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VOLUME 1 NUMBER 2 | SPRING 2011 Partnering with Michael J. Fox Investing in the Future Making it Personal SANFORD-BURNHAM MEDICAL RESEARCH INSTITUTE

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Join Sanford-Burnham Medical Research Institute, be a part of the quest to cure disease.

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Page 1: Spring 2011

VOLUME 1 NUMBER 2 | SPRING 2011

Partnering with Michael J. FoxInvesting in the FutureMaking it Personal

S A N F O R D - B U R N H A M M E D I C A L R E S E A R C H I N S T I T U T E

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Blair BlumSENIOR VICE PRESIDENT, EXTERNAL RELATIONS

Elizabeth Birlet, M.A.Stephanie Boumediene, M.P.H.Edgar GillenwatersPhilip Graham, M.B.A. Chris Lee, M.B.A.VICE PRESIDENTS, EXTERNAL RELATIONS

Elizabeth GianiniVICE PRESIDENT, GOVERNMENT RELATIONS

Kristina Meek, M.A.PORTAL EDITOR

Patricia FullerCONTRIBUTOR

Creative FusionDESIGN

ON THE COVERSanford-Burnham invests in the future, as illustrated by our campus in Lake Nona, opened in 2009. Read more on page 8.

Upcoming Events 1Discovering the Future 2A Message from Blair Blum 4Partnering with Michael J. Fox 5Talking with a Donor 6Talking with a Scientist 7Investing in the Future 8Planned Giving 9Recent Events 10External Relations Profile 12Scientists of Tomorrow 13Partners in Science Back Cover

FOUNDERSDr. William H. and Lillian Fishman

HONORARY TRUSTEESRoberta and Malin BurnhamJoe LewisConrad T. PrebysT. Denny Sanford

TRUSTEES AND OFFICERSGregory T. LucierCHAIRMAN

John C. Reed, M.D., Ph.D.CHIEF EXECUTIVE OFFICERPROFESSOR AND DONALD BREN CHIEF EXECUTIVE CHAIR

Kristiina Vuori, M.D., Ph.D.PRESIDENTPROFESSOR AND PAULINE AND STANLEY FOSTER PRESIDENTIAL CHAIR

Gary F. Raisl, M.B.A., Ed.D.EXECUTIVE VICE PRESIDENTCHIEF ADMINISTRATIVE OFFICERCHIEF FINANCIAL OFFICERTREASURER

Margaret M. DunbarSECRETARY

Arthur BrodyShehan Dissanayake, Ph.D.M. Wainwright Fishburn, Jr.Pauline M. FosterDavid F. HaleJeanne Herberger, Ph.D.Brent JacobsJames E. Jardon IIRobert J. LauerRobert A. MandellNicolas C. NierenbergDouglas ObenshainPeter PreussStuart TanzJan Tuttleman, Ph.D., M.B.A.Andrew J. Viterbi, Ph.D.Carl Ware, Ph.D.Bobbi WarrenAllen R. WeissGayle E. WilsonDiane Winokur

EX-OFFICIORaymond L. White, Ph.D.SCIENCE ADVISORY BOARD CHAIRMAN

Sanford | Burnham Medical Research Institute10901 North Torrey Pines Road, La Jolla, CA 92037 • 858.646.3100

Sanford | Burnham Medical Research Institute at Lake Nona6400 Sanger Road, Orlando, FL 32827 • 407.745.2000

Sanford | Burnham Medical Research Institute at Santa Barbara2324 Life Sciences Building, University of California, Santa BarbaraSanta Barbara, CA 93106 • 805.453.0259

www.sanfordburnham.org Toll-free: 1-877-454-5702

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UPCOMING EVENTS

Sanford-Burnham invites you to Bring It! April 21 in San Diego. This gameshow-themed experience is filled with wacky trivia and fearless displays of hidden talent.

Proceeds will support stem cell research for victims of spinal cord injury. Tickets and sponsorships are available by calling Chelsea Luedeke at 858-795-5239. Visit www.sanfordburnhamevents.org/bringit for more information.

Save the date for this President’s Circle event to be held in La Jolla. President’s Circle members are those donors who contribute $1,000 or more annually. Stay tuned for details!

APRIL 21, 2011

JUNE 5, 2011

Bring It! Rock On for Stem Cell Research

President’s Circle

Stay up-to-date about our events by following us on Twitter: @SBI_Events.

A contestant rocks out at Bring It! Orlando.

Long Live

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PORTAL | www.sanfordburnham.org2 PORTAL | www.sanfordburnham.org2

As we move forward, the Institute is intensifying its focus on translational medicine. In November, we introduced the Art Brody Innovation Fund, which provides strategic funding to optimize Sanford-Burnham discoveries for development into drugs and diagnostics. In December, along with Florida Hospital, we began a new collaboration with Takeda Pharmaceutical to develop drugs that target obesity (more on page 8). In January we announced a partnership with drug maker Ortho-McNeil-Janssen Pharmaceuticals (Johnson & Johnson) to develop medicines for Alzheimer’s and neuropsychiatric disorders. These developments are just the beginning; research being conducted today has the potential to challenge disease and revolutionize human health.

The Institute has been blazing trails and achieving scientific breakthroughs since its founding in 1976. Our publications were cited more often in peer-reviewed biology and biochemistry journals between 1999 and 2009 than any other organization worldwide. Sanford-Burnham research has led to more than 600 patents and generated more than 90 agreements with strategic partners.

