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With a shared urgency and unwavering passion, Pfizer is committed to unlocking the potential of gene therapy to make breakthroughs that change patients’ lives—today and in the future.

Pfizer is looking to go beyond just helping people manage their diseases. Our goal is to enable them to thrive in every stage of life. By using genes as medicine, we can address the underlying cause of a disease at the cellular level, and results could potentially be achieved in just one treatment.

“At Pfizer, we believe gene therapy holds tremendous potential. Through strategic partnership and investments, we’ve acquired the ability to advance this technology and guide a gene therapy through its entire life cycle. We hope to unlock the potential of using genes as medicine to make breakthroughs that change patients’ lives—today and in the future.” – Bob Smith, Senior Vice President, Global Gene Therapy Business, Pfizer

Pfizer’s Gene Therapy Focus: Frequently Asked Questions

  • How is Pfizer advancing research in gene therapy?

    Pfizer is advancing research on gene therapy as one of the next developments in delivering potentially transformational medicines to patients living with genetic diseases. With our commitment to rare disease patients, we look to gene therapy as an opportunity to improve the lives of patients who have complex diseases with significant unmet needs.1

  • What is Pfizer’s focus in gene therapy?

    Currently, Pfizer is focused on gene therapy treatments for diseases that have single-gene alterations. Our approach is highly specialized, potentially one-time gene therapy treatments that use custom-made vectors modeled after the recombinant adeno-associated virus (rAAV) that are designed to deliver treatment effectively to the targeted tissue.2 It is a technology that can be standardized, streamlining the manufacturing and regulatory path to medicine approval.1

  • Where are Pfizer’s focus areas in gene therapy?

    Pfizer’s gene therapy clinical trials for various diseases are currently underway. At Pfizer, we are committed to fully understanding the efficacy and safety of these transformational medicines with the ultimate goal of providing them to patients in need. Our clinical development portfolio includes programs for hemophilia A, hemophilia B, and Duchenne muscular dystrophy (DMD). At the same time, we’re building a robust pipeline through preclinical research investigating potential treatments for endocrine/metabolic diseases, such as Wilson Disease, and neurologic diseases, such as Friedreich’s Ataxia, Dravet Syndrome, and amyotrophic lateral sclerosis (ALS).1


1. Data on file. Pfizer Inc, New York, NY.

2. NIH Genetics Home Reference (GHR). What is gene therapy? Accessed February 10, 2020.

Our Focus in Rare Disease   -  

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