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Gene Therapy Biotech Raises $161M in IPO

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(Michael Daddino/Flickr)

30 January 2015. Spark Therapeutics Inc., a biotechnology company in Philadelphia, is raising $161 million in its initial public stock offering. The company, developing genetic therapies for inherited diseases, priced 7 million shares of its common stock at $23.00 a share. The stock trades on the NASDAQ exchange under the symbol ONCE, and as of 12 noon on 30 January, was trading at $42.28.

Spark Therapeutics develops one-time 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 hematologist Katherine High, who now serves as Spark’s president and chief scientist.

Spark’s lead product, code-named SPK-RPE65, is an experimental treatment for the inherited retinal disorders Leber’s congenital amaurosis and retinitis pigmentosa now in late-stage clinical trials. The therapy is designed to fix a mutation in the RPE65 gene expressed in the retina. The gene encodes a protein that helps convert 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.

In addition, Spark has a gene therapy for choroideremia, another rare inherited retinal disease, code-named SPK-CHM in early-stage clinical trials. Choroideremia is associated with mutations in the CHM gene that block production of a protein needed to transport other key proteins to various cells in the body, including the retina.

Another Spark program develops gene therapies to treat hemophilia B, 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.

A Spark-developed therapy, 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.

In December, Spark began a licensing and collaboration agreement with drug maker Pfizer to further develop and commercialize SPK-FIX. Under the deal, Spark can gain as much as $280 million in return for sharing worldwide rights to SPK-FIX with Pfizer.

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