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Gene therapy; size matters

Pfizer is leading the way to unlock the promise of gene-therapy

Presentation abstract:

Gene therapy is the next generation of medicine targeting the underlying cause of genetic disease. It has the potential to offer transformational clinical benefit and dramatically improve the quality of life of patients.

Our recombinant Adeno Associated Virus (rAAV) vector delivers genes directly to targeted cells. When the vector is integrated in the cell, the functioning gene is transferred and used as a blueprint to produce the missing or non-functioning protein. This is different from other gene therapy techniques like CRISPR, in which the functioning gene is integrated into a patient’s chromosome. Pfizer is currently focusing on diseases caused by single-gene alterations such as hemophilia A/B and Duchenne’s. Moreover, our robust pipeline includes potential gene therapy solutions for a.o. Wilson’s disease, Friedreich’s ataxia and ALS.

Pfizer’s gene therapy platform, comprised of internal capabilities and strategic partnerships, enables us to provide end-to-end solutions for all challenges of gene therapy; from drug design to global commercialization. Together with all stakeholders involved we need to lay the groundwork now; by 2023 there could be 40 gene therapies available for patients.

In this presentation a selection of the many challenges related to gene therapy are highlighted. Size matters when it comes to offering gene therapy to patients, in terms of size of transferred genes as well as size of manufacturing capabilities!