Gene Therapy

Pfizer is committed to leading in this space by bringing together the foremost expertise in gene therapy vector design and development with in-house knowledge of disease biology and manufacturing capabilities. We currently have 3 industry-leading phase 3 clinical gene therapy programs and have invested over $800M to expand our AAV manufacturing capacity. We have entered into multiple gene therapy collaborations, each of which is an important part of our continuing effort to build our gene therapy programs and capabilities. We 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), neuromuscular,heart, renal, hematology and metabolic diseases. We are also seeking tissue-selective and tunable expression promoter technologies, as well as vector analytics and immune surveillance approaches, and in-vivo gene editing technologies.

We are interested in partnering to develop and access:

• Rare Neurologic Diseases
  • Huntington’s Disease
  • Genetic Parkinson’s Disease
  • ALS/FTLD
  • Dominant Inherited Alzheimer’s Disease
• Muscular Dystrophies
• Rare Cardiac Diseases
• Sickle Cell Disease
• Rare Metabolic Disease
• Rare Renal Diseases
• Lysosomal Storage Diseases
• Novel AAV vectors and LNPs with strong tissue-specific tropism (CNS, muscle, heart kidney and liver) with favorable transduction/expression
• Promoter or gene regulation technology to ensure regulated and sustained tissue-specific gene expression
• Vector analytics to identify viruses with superior bioactivity
• AAV immunology expertise to test/challenge existing hypotheses and develop more robust gene therapy products