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VIDEO: a turning point for gene therapy production

Ultragenyx begins a new era in gene therapy manufacturing

VIDEO: overview of Ultragenyx’s new gene therapy manufacturing facility

This month, Ultragenyx officially opened its state-of-the-art gene therapy manufacturing facility in Bedford, Massachusetts with a ceremonial ribbon-cutting attended by hundreds of employees and community partners.

This 110,000-square-foot facility represents the very latest advancements in the field of adeno-associated virus (AAV) gene therapy production. The 120 dedicated employees who operate this facility are leading a new era of gene therapy manufacturing – producing these potential gene therapies at a greater scale than previously possible. As we celebrate this achievement, we want to share a little bit about how we got to this moment and what comes next.

Why build a gene therapy manufacturing facility?

First, let’s talk about why we made the decision to invest in gene therapy manufacturing and why we chose this site in Bedford for our facility.

Ultragenyx entered the gene therapy space by acquiring Dimension Therapeutics in 2015. Dimension had developed a proprietary producer cell line (PCL) that could manufacture AAV vectors more efficiently and reliably, and at greater scale. The organization was also a perfect fit for our mission of transforming the lives of people living with rare disease; they were already developing potential gene therapies for the treatment of rare genetic conditions. With the acquisition completed, the Greater Boston area became the home for Ultragenyx’s gene therapy organization.

Members of the Bedford facility leadership team

PHOTO: Members of the Bedford facility leadership team, along with Ultragenyx executives, patient advocates and community leaders, cut the ribbon to officially open the gene therapy manufacturing plant in Bedford, Massachusetts on June 21, 2023

In 2018, Ultragenyx made the decision to become a truly end-to-end rare disease medicine company – from discovery and preclinical research to clinical development, commercialization and manufacturing – by designing and building a gene therapy manufacturing plant that could meet our needs for clinical and commercial gene therapies for many years to come.

In 2020, we found a site in Bedford that was near our existing gene therapy research and development locations, and in close proximity to the world-class talent and educational institutions that have made the region a hub for biotechnology. Over the last three years, the Town of Bedford has become an incredible partner to help make this facility a reality, along with many other community partners who believed in our mission and were motivated by the needs of the rare disease community.

Advantages of in-house manufacturing

This decision to build our own manufacturing facility was made in part based on the limited supply of gene therapy manufacturing capacity across the industry. This is an exciting time of growth in the field, and we are seeing an unprecedented volume of studies underway – there are approximately 2,000 gene therapy studies now underway across the industry – putting pressure on the contract development and manufacturing organizations that many biopharmaceutical companies use to make these products.

While we will continue to use a combination of in-house and contracted manufacturing capacity to meet the needs of our robust gene therapy programs, having our own manufacturing plant allows our teams more flexibility and optionality to set the priorities and timelines for manufacturing our products. In addition to helping insulate us from some supply chain issues, we also believe this facility will help us increase speed of production, lower costs and improve quality – so we can make these therapies more accessible to the people who need them.

Bedford Massachusetts

Photo: A staff member uses a specialized isolator and filling system at the Ultragenyx gene therapy manufacturing facility in Bedford, Massachusetts

What this facility can do

One thing that sets our Pinnacle PCLTM technology apart is the scale of production. We are manufacturing at the 2,000-liter scale and can run up to 30 batches per year in one suite. We designed this plant with the future in mind and we can double our current capacity if needed by activating a second manufacturing suite in the facility. In addition to higher volume, the Pinnacle PCL platform produces higher product quality and yield.

This facility was built to meet Good Manufacturing Practices for both clinical- and commercial-grade gene therapy products.

Impact on the community

During the grand opening ceremony, we heard remarks from Co-Founder and President of the International Foundation for CDKL5 Research Karen Utley, who shared, “When my daughter, Samantha, was diagnosed with a mutation on the CDKL5 gene, there were no resources for family support, no treatment pipelines and it felt like there was no hope. Today, the opening of this gene therapy production facility…brings me so much hope for the future of our community and chance for improved quality of life for our loved ones.”

The opening of this facility brings me so much hope for the future of our community and chance for improved quality of life for our loved ones.

In addition to supporting the needs of the rare disease community, this facility is also engaged with the local community. One example is our longstanding partnership with Middlesex Community College (MCC), whose Learn and Earn program helps local college students train for careers in the biopharmaceuticals industry. Several of the MCC students who came to us through the program have been hired into full-time positions at our Bedford facility.

We look forward to continued partnership with the rare disease community and our neighbors in Massachusetts.

What comes next

While we just celebrated the “official” grand opening, the facility has been occupied since late 2022 and initial manufacturing runs are currently underway. As magnificent as it is, this is only a building; it is the people who work inside that are changing the future for those living with rare disease.

We’re so excited for what this site symbolizes – the commitment Ultragenyx has made to the rare disease community. And every day we challenge ourselves to live up to that commitment as we progress our gene therapy programs through this facility. We’re thrilled about our new facility and we’re proud to celebrate its official opening.

Now, we’ve got work to do.

Sue Marrichi is VP and site head, Bedford gene therapy manufacturing plat at Ultragenyx

Tom Lauzon is VP of Ultragenyx gene therapy manufacturing