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Pfizer And Spark Therapeutics: Rare Disease Gene Therapy Collaboration For Hemophilia B

December 8, 2014




Pfizer and Spark Therapeutics announce on December 8th a collaboration agreement to :

•   Develop SPK-FIX, a program that uses a bio-engineered AAV vector (gene therapy) for the potential treatment of Hemophilia B

•   Initiate Phase I/II clinical trials in 1st half of 2015 for the SPK-FIX program.

Additionally, Pfizer announces on December 8th, the appointment of Michael Linden, Ph.D., Professor at King’s College London and Director of the University College London Gene Therapy Consortium, who will lead gene therapy research for a 2-year secondment.

One year ago, Children’s Hospital of Philadelphia (CHOP) invests $50 million in Spark Therapeutics, a Philadelphia-based rare diseases biotech start-up, with the goal of developing gene therapies for orphan diseases. Since the company’s launch, $83 million in financings have been secured from eight life sciences institutional investors. In September 2014, FierceBiotech, names ST as one of 2014’s “Fierce 15 biotechnology companies” and is named as one of BioSpace’s “Top 30 Life Science Start-ups To Watch in the U.S.”. Spark Therapeutics assumes control of two gene therapy clinical trials that started at CHOP:

•   Phase III study for Inherited Retinal Dystrophies (IRDs) caused by autosomal recessive mutations in the RPE65 gene – most advanced product candidate, SPK-RPE65

•   Phase I/II study for Hemophilia B – product candidate SPK-FIX.

Under the collaboration agreement, Spark Therapeutics is responsible for the clinical development through Phase I/II for the AAV Vector Program in Hemophilia B. Pfizer is responsible for the pivotal studies, regulatory approvals, and future global commercialization of the gene therapy product.

Hemophilia B patients do not make Factor IX, which is a protein that allows the blood to clot. As a result, when a blood vessel is injured, the blood vessel is not sealed and blood continues to leak out. During gene therapy, an Adeno-Associated Virus (AAV) carries a working gene into the body to replace a faulty gene. In Hemophilia B, if gene therapy is successful, the body will start to make its own Factor IX again.

Please Note: “LuMaxArt Golden Guys Teamwork Gear Runners” by lumaxart [CC-BY-SA-2.0] | Wikimedia Commons.

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