Gene Therapy: Imagine the Possibilities

More than 80% of rare diseases have a known genetic component. At Pfizer Rare Disease, we are exploring the potential of gene therapy to treat these rare genetic diseases through potentially life-changing innovations, trusted partnerships, and relentless passion. Through our research, it has become clear that genetics may not have to determine the future of a person living with a rare disease.

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. If we are successful, imagine the possibilities.

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



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Gene Therapy: Pioneering A New Era of Care

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Gene Therapy: Frequently Asked Questions

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Pfizer Rare Disease has been and will continue to be a part of the journey with the rare disease community. We are here to listen, to learn, and to make a difference in the lives of those affected by rare diseases.

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. https://ghr.nlm.nih.gov/ primer/therapy.pdf. March 28, 2017. Accessed May 30, 2017.
v. Smith, D. How Many Possible Combinations Of DNA Are There? Forbes. https://www.forbes.com/sites/quora/2017/01/20/how-many-possible-combinations-of-dna-are-there/#3510367c5835. January 20, 2017. Accessed July 19, 2017.
vi. National Institutes of Health (NIH). How does gene therapy work? https://ghr.nlm.nih.gov/primer/therapy/procedures. 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. https://patient.info/health/antibody-and-antigen-tests. Accessed May 30, 2017.
ix. Your Genome. What is Genome Editing. http://www.yourgenome.org/facts/what-is-genome-editing. Published November 11, 2016. Accessed May 30, 2017.