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Gene Therapy

Gene therapy is a technology aimed at correcting or fixing a gene that may be defective.

Gene therapy is a technology aimed at correcting or fixing a gene that may be defective. This exciting and potentially transformative area of research is focused on the development of potential treatments for monogenic diseases, or diseases that are caused by a defect in one gene.

The technology involves the introduction of genetic material (DNA or RNA) into the body, often through delivering a corrected copy of a gene to a patient’s cells to compensate for a defective one, using a viral vector.

The technology involves the introduction of genetic material (DNA or RNA) into the body, often through delivering a corrected copy of a gene to a patient’s cells to compensate for a defective one, using a viral vector.

Viral vectors can be developed using adeno-associated virus (AAV), a naturally occurring virus which has been adapted for gene therapy use. Its ability to deliver genetic material to a wide range of tissues makes AAV vectors useful for transferring therapeutic genes into target cells. Gene therapy research holds tremendous promise in leading to the possible development of highly-specialized, potentially one-time delivery treatments for patients suffering from rare, monogenic diseases.

Gene therapy research holds
tremendous promise…

Pfizer aims to build an industry-leading gene therapy platform with a strategy focused on establishing a transformational portfolio through in-house capabilities, and enhancing those capabilities through strategic collaborations, as well as potential licensing and M&A activities.

We're working to access the most effective vector designs available to build a robust clinical stage portfolio, and employing a scalable manufacturing approach, proprietary cell lines and sophisticated analytics to support clinical development.

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In addition, we're collaborating with some of the foremost experts in this field, through collaborations with Spark Therapeutics, Inc., on a potentially transformative gene therapy treatment for hemophilia B, which received Breakthrough Therapy designation from the US Food and Drug Administration, and 4D Molecular Therapeutics to discover and develop targeted next-generation AAV vectors for cardiac disease.

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With the recent acquisition of Bamboo Therapeutics, Inc., we've also expanded our portfolio to include potential best-in-class advanced recombinant AAV-based gene therapies. Bamboo utilizes unique suspension cell based production technologies to generate a number of novel AAV vectors, including chimeric, self-complementary and dual glycan receptor AAV, that together make up a powerful platform for targeting a wide range of central nervous system disease and neuromuscular diseases. The acquisition combines Bamboo's advanced vector design and production technologies with Pfizer’s global scale, R&D and commercialization experience to support the development of potentially life-changing therapies.

Gene therapy holds the promise of bringing true disease modification for patients suffering from devastating diseases, a promise we’re working to seeing become a reality in the years to come.