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Pfizer, Biotech Partner on Hemophilia B Gene Therapy

Adeno-associated virus

Adeno-associated virus (

8 December 2014. Spark Therapeutics, a biotechnology firm in Philadelphia, is collaborating with the pharmaceutical company Pfizer to commercialize Spark’s gene therapy technology as a treatment for hemophilia B. Spark Therapeutics can gain as much as $280 million in the deal that involves sharing worldwide rights to Spark’s technology for hemophilia B with Pfizer.

Hemophilia B is an inherited disorder that results from the absence of a clotting protein in blood known as factor IX or FIX. People with hemophilia B bleed longer than people with FIX, with some 60 percent of cases considered severe, meaning people with the condition experience bleeding after an injury, including spontaneous bleeding into muscles and joints. Spark Therapeutics cites data from the 2012 World Federation of Hemophilia Annual Global Survey, indicating some 26,000 males worldwide suffer from hemophilia B.

Spark Therapeutics develops gene therapies for inherited diseases based on research conducted at Children’s Hospital of Philadelphia, a teaching hospital of University of Pennsylvania. The company’s technology uses engineered adeno-associated viruses, benign microbes designed to deliver genetic material into the cells of patients with defective genes. The company began in October 2013, founded by four scientists from Children’s Hospital, including hemotologist Katherine High, who now serves as Spark’s president and chief scientist.

One of Spark’s therapies, code-named Spk-FIX, is designed to deliver genetic material with an adeno-associated virus to the liver, where coagulation factors including FIX are produced. The company says proof-of-concept and early-stage clinical trials show Spk-FIX expresses a therapeutic gene in the liver that stimulates FIX at sufficient levels for patients to discontinue preventive or replacement treatments for some periods of time.

Under the deal with Pfizer, Spark Therapeutics will continue early and intermediate-stage clinical trials of Spk-FIX, while Pfizer will be responsible for subsequent late-stage trials, regulatory approvals, and worldwide commercialization of the product. Spark is receiving an initial $20 million payment from Pfizer, and will be eligible for for up to $260 million in further milestone payments for multiple hemophilia B treatment candidates. Spark can also receive royalties on sales of products developed in the partnership.

Pfizer is developing other drugs for hemophilia, including BeneFIX, an engineered replacement FIX treatment. In July, Pfizer reported a late-stage clinical trial showing BeneFIX taken once a week reduced annual bleeding rates of people with hemophilia B compared to a placebo.

Pfizer also announced today the appointment of Michael Linden, a virology professor at King’s College London, to a 2-year assignment leading the company’s gene therapy program in rare deseases. Linden’s research includes studies of the biochemistry and structure of adeno-associated virus proteins.

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