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

Orphan Druganaut Blog

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…

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