Corrections or additions?
This article by Barbara Fox was prepared for the May 14, 2003 edition of U.S. 1 Newspaper. All rights reserved.
Leading Edge R&D: Pharmas & Biotechs
Most of the young research and development companies
on the Princeton scene are in the pharma/biotech areas, and the six
new companies profiled below offer some intriguing demographics. Three
of them — AstaTech, GeneWiz, and Pelican — are technical service
firms, meaning that they do analyses for larger companies. The first
two were founded by Chinese Americans who outsource work economically
to their native land.
The other three companies — Semorex, Amicus Therapeutics, and
Access Bio — are presenting their own new technologies. Semorex
was founded in Israel, the intellectual property of Amicus Therapeutics
was developed first in Japan, and Access Bio’s founder is from Korea.
All six companies reside in incubator-like spaces, at either the state-sponsored
"post incubator" space that comes furnished at Technology
Center of New Jersey, the laboratory complex built by the New Jersey
Economic Development Authority on Route 1 South in North Brunswick,
or the private one at Harold Kent’s Princeton Corporate Plaza on Deer
Say "chaperone" and one thinks of class trips
with parents herding kindergartners into a museum or a zoo. But a
firm at the Technology Center of New Jersey, Amicus Therapeutics,
uses the term chaperone to describe pharmacological solutions to genetic
Amicus is developing new therapies to treat genetic diseases, particularly
orally active drugs that are simple and convenient to administer.
Occupying four of the furnished spaces in the Commercialization Center
at the Technology Center of New Jersey, it is one of the new bright
stars on the pharma/biotech horizon. The firm has 10 employees and
hopes to hire 20 more people, including a CFO and chief medical officer,
by the end of the year, says CEO Norman Hardman. He expects to raise
$20 to $23 million by the end of the summer and begin clinical trials
Amicus can create "pharmacological chaperones" to help misplaced
proteins get to the appropriate site of activity and perform their
appropriate biological function. These misplaced or "misfolded"
proteins are the cause of some diseases of genetic origin. When the
proteins can "fold" correctly, they can bypass the cell’s
protein quality control mechanism, get to the right place, and perform
effectively, says Hardman.
"A protein is a long chain of amino acids, like beads on a string,"
explains Hardman. "It folds in a certain shape, and the shape
determines how the protein functions. If it gets changed by mutation,
it doesn’t fold the way it should, so it doesn’t function. We want
to correct that by a small drug that interacts with the protein so
it folds in the right way."
"As far as we know, we are the first commercial enterprise that
is focused on commercializing pharmacological chaperone technology.
Institutions currently working on pharmacological chaperone technology
are the University of California at San Francisco, the Scripps Institute,
and some scientists in the United Kingdom, but they are not corporately
focused," Hardman says.
Co-founder Jian-Qiang Fan, a graduate of Kagoshima University with
a PhD from Kyoto University, did a post-doctoral fellowship at Johns
Hopkins University. But he was back in Japan, working on lysosomal
storage diseases, when he discovered that when a small molecule drug
is bound to the protein in a particular way, the protein will work
more efficiently. At an academic meeting he met his future mentor,
Robert J. Desnick, an export on lysosomal storage diseases, and Desnick
— seeing the possibilities — brought Fan from China to Mount
Sinai School of Medicine. Fan paid for the patent filing, but Mount
Sinai helped him pursue the patent and has an equity stake in the
The consequences of protein misfolding have been known for some time,
but being able to develop drugs based on correcting the misfolding
is new, says Hardman.
Hardman grew up in north of England, near Manchester,
the same part of England as David Palling, the company’s director
of clinical development. His parents had a bakery, and his father
had also been a tailor. With good grades he earned his place in an
academic secondary school, where he says, "I excelled more than
I had ever done — I became top of the class and it tasted good.
I never looked back." An amateur pianist who plays Beethoven,
Chopin, and Satie, he has the theory that scientists are like artists
because both are passionate about their work.
He says his parents taught him to think independently, "to believe
that you don’t need to know everything about everything, that you
can figure something out along the way." He met his wife, a molecular
immunologist, when they were both studying in Scotland, and they live
in Cranbury with their two daughters, ages 10 and 12, who are being
A chemistry major at the University of London, Class of 1967, Hardman
earned his doctorate in biochemistry from the University of Manchester.
