilia and other rare blood disorders, for $11.6 billion in January 2018.

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