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Rare Disease Gene Therapy Press Kit

Gene Therapy: Imagine the Possibilities

Pfizer Rare Disease is exploring the potential of gene therapy to treat rare genetic diseases. Through our research, it has become clear that genetics may not have to determine the future of a person living with rare disease. Imagine what we can learn if we unlock the true potential of using genes as medicine to help treat more than 6,000 known rare genetic diseases worldwide.

Gene therapy is a potentially transformative treatment approach that, if successful, may help reduce the burden of ongoing treatments for nearly 320 million people worldwide who are affected by rare genetic diseases.

Learn more about our innovative approach to addressing rare genetic diseases.



Gene Therapy: Pioneering A New Era of Care

Please click on image below to download full Fact Sheet as a PDF.

Gene Therapy Fact Sheet Image

Gene Therapy: Frequently Asked Questions

Please click here to download full FAQ as a PDF.

How is Pfizer Rare Disease advancing research in gene therapy?

What is Pfizer’s focus in gene therapy?

What is a genetic disease?

What is gene therapy?

What does gene therapy do to an individual’s DNA?

What are the potential benefits of gene therapy?

What are antibodies?

How do you know if you are eligible for gene therapy?

What are other approaches to genetic medicine currently being explored?

How has gene therapy evolved since the 1990s?

When will gene therapy be available?

Where is Pfizer Rare Disease in the process in regards to gene therapy?

i. Data on file. Pfizer Inc, New York, NY.
ii. Roberts SA, Dong B, Firrman JA, Moore AR, Sang N, Xiao W. Engineering Factor VIII for hemophilia gene therapy. J Genet Syndr Gene Ther. 2011;1:S1-006. doi: 10.4172/2157-7412.S1-006
iii. Thomas CE, Ehrhardt A, Kay MA. Progress and problems with the use of viral vectors for gene therapy. Nat Rev Genet. 2003;4(5):346-358. iv. Genetics Home Reference (GHR). Help me understand genetics: gene therapy. primer/therapy.pdf. March 28, 2017. Accessed May 30, 2017.
v. Smith, D. How Many Possible Combinations Of DNA Are There? Forbes. January 20, 2017. Accessed July 19, 2017.
vi. National Institutes of Health (NIH). How does gene therapy work? March 28, 2017. Accessed May 30, 2017.
vii. Mingozzi F, High KA. Immune responses to AAV vectors: overcoming barriers to successful gene therapy. Blood. 2013;122(1):23-36.
viii. Patient Info. Antibody and Antigen Tests. Accessed May 30, 2017.
ix. Your Genome. What is Genome Editing. Published November 11, 2016. Accessed May 30, 2017.