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What Is Vimizim

Developing a life science economy around the Buck Institute is good for Marin — and humanity.

Tim porter

The Buck Institute.

INFUSIONS OF VIMIZIM treat people with low levels of the N-acetylgalactosamine-6 sulfatase enzyme that breaks down the glycosaminoglycans that cause a form of mucopolysacclaridosis known as Morquio A. Got that?

In simpler terms, Vimizim means that Annabelle, age 7, will have the energy and stamina to lead a mostly normal life. In the U.S., only about 300 people suffer from Morquio A, and they require weekly infusions of Vimizim that can cost $400,000 a year. To learn more about Annabelle, Google BioMarin; her photo will appear and you’ll be inspired.

Meanwhile, it might be worthwhile to understand an emerging commerce in Marin known as “orphan drugs.” These drugs bear this nickname because they treat diseases afflicting no more than 250,000 people worldwide. If the orphan drug side of the biotech industry succeeds in Marin, it will mean great things for the vitality of our county’s economy.

BioMarin Pharmaceutical, the maker of Vimizim, was founded 20 years ago in Novato and is now headquartered in those downtown San Rafael high-rises you see from the freeway. With 2,200 employees in the U.S. and Europe (1,600 in Marin), the publicly traded company gets almost $900 million in annual revenue, though it has yet to turn a profit. “BioMarin’s focus is on patients, mostly children, suffering from rare genetic diseases,” says spokesperson Debra Charlesworth. “Vimizim is one of our five products, with several more in the pipeline.”

Another Marin biotech firm is Ultragenyx Pharmaceutical, founded in Novato in 2010. It is also publicly traded, has more than 275 employees and specializes in treatments for rare and debilitating genetic diseases. According to reports, the company is close to bringing several products to market. Recently, Ultragenyx signed a $65 million agreement with Takeda Pharmaceutical Company Limited of Japan to license and co-develop rare disease drugs.

Other Marin companies developing orphan drugs include Raptor Pharmaceuticals, a 10-year-old public company in Hamilton Landing with a drug designed for sufferers of a rare form of Huntington’s disease, and Mount Tam Biotechnologies, a small firm hoping to bring to market medical compounds to treat lupus. Mount Tam Biotechnologies is working with the Buck Institute for Research on Aging, in that familiar I. M. Pei–designed building sitting on the hillside just north of Novato.

Created in the late 1980s from the trust of a wealthy Ross couple, Leonard and Beryl Buck, the Buck Institute is an independent research facility whose mission is “to increase the healthy years of life.” Within its walls dozens of world-class scientists work in a collaborative environment to understand how getting older contributes to Alzheimer’s, Parkinson’s and heart diseases, as well as cancer, stroke, diabetes and glaucoma.

“The Buck Institute is absolutely an asset to the growth of life science commerce in Marin,” says Robert Eyler, Sonoma State University professor of economics and chief economist for the Marin Economic Forum. “Short of a leading research university, there’s nothing like it between the Golden Gate Bridge and the University of Oregon in Eugene.”

The Marin Economic Forum, whose founding members include Autodesk, Bank of America, Marin General Hospital and Whole Foods, is another group aiming to vitalize the area’s life science economy. So too is the North Bay Life Science Alliance, a consortium in Marin, Sonoma, Napa and Solano counties in which companies like Genentech, Medtronic and Novartis — along with BioMarin, Ultragenyx and Raptor Pharmaceuticals — seek to create corporate synergy and attract more like-minded corporations to the region. According to Eyler, the alliance currently involves 90 globally involved corporations that employ more than 10,000 individuals that contribute $4.9 billion to the local region’s economy.

If a vibrant life science economy can continue to grow and thrive here, with the Buck Institute as its nucleus, it will be incredibly good for Marin — and humanity.

That’s my point of view. What’s yours? Email pov@marinmagazine.com.

The views and opinions expressed in this article are those of the author and do not necessarily reflect the policy or position of Marin Magazine and its staff.

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