gives them the strength to walk farther and climb more stairs,
and data suggests patients on the therapy also live longer.
A marketing director was recently trying to arrange
a video shoot with the first patient to receive commercial
Naglazyme, now a student at Louisiana State University.
The patient’s exams schedule was interfering. “Jill sends
a note and says ‘School first!’,” says spokeswoman Debra
Charlesworth with a grin.
With such tiny customer bases and high research and
development costs, BioMarin must charge high prices for
these drugs. Infusions of Vimizim cost up to $400,000 a year
for one patient.
In addition to its expanded Novato production facility,
part of which used to be a Birkenstock warehouse, BioMarin
is expanding its new San Rafael headquarters and laboratories. Two start-ups that spun off from BioMarin, Raptor
Pharmaceuticals and Ultragenyx Pharmaceutical, are both
based in Novato. Marin is also headquarters to a foundation
that pushes for biotech-friendly public policy, the EveryLife
Foundation, led by Dr. Emil Kakkis, who left BioMarin to
start the foundation and later launched Ultragenyx.
THE BUCK STARTS HERE
Supporting these companies with research partnerships
and talent are the Buck Institute for Research on Aging and
the University of California, San Francisco. UCSF is more
commonly associated with the much larger biotech cluster
in South San Francisco, but BioMarin CEO Jean-Jacques
Bienaimé (“JJ” to his staff) points out that the university
is “not that much closer to South San Francisco than it is
to here.” BioMarin has partnered with both, as well as with
research institutions outside the region.
The Buck is helping to grow the Marin biotech cluster
by spinning off independent companies to bring its scientists’ discoveries on prolonging healthy life to market. The
nonprofit research facility has also supported the growth of
BioMarin and other existing biotech drugmakers through
scientific partnerships and by sharing resources like laboratory space; Ultragenyx leases its lab space from the Buck,
for instance. The Buck also continually draws new research
talent to the region through its undergraduate, graduate and
postdoctoral programs, some of which are run in partnership
“It’s very important for us to have a growing biotech sector
in this region,” says Buck president and CEO Brian Kennedy.
“While we’re happy to partner with pharmaceutical compa-
nies any where if it makes sense, it’s nice to be able to bounce
ideas off people who are local and get that exchange of ideas
that it’s hard to get at a distance.”
Over time, more companies are expected to spin off
from the Buck and BioMarin and even from their spin-offs.
Despite all this growth, Marin’s biotech cluster is still far
from rivaling the size of the cluster surrounding Genentech
in South San Francisco, or even San Diego’s cluster. But it
has progressed from the early start-up phase to the second
stage of development, says Robert Eyler, chief executive of
the Marin Economic Forum.
GROWTH WITH CHALLENGES
Many of the smaller companies, like Ultragenyx, are also
expanding. The firm grew its staff to more than 100 from
59 this year and is likely to need more space as its products
move into later-stage development, says Chief Financial
Officer Shalini Sharp. Rounding out the cluster are biotech
firms that don’t make drugs, most of them also in Novato,
such as Cytograft Tissue Engineering, which repairs diseased cardiovascular tissue using the patient’s own cells;
Biosearch Technologies, which makes nucleic-acid-based
products for genetic research; XCell Science, which creates human neural cells for research, and Marin Biologic
Laboratories, a research contractor.
“You’re seeing a lot of the businesses in Marin County are
maturing, but are not yet mature enough to have multiple
product lines and multiple years’ worth of contracts. A lot of
the companies are still trying to get drugs through the testing
process and are in a fundraising phase,” Eyler says.
That applies to Ultragenyx, which has four products in clinical trials, and Raptor, which has one drug on the market and
three in clinical trials. BioMarin is more advanced, with five
drugs on the market, including Vimizim and Naglazyme, as well
as Kuvan (for PKU, the disorder tested in newborns with a heel