He worked for Ciba-Geigy Pharmaceuticals in Switzerland and the UK,
and he led its antibody engineering program from 1986 to 1993. He
spent a year as head of R&D at Novartis in Horsham, UK, and then moved
to Texas-based GeneMedicine, where as president and COO he helped
set up the company’s 1999 merger with California-based Megabios Corporation.
Then he was COO of Onyx Pharmaceuticals, also based in California,
and most recently was senior vice president of technology at Enzon
Inc., where he expanded the firm’s single-chain-antibody pipeline.
Before he took the Amicus job he was on the board and had helped to
put the business plan together and recruit the management team.
Venture capitalist Ronald W. Lennox, chairman of the Amicus board,
had been with Hancock Venture Partners and is now with Collinson Howe
& Lennox (CHL) in Stamford, Connecticut. Ten of the 16 companies he
founded at Hancock have gone public.
David Palling, vice president of pre-clinical development, has undergraduate
and graduate degrees from the University of London, King’s College,
and did postdoctoral work at Brandeis. Most recently he was vice president
of worldwide assay R&D at Ortho Clinical Diagnostics, a Johnson &
Johnson Company, where he supervised new product development in transfusion
medicine and immunodiagnostics. He has also been senior director for
pharmaceutical development at Johnson & Johnson Pharmaceutical Research
and Development and had worked at Hoffman-LaRoche.
"In smaller companies you can afford to be more nimble," says
Hardman, telling how he is working with clinical research organizations
(CROs) on the initial stages of his clinical development plan. Amicus
plans for its first product to be used by patients in the first quarter
of next year. Then it will begin to get attention from larger companies
that are active in similar technology areas, such as Genzyme in Boston
and Biomarin in Marin County, California.
Another advantage to being small is that small organizations can afford
to look at the opportunities offered by "orphan" diseases,
with fewer than 100,000 patients in the United States. "If you
are the first in the class to move forward, you get seven years protection,
once you have the drug on your market. And if your molecule is giving
considerable perceived medical benefit, you can get fast track designation,
which means early evaluation of your drug for approval," says
For deals with larger firms, Amicus focuses its partnering efforts
on the front end, on helping academic scientists work on exploratory
programs in areas such as cystic fibrosis. "If we get to the point
where we have an application in a larger therapeutic area, then those
are the deals we would partner with a larger company."
Hardman is talking to a half-dozen venture capital companies and by
mid year he hopes to have second stage funding, $23 million for the
next two years. Roughly two-thirds of that money would be used for
clinical trials and 20 percent for subsidizing research in academic
laboratories, the rest for infrastructure.
"Even with a small company we could work on orphan drugs without
partnering, versus developing a drug for large markets that require
$4 to $5 million to get the drug to market," says Hardman.
CHL has contributed $2.5 million and currently owns about half of
the company, and the two founding scientists "have a considerable
stake," says Hardman.
The company started in incubator space at Mt. Sinai and moved to Columbia
University in the Audubon incubator, where one of its current neighbors
— GeneWiz — was also staying. Andy Schiffer of Cushman & Wakefield
helped find the four laboratories and four offices that Amicus occupies
in the post-incubator facility at the NJEDA’s Technology Center of
New Jersey. "We moved in last August and will probably renew until
we find our permanent facility," says Hardman.
"Unlike some new technology companies we are already developing
drugs. The beauty is, we can keep our options open," says Hardman.
"We have to plan to build the business as far as we can see in
the distance, and we can also see alternatives to that, based on how
the interest in the company might develop."
Center of New Jersey, North Brunswick 08902. Norman Hardman, CEO.
732-745-9977; fax, 732-745-9769. Home page: www.amicustherapeutics.com
Steve Sun and Amy Liao are Chinese American scientists
who started their successful pharmaceutical service firm, GeneWiz,
three years ago and moved to the Technology Center of New Jersey (TCNJ)
last year. Sun, who is living his entrepreneurial dream, plans to
tap economical offshore labor by opening a branch in China, which
is now open to such opportunities. "At the time I left China I
could not decide my future myself," says Sun.
