Internal capabilities ‘the backbone’ of Pfizer’s gene therapy strategy

With a $500 million facility in North Carolina set to come online this month, Pfizer has lauded its approach to gene therapy development and manufacture.

Big Pharma peers Roche and Novartis (via the acquisitions of AveXis and Spark, respectively), have achieved regulatory success in the gene therapy field, and Pfizer hopes to replicate similar achievements in the coming months and years.

To get there, the firm is following a “three-pronged strategy,” according to Robert Smith – SVP of Global Gene Therapy Business, Pfizer Rare Disease – involving the “building of internal capabilities, complemented with selected acquisitions and an extensive network of partnerships with both leading academic centers and leading biotech stay in the space.”

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Pfizer became a potential major player in the gene therapy space due to a number of partnerships and deals in the sector over the past five years.

The acquisition of Bamboo Therapeutics in 2016 brought what is now a Phase III gene therapy candidate for Duchenne Muscular Dystrophy (DMD), while a partnership with Sangamo Therapeutics is hoping to bring hemophilia gene therapies to the market. An agreement in 2020 sees Pfizer making supplies of VTX-801 – Vivet Therapeutics’ candidate gene therapy for the liver condition Wilson disease – with the Big Pharma having an option to acquire Vivet after the delivery of certain data from the Phase I/II clinical trial.

But these deals are heavily reliant on the heavy investment Pfizer has made in its internal manufacturing capabilities, Smith said at the Evercore ISI HealthCONx Virtual Conference earlier this month.

The firm has three dedicated gene therapy located in Morrisville, Durham, and Sanford – all in North Carolina. The Sanford site has been subject to a $500 million investment, announced in August 2019, which is set to come online later this month, Smith confirmed.

“In Stanford, North Carolina – which was a biologics campus for manufacturing – we have two facilities, one as we called stage one, which is both for late stage clinical and commercial manufacturing and then we have a second more expansive facility which is coming online with GMP production actually this month.”

He added: “We feel really confident that with the portfolio [and] the capabilities that we build, we’ll be able to efficiently advance these programs into the clinic and potentially even to the market.”