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Rare Disease


With the vision to be a leader in Rare Disease treatment, we have focused our business development strategy to align with the category’s objective to address areas of high unmet need for rare disease patients. 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 gene therapy has resulted in a broad pipeline of potentially transformative medicines across multiple disease areas. Therefore, we are seeking new business opportunities that span a multitude of rare disease indications and are not limited by molecule type.

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

  • Rare cardiology / pulmonology (including systemic disease such as AA amyloidosis)
  • Endocrinology
  • Inborn Errors of Metabolism
  • Rare Neurologic
  • Hematology
  • Gene Therapy

Specific areas of interest include:

  • Gene Therapy and Nucleic acid approaches to therapy
  • Oral small molecule and biologics approaches
  • Modifiers of DNA expanded repeat disorders
  • Pharmacologic chaperones and other modifiers of protein trafficking, misfolding, or degradation that could apply to multiple diseases
  • Lysosomal Storage Disorders

Not actively seeking partnering opportunities in:

  • Dermatology
  • Rare oncology
  • Ex-vivo gene therapies