A molecular biology contract research company, GeneWiz specializes
in DNA sequencing, cloning, and protein expression services. Its services
range from simple sequencing (a commodity product that costs $16 per
reaction) to big projects that can cost hundreds of thousands of dollars.
Its clients are researchers at academic, commercial, and government
With six full-time workers, GeneWiz is growing — it just posted
two new scientist jobs. "We are the most established company in
the corridor from North Carolina to Boston, and as far as we know
we are the only one doing these services in New Jersey," says
Sun. "Most of our competitors are in Texas, Illinois, and California."
Sun met Liao in Beijing, where both were going to school, and they
met again at Columbia University. Both are married and live near Edison.
Sun’s wife is a neurobiologist who works at Columbia, and they have
a school-aged son and daughter. His mother, who is a retired obstetrician,
lives with them. Liao’s husband works for Lucent, and they have a
Liao went to Nankai University in Tianjin and earned her master’s
degree at Tsinghua University, where Sun did both his undergraduate
and master’s degrees. Liao got her doctor’s degree at State University
of New York in Stony Brook, and did post doctoral study at Columbia.
Sun earned his PhD at Columbia and did post doctoral work in molecular
neurobiology at Rockefeller University and Howard Hughes Medical Institute.
"I was working at Rockefeller for three years, considering my
next career move, when I decided to be an entrepreneur, to see what
we can achieve that way," says Sun.
"Now it is possible to be an entrepreneur in China," says
Sun. His high school friend, in fact, had started his own company
in 1995 and helped Sun get the substantial amount of money he needed
for equipment. The friend’s firm, Freeman International, makes intermediates,
a pharmaceutical raw material, that Sun was able to import, store,
and sell — to raise seed capital for GeneWiz.
Sun started the business in 1999 on Long Island and moved to Audubon
Technology Center in Manhattan for the second and third years. At
this state-sponsored incubator near Columbia University, it had just
900 square feet, not enough space for the growing company. Matt Malatich
of CB Richard Ellis represented Sun in finding the space in the Commercialization
Center at the Technology Center of New Jersey (TCNJ).
TCNJ is supposed to be a synergistic place, where the work of one
tenant can help another. Such synergy actually happened, says Sun,
with GeneWiz. When he moved into two units of the Commercialization
Center, the EDA officials introduced him to George Matcham, senior
vice president of Celgene in charge of its agricultural subsidiary,
Celgro. Now Celgro is Genewiz’s client.
Genewiz’s client profile includes big pharmaceuticals such as Merck,
Pfizer, Novartis, and Schering, plus biotech companies "all over
the map," he says, such as Genentech in California, and academic
clients at Columbia, Yale, Harvard, and NIH. Many commercial clients
are in New Jersey. "Here it is much easier for us to visit them,
and they know we are in the neighborhood if they need to visit us."
Conversely, most of his former neighbors in the Audubon incubator
regret his leaving, says Sun. "Now they can’t come next door and
drop their samples. They have to pay for overnight delivery."
His marketing plan: "Scientists come to us because they need our
services, or we get referrals from colleagues, the Internet, or our
ads in trade journals. We also visit the laboratories to find out
what they need, and we have a sales rep, Ed Harris."
To access the economical human resources in China, he had planned
to set up an operation there later this year but, because of the SARS
outbreak, that will be delayed. "We have our own research component
that we want to develop also," he says. "And I want to expand
our services into forensic ID and into the consumer market — and
into diagnostics and personalized medicine. We have the expertise,
and I see that genetic medicine is on the horizon."
"I am not against going public or being purchased but that is
not the goal. I want to focus on building our business. As an entrepreneur,"
Sun says with enthusiasm, "the sky is the limit."
New Jersey, North Brunswick 08902. Steve Sun, president. 732-828-8996;
fax, 732-828-8790. Www.GeneWiz.com
Access Bio is a good example of how TCNJ’s Commercialization
Center is supposed to work. Founded by Yung Choi, a native of Korea,
Access Bio was one of the earliest tenants of the incubator (U.S.
1, June 26, 2002). Now it has run out of space and will expand from
800 square feet at TCNJ to 15,000 square feet, one-fourth of a new
building at Brunswick Business Park, 2033 Route 130 in South Brunswick.
It had six people last year and has added three more.
