Company Growth, Ambition Drive Ultragenyx’s Continued Success

Ultragenyx bull statue

Two years after its acquisition of Dimension Therapeutics, Bay Area-based Ultragenyx Pharmaceutical, Inc. continues to see success in its clinical programs for the company’s adeno-associated virus (AAV) gene therapy treatments and in its overall growth.

At the end of December 2018, the company had more than 600 employees and it aims to add even more. Currently, the company is hiring for more than 80 open positions that will continue to strengthen Ultragenyx’s growth strategies. About 40 percent of the open positions are in Massachusetts, which is where Dimension, now headquarters of Ultragenyx Gene Therapy, is located.

Part of what’s driving the growth at Ultragenyx is its AAV programs for the treatment of rare and ultra-rare diseases, with a focus on serious and debilitating genetic diseases. In order to accomplish the development of those therapies though, Ultragenyx said a company like theirs can “only make a meaningful impact when your team has big ambitions.” The company has onboarded a number of leadership members to help foster that growth, including Chief Medical Officer Camille L. Bedrosian, who joined the company in early 2018 from Alexion Pharmaceuticals. Also helping lead the way on the research side is Sam Wadsworth, head of gene therapy at Ultragenyx, who came over to the company as part of the Dimension Therapeutics acquisition.

The strong research team Ultragenyx has assembled and will continue to assemble has led to some impressive results, including two U.S. Food and Drug Administration approved treatments, Crysvita for the underlying cause of X-linked hypophosphatemia and Mepsevii for the treatment of Mucopolysaccharidosis VII. Also, the company is pushing forward with its cutting edge gene therapy programs. In February, the Novato, Calif.-based company released top-line safety and efficacy data from the first, lowest dose cohort of the ongoing Phase I/II study of DTX401, an adeno-associated virus (AAV) based gene therapy under development for the treatment of glycogen storage disease type 1a (GSDIa), the most severe genetically inherited glycogen storage disease.

DTX401 is one of Ultragenyx’s investigational AAV8 gene therapies designed to deliver a potential life-changing therapy on the back of a virus. The company is also developing DTX301, an investigational AAV type 8 gene therapy, for the treatment of ornithine transcarbamylase (OTC) deficiency. Ultragenyx is driving forward in this white hot area as it aims at bringing potential one-and-done treatments to patient populations. In an interview with BioSpace, Wadsworth touted the gene therapy research being conducted by Ultragenyx. At one time, he said gene therapy was something that had to be defended as a viable method of treating a genetic disorder. Now though, with the regulatory approval of Spark Therapeutics’ Luxturna and AveXis’ (highly probable) soon-to-be-approved spinal muscular atrophy gene therapy, Wadsworth said people know these methods work.

“We know they work. There could be some setbacks, but we know that gene therapy can provide benefit,” said Wadsworth, who served as CSO prior to his current role at Ultragenyx Gene Therapy.

If Ultragenyx brings its AAV products to market, the hiring at the company will certainly continue as it expands its research into other areas. The company would also have to hire experienced sales teams for any commercial products. One group that could likely see some expansion is the company’s team of patient diagnosis liaisons. This team is responsible for finding new doctors with patients with the specific disease for which Ultragenyx has developed a therapy. The patient diagnosis liaisons are a separate team from the UltraCare Liaisons who assist physicians in placing patients on therapies and the UltraCare Guides who support patients and families with treatment or reimbursement needs.

When it comes to filling current and future roles, Ultragenyx said it is dedicated to maintaining a healthy, inclusive company culture where employees feel respected and valued. By following this company culture practice, the company said it will ensure its employees never lose sight of its mission, to be a hero to rare disease patients.

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