Gene Therapy

Gene therapy (GTx) has changed significantly with the discovery of novel AAV vectors that have the potential to be safe and more efficient than previous approaches. Pfizer is committed to leading in this space by bringing together the foremost expertise in vector design and development with in- house knowledge of disease biology and manufacturing capabilities. Our current collaboration with Spark Therapeutics Inc. on hemophilia, as well as the establishment of the Genetics Medicines Institute at Pfizer, led by Dr. Michael Linden, help to form the basis upon which we intend to continue building our gene therapy programs and capabilities. Toward that end, we are interested in creating additional relationships to build one of the leading gene therapy companies in the world.
With Pfizer’s commitment to rare monogenic diseases, we also see gene therapy as a key pillar of our Rare Disease strategy. In addition we are interested in the application of gene therapy to select central nervous system (CNS) and heart diseases. Beyond expansion of our development portfolio, we seek to build our technical capabilities by partnering in the gene therapy space on novel vectors that can target the liver, brain, and heart. We are also seeking promoter technologies, as well as industry leading vector analytics and immune surveillance approaches.

We are interested in partnering to develop and access:

  • Novel Targets in key areas of interest
    • Parkinson’s Disease
    • Friedrich's Ataxia
    • Spinal Muscular Atrophy
    • Duchene’s Muscular Dystrophy
    • Heart Failure
  • Novel AAV vectors with strong tissue-specific tropism (CNS, liver, and heart) with favorable transduction/expression
  • Promoter technology to ensure regulated and sustained tissue-specific gene expression
  • Vector analytics to identify viruses with superior bioactivity
  • AV immunology expertise to test/challenge existing hypotheses and develop more robust gene therapy products

External R&D Innovation Contact: Donna Armentano or Barbara Sosnowski