Not only has Choi filed for a patent on his protein chip technology
for DNA diagnostics but last winter he received a $63,000 Small Business
Innovation Research grant from the U.S. Department of Defense for
early diagnosis of scrub typhus, a disease found in Asia. Caused by
a mite, untreated scrub typhus can have up to a 30 percent mortality
rate. "We developed a test kit for the SBIR program and successfully
demonstrated the feasibility of the test kit," says Choi. At the
time of last week’s telephone interview he was on deadline to submit
his application for a Phase II SBIR grant worth $700,000.
With its protein chip technology, Access Bio hopes to develop new
in-vitro diagnostic tests. "The current technology uses expensive
instruments and requires highly trained persons. We are developing
simple tests using a small instrument and simple procedures that an
emergency room nurse can do. It will give better results with good
outcome at less cost in convenient way," says Choi.
The technology for diagnostic procedures involves a drop of blood,
saliva, urine, or serum on what looks like a small plastic card, five
centimeters square. It is inserted into a small instrument, which
gives the results in one minute. The equipment costs about $3,000.
"Test results will be converted to a digital signal off the chip,"
says Choi, "enabling the quantitative test results to be transmitted
between the point of test and medical experts through the use of wireless
or on-line technology."
Access Bio’s point of care test system contrasts with one made by
I-Stat, the two decade old company with 150 people on Windsor Center
Drive (www.i-stat.com). Choi explains that I-Stat focuses on convenience
and detects elements such as urea, sodium, potassium, chloride, and
glucose. "Detecting these kinds of materials they don’t need a
very sensitive system. In contrast, our market is to detect markers
that exist at very low levels in the blood. Few companies can make
point of care ultrasensitive test system."
The son of a Presbyterian pastor, Choi studied in Seoul at the Advanced
Institute of Science and Technology (Class of 1985), and then worked
as a project manager at Samsung’s R&D center. Moving to the United
States, he worked in New Jersey before establishing his own company
in September, 2001. He searched for incubator space on the Internet,
and last year moved into the Commercialization Center at the Technology
Center in North Brunswick. He and his wife, a web designer, and her
mother live in Montgomery Township and have three school age sons.
Choi is aiming for $15 million in sales in four years. Early revenue
might come from the blood alcohol screening tests. Tests that could
provide a higher profit margin might be diagnostics for diabetes,
cardiovascular disease, and neonatal care.
"The big companies keep focusing on the $500,000 instruments and
but we are seeking to carve out a different market," says Choi,
"testing in the physicians’ office and at home, and the Third
World." Currently under development are tests for such infectious
diseases as the hepatitis C virus, complete diabetes tests, and an
alcohol test for drunk drivers.
"We don’t have any venture capital yet, but we are talking to
some big companies," says Choi.
Access Bio is not quite the perfect poster child for TCNJ. If it were
perfect, it would have elected to expand within the Tech Center, where
there is plenty of available space. "The Tech Center is fine,
but we thought the larger spaces would not be good for production.
They are more expensive, and we also wanted to design our own space,
specific to us. Our CFO drove down Route 130 and found a new building
with a very competitive price."
Center of New Jersey, North Brunswick 08902. Yung Choi, CEO. 732-246-7400;
Robert Umpleby is working by himself in a unit at the
Commercialization Center at the Technology Center of New Jersey. His
company Semorex, based in Israel, believes its technology might help
with the following:
does not get absorbed into the bloodstream.
hypercholesterolemia, colon cancer, cholestatic liver disease, and
by Semorex, which is a shortened term for "selective molecular
recognition." Semorex has filed a number of patents to protect
its high affinity MIPs.
Molecularly imprinted polymers bind to molecules selectively, and
therefore they can be useful in detecting chemical weapons, says Umpleby.
"It is feasible to have an imprinted polymer that is selective
for one molecule but it can also be designed for certain class of
molecules," says Umpleby.
A polymer, he explains, is a large molecule made up of smaller repeating
molecules — the same molecule linked to itself over and over again,
as in plastic and rubber. Because of the physical and chemical properties
of polymers, it is difficult to work with them in the usual ways,
using small molecules. "Polymers can have properties that small
molecules by themselves don’t have," says Umpleby.
