Pfizer Expands Rare Disease R&D Activities

Pfizer Inc. has announced plans to expand the company's rare disease research and development activities through the establishment of a gene-therapy platform. The company has signed an agreement with Spark Therapeutics, a Philadelphia-based biopharmaceutical company, to develop SPK-FIX, a program incorporating a bio-engineered AAV vector for the potential treatment of hemophilia B expected to enter Phase I/II clinical trials in the first half of 2015. Additionally, Pfizer has appointed Michael Linden, PhD, professor at King's College London and director of the University College London Gene Therapy Consortium, who will be with the company for a two-year secondment to lead gene-therapy research in the rare disease area. The appointment took effect December 1, 2014.

Under the companies’ agreement, Spark will maintain responsibility for clinical development through Phase I/II studies. Pfizer will assume responsibility for pivotal studies, any regulatory approvals, and potential global commercialization of the product.

Pfizer’s current focus on rare disease includes a global portfolio of 22 medicines approved worldwide that treat rare diseases in the areas of hematology, neuroscience, inherited metabolic disorders, pulmonology, and oncology.

Source: Pfizer

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