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Behind-the-Scenes of Gene Therapy

Gene therapy represents the next wave of treatment innovation and holds promise for patients living with rare genetic diseases.

We are developing highly specialized treatments that use custom-made recombinant adeno-associated virus (AAV) vectors to deliver gene therapy directly to targeted cells. Currently, we are focusing on diseases caused by a single-gene alteration.

When the vector reaches the targeted cell, the functioning gene is transferred and used as a blueprint to produce the missing or non-functioning protein.

Interactive Look at Gene Therapy Manufacturing

Take a journey with us as we tour our Sanford, North Carolina gene therapy manufacturing facility and explore how gene therapies are made.

End-To-End Gene Therapy Capabilities

Please click on the image below to learn more about manufacturing.

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Gene Therapy Manufacturing: Frequently Asked Questions
Question Pairs: 
Question: 

How do you manufacture a gene therapy treatment?

Answer: 

Pfizer will manufacture gene therapy treatments using production processes that are very similar to other biotechnology products such as monoclonal antibodies and vaccines. The process uses recombinant cell culture technology and purification followed by sterile vial filling. The similarity to traditional biotech processes means higher confidence on the scale up, reproducibility, and the design of manufacturing facilities, than past gene therapy manufacturing processes.

Pfizer’s investment in enabling gene therapy discovery, development, and manufacturing occurs in three manufacturing facilities in the continental United States, including a state-of-the-art, commercial scale, gene therapy-focused manufacturing complex in Sanford, North Carolina. With these capabilities, we have the ability to guide a gene therapy medicine through its entire life-cycle: from vector design and development to preclinical and clinical testing to regulatory approvals and potential global, commercial distribution.

Question: 

What are the steps that go into manufacturing a gene therapy treatment?

Answer: 

There are four steps to the manufacturing process.

  • Step 1: Raw Material Preparation: A gene therapy vector consists of three parts – the vector genome, AAV protein, and helper proteins – to ensure the AAV functions optimally. In the first step of the process, we secure and combine these components to manufacture the shell, called a capsid, which houses the functioning gene that will be delivered to the affected tissue.
  • Step 2: Upstream Process: Next, the newly created capsids are encapsulated into the viral vector. HEK cells are used to produce the capsids and package the functioning gene to create the vector.
  • Step 3: Downstream Process: This is the purification stage, where the non-essential cellular matter is removed from the vector. Next, the vector is reviewed to ensure it is working properly and measured to confirm there is an appropriate volume of the therapy to provide treatment.
  • Step 4: Product Completion & Packaging: The final step is to package the gene therapy for clinical or commercial use.
Question: 

How long does it take to manufacture a gene therapy treatment?

Answer: 

While the total amount of time needed to develop gene therapies depends on the targeted disease and the impacted body tissue, it generally takes 9-10 months from start-to-finish.

Question: 

Where is Pfizer in the process in regard to gene therapy?

Answer: 

Gene therapy clinical trials for various diseases are currently underway. At Pfizer, we are committed to fully understanding the efficacy and safety of these potentially 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 committed to building a robust pipeline through preclinical research investigating potential treatments for endocrine/metabolic disorders, such as Wilson Disease, and neurologic disorders, such as Friedreich’s Ataxia, Dravet Syndrome, and amyotrophic lateral sclerosis (ALS).