"Imprinted polymers are designed to bind with a specific molecule
or class of molecules. The polymer is made in the presence of the
target molecule. To make an imprinted polymer, you put the target
molecule in with monomers (the type of small molecule used to construct
a polymer). By judiciously selecting the monomers you can create a
polymer that is imprinted with the target molecule and convert the
monomer mixture into a polymer by using heat or ultraviolet radiation."
Semorex says its MIPs can work at lower concentrations and are more
chemically stable than natural antibodies. Also they may be manufactured
with less cost and more flexibility.
Its sensors can detect individual chemical warfare agents in water,
soil and air, and distinguish them from pesticides and related commercial
products which might be found in a normal environment. The MIPs might
also help find chemical toxins in the human body, help protect people
before a chemical attack, and be used with clean-up systems following
a chemical warfare attack.
The first generation of MIPs can selectively bind to toxins linked
to the onset of heart disease and several major cancers. Development
of these polymers is expected to facilitate new treatments for these
diseases, through the use of non-absorbable pills that cannot enter
the bloodstream. Another MIP can selectively remove toxic bile acids
in the GI tract, to treat patients with gastro-esophageal reflux disease
Umpleby grew up in Erie, Pennsylvania, where his father was an industrial
engineer at a steel forging company. Umpleby stayed in Erie to earn
an undergraduate chemistry degree from Gannon University and MBA from
Gannon. He had an accounting job in Pittsburgh before going to the
University of South Carolina to get his PhD in organic chemistry,
graduating last year. For this position he answered an ad placed in
a journal for molecular imprinting "but one of the founders had
already been in contact with my advisor at USC."
Founders are Bernard Green, a pharmaceutical chemist and pioneer in
catalytic antibody research at the Hebrew University of Jerusalem,
and CEO Morris Priwler, an Australian attorney and biotech entrepreneur.
Guenter Wulff, a leader in the field of molecularly imprinted polymers,
is on the scientific advisory board, as are two officers of Celgene
Corporation, Sol J. Barer and Jerome B. Zeldis, who recommended TCNJ’s
The company was founded in 2001, and though Umpleby is working by
himself in the incubator, he expects to have the company of two or
three other scientists by year’s end.
New Jersey, North Brunswick 08902. Robert Umpleby, research chemist.
732-545-7070; fax, 732-745-7270. Www.semorex.com
Paul Guo is living out his version of the American entrepreneurial
dream. He grew up on a farm in northeast China, earned his master’s
degree in Canada, and now owns AstaTech, a pharmaceutical service
laboratory that outsources much of its work to its economical offshore
lab in China. AstaTech does custom synthesis/manufacture and product
With 11 employees at Deer Park Drive plus the 40-person operation
in Chengdu, China, AstaTech draws pharmaceutical clients from North
America, Europe, and Japan. "In China they do basic research and
light production. Here we do the marketing, customer service, research
and process development, and custom synthesis," says Guo. "It
is a challenge."
Guo did his undergraduate degree in China and his master’s degree
at the University of Manitoba. He came to the United States in 1992
to work for an Eastman Kodak company in Pennsylvania and was a bench
scientist at Bristol-Myers Squibb on Route 206 before starting his
own firm in 1999. He and his wife, Rona, a scientist who also works
for the company, are naturalized citizens living in Hopewell with
their teenage daughter.
"We make the unique building blocks, advanced intermediates for
drug discovery," he says. "An intermediate requires 5 to 10
steps, with the first three steps made in China, and we make the rest."
The company has devised more than 1,000 unique drug-related products
that were synthesized in its own laboratories, kilo-lab and pilot
plants. AstaTech also collaborates with four other manufacturing companies
in China to be produce quantities of materials. One order might be
worth from $500 to $10,000.
"For our name, we took the first four letters of an elemental
chemical, astatium," says Guo, who started small, borrowing money
from friends, and commissioning a website from a friend. How big does
he want the company to grow? "As big as I can," says Guo.
Junction 08852. Paul Guo. 732-355-1000; fax, 732-355-1122. Home
John Fiorino moved his commercial analytical testing
laboratory from Lakewood to Princeton Corporate Plaza to acquire better
laboratory space. His company, Pelican Analytics LLC, is a contract
analytical chemistry laboratory that provides consulting and analytical
services to the pharmaceutical, chemical, specialty metals, and energy
Formed in 1999, Pelican Analytics focuses on determining precious
metals and analyzing materials such as high value catalysts and high
purity plastics for the types and amounts of metals that they contain.
