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Rare Disease

With the vision to be a leader in the development of therapeutic solutions for Rare Diseases, we have focused our business development strategy to align with the Category’s objective to identify and prioritize collaborations and partnerships to advance therapies for rare diseases where there remains high unmet need. Pfizer has adopted an innovative and collaborative approach to the development of new medicines for patients with rare diseases. We have a track record of creating innovative strategic partnerships with academic institutions, patient advocacy groups, and commercial enterprises to accelerate the development of novel therapeutics across a wide spectrum of rare diseases. Our expertise in small and large molecule therapeutics, and genetic therapies has resulted in a broad pipeline of potentially transformative medicines across four primary therapeutic areas, namely rare hematology, neurology, renal, cardiology and metabolic diseases. Therefore, we are seeking new business opportunities that span these disease indications and are not limited by molecule type.

Pfizer is interested in partnering to develop therapeutics, expanding our understanding of disease biology, and identifying biomarkers that can help us impact:

  • Rare renal and cardiac diseases
  • Rare endocrinology & metabolic diseases
  • Rare neurologic diseases
  • Rare hematologic diseases
  • Genetic-based approaches (e.g., gene therapy and gene editing) in the above disease areas

Specific areas of interest include:

  • Gene therapy-, gene editing-, and mRNA-based therapies including methods to minimize host immune responses and afford redosing with viral vectors
  • Oral small molecule- and biologics-based approaches
  • Modifiers of expanded repeat disorders
  • Pharmacologic chaperones and other modifiers of protein trafficking, misfolding, or degradation that could be applied to multiple diseases

Not actively seeking partnering opportunities in:

  • Dermatology
  • Rare oncology
  • Ex-vivo gene therapies