"Our clients are largely specialty companies interested in the
highest accuracy, or trace and ultra trace elements," says Fiorino.
Fiorino’s wife of 31 years will work with him in the firm. With a
PhD in analytical chemistry from the University of Florida, she worked
at Bell Labs in materials science, batteries, corrosion and analytical
chemistry, and she has also been a product manager for the telecommunications
equipment. They live in Bridgewater with their two Shetland sheepdogs.
Fiorino grew up in Rochester, New York, graduated from Rochester Institute
of Technology in 1962, and has a PhD from Iowa State. He worked for
such companies as Kodak, Exxon, and Degussa, and he taught analytical
chemistry at Virginia Polytech, and the University of Florida.
He left Degussa, a German firm, to found this three-person firm. "I
have consulted for years, and the consulting has almost always had
an analytical component. The need and expertise was there — and
I couldn’t think of retiring. This is the most kind of fun a person
can have," says Fiorino.
To find his space, he did extensive networking. "We were offered
a shot at a space in Newark, but the environment here and the space
here was excellent," he says.
203, Monmouth Junction 08852. John A. Fiorino, director of technology.
732-274-2600; fax, 732-274-0800. Home page: www.PelicanAnalytics.com
The NJEDA’s Technology Center of New Jersey has 146,000
square feet of empty space. In addition to the tenants noted above,
the lineup includes Commercialization Center tenants Ortec International,
Aeropharm (now known as Kos), and Chromocell, plus such big companies
as Merial, Celgro, and Cambrex. Four of the 17 800-square foot laboratories
are available, as are 146,000 square feet of technology space in two
In contrast, the private landlord who owns Princeton Corporate Plaza
on Deer Park Drive has 250,000 square feet of R&D space that is mostly
leased and is building more. "Most of our R&D companies rent about
5,000 square feet, spaces that have been designed specifically for
their needs," says Harold Kent, the architect who designed the
first buildings on Deer Park Drive in 1988. "There is a lot of
synergy between the tenants — they exchange ideas and equipment."
Because most of the smaller tenants do want to expand, Kent is building
60,000 new square feet now. And because he wants to keep the pipeline
full, he will set up his own incubator in 10,000 square feet that
used to belong to Gold’s Gym (the fitness center that moved across
the street). By this summer there will be a place for 10 companies
to rent 1,000-foot fully furnished labs.
Transave Inc., which works on drug delivery for lung disease, can
be considered a poster child for Kent. Founded in 1997, it had 2,500
square feet in 2000, and with 17 full-timers recently expanded to
10,000 square feet. It offers inhalation therapeutics as an improvement
on injectables for treating lung disease because they cost less, provide
higher drug levels, and are easier to use. Transave’s lipid complexes
and liposomes can be released as a nebulized spray, a dry powder,
or an aerosol. Theradex, the firm at 14 Washington Road that manages
90 percent of the cancer trials in the United States, is in charge
of Transave’s clinical trials. After these trials, which should take
12 to 18 months, Transave will start trials on its anti-infective
Transave CEO Frank Pilkiewicz, who has raised nearly $16 million
in venture capital, will share the secrets of success on Friday, May
16, at 8 a.m. and 1 p.m. when Silicon Garden Angels hosts a Life and
Info Sciences venture capital and angels fair at the New Jersey Hospital
Association conference center (732-873-1955).
"We raised $12 million in second round funding and will use that
money to take our product through clinical studies," says Pilkiewicz.
Among his new investors is Sycamore Ventures at 5 Vaughn Drive. Leading
the second round were include Easton Hunt Capital Partners from New
York and ABN AMRO Capital from the Netherlands. Also CDIB Bioscience
of Taiwan plus first round investors Techno Venture Management (TVM)
of Boston and Munich and Musket Research Associates of Boston.
Says Pilkiewicz: "Happy is not the word, I’m ecstatic."
Junction 08852. Claire Strupinsky, manager of corporate administration.
732-438-9434; fax, 732-438-9435. Www.transaveinc.com
at Nassau Park.
at Princeton Theological Seminary.
and Heating Contractors.
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