The Institute has grown geographically as well. The Vascular Mapping Laboratory led by Dr. Erkki Ruoslahti, and the Center for Nanomedicine, led by Dr. Jamey Marth, are located in Santa Barbara. The Institute’s newest facility, the Lake Nona campus, is at the heart of Orlando, Florida’s burgeoning “Medical City.” It is home to the Institute’s Diabetes and Obesity Research Center, headed by Dr. Dan Kelly.

The future looks even brighter. In just the past few months, researchers have created a peptide (a piece of

Discovering the FutureDuring 2011 we invite you to join us in celebrating the Institute’s 35th anniversary. This year, we will celebrate our achievements, which were made possible, in part, by our donors, and envision what the next 35 years might hold. By becoming involved with Sanford-Burnham you can help us discover that future.

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www.sanfordburnham.org | PORTAL 3

a protein) that makes tumors more permeable to treatment, gained new insights into diabetic heart disease, learned how muscle stem cells could be used to treat muscular dystrophy, investigated ways to attack cancer at its roots by targeting cancer stem cells, and much more. In 2010, Sanford-Burnham researchers produced more than 300 peer-reviewed papers.

Please visit our blog at http://beaker.sanfordburnham.org, updated frequently, to learn about many other developments.

We can only imagine how researchers will continue to build on what we have already learned. Picture the world 35 years from now, in 2046, and how different life might be for people living with diseases. Several diseases that plague us now will very likely be eradicated.

This anniversary will be marked by celebration. We will be letting you know about exciting events and philanthropic opportunities, so keep in touch.

Picture the world in 35 years, in 2046, and how different life might be for people living with diseases.

T. Denny Sanford and Malin Burnham, the two dedicated philanthropists and friends for whom the Institute is named, are men of vision—always looking to the future.

Imagine for yourself how you would like to see the future of medical research. Be a part of it here at Sanford-Burnham.

www.sanfordburnham.org | PORTAL 3

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It has been more than a year since the Burnham Institute for Medical Research underwent its transformation to Sanford-Burnham Medical Research Institute. The change was prompted by the receipt of an unprecedented $50 million gift from T. Denny Sanford, in response to our $100 million, ten-year fundraising goal. It is largely thanks to donors that our scientists are able to make such an impact on human health.

This year, the Institute celebrates 35 years of success, and we are looking ever forward. We are blazing trails from innovative laboratory research to life-changing treatments and cures for patients.

In this issue we recognize all of the donors who gave in 2010. We are humbled by the length of this list, but know that there is always room for our community of supporters to grow. I offer my sincerest thanks to all those who have donated and who will continue to do so in the future. I hope to see many of you at upcoming Institute events. New discoveries will continue to unfold at Sanford-Burnham, and there is virtually no limit to what can be achieved. Please join us in our quest to challenge disease and revolutionize human health. Thank you.

Blair BlumSenior Vice President, External Relations

A Message from Blair Blum

SAVE THE DATE FOR OUR

35TH Anniversary Gala Mining for the Cure October 15, 2011 | La Costa Resort and Spa | Carlsbad, California

PORTAL | www.sanfordburnham.org4

“I offer my sincerest thanks to all those who have donated and who will continue to do so in the future.”

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Dr. Stuart Lipton recently received a grant from the Michael J. Fox Foundation. The opportunity to be associated with this high profile foundation working to eradicate Parkinson’s disease (PD) is a great honor. Our commitment is to raise $106,000 in philanthropic support. By helping us meet this goal, you can be a part of this remarkable partnership.

Founded by the actor well known for roles on television’s Family Ties and the Back to the Future movie trilogy, the Michael J. Fox Foun-dation is highly selective. In a grantee, the Fox Foundation looks for “the ideas that are most readily translatable into new treatments, the teams that can execute those ideas, thoughtful and realistic work plans, and outcomes that can take us closer to our goal: delivering bet-ter treatments and, ultimately, a cure.”

Dr. Lipton, director of the Institute’s Del E. Webb Neuroscience, Aging and Stem Cell Research Center, received the grant for a study titled Qualitative Analysis of S-Nitrosylated Proteins. His co-principal investigator for the project is colleague Dr. Tomohiro Nakamura. The research team will study how free radicals contribute to PD. Free radicals are highly reactive chemicals that attack molecules by capturing electrons and thus modifying their chemical structures. In this case, they cause critical proteins to malfunction.

The goal of the study is to demonstrate changes in specific proteins in PD brains. The study could reveal new disease biomarkers and novel therapies.

Dr. Lipton is a highly accomplished researcher. His laboratory’s achievements

include the development of Memantine, the most recent therapeutic to be approved for treatment of Alzheimer’s disease. Under Dr. Lipton’s leadership, Sanford-Burnham was selected by the NIH and NIEHS as one of three national centers of excellence in neurodegenerative diseases research.

While Sanford-Burnham holds an impressive track record for securing federal grant funding for its research—at a time when that funding is ever more scarce—grants from private foundations can propel research in specific areas. Our association with this highly respected foundation not only increases Sanford-Burnham’s profile as a leader in seeking cures for PD, but may one day solve the riddle of this debilitating disease. Your support will ensure that this study reaches its full potential.

Join us! To support this promising project, please use the envelope enclosed in this issue of Portal, call us at 1-877-454-5702, or e-mail Jane Langer at [email protected]. To learn more about Dr. Lipton’s work, please visit www.sanfordburnham.org/lipton.

Partnering with Michael J. Fox

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The Future ofPhilanthropy:

LindyHumber

In thinking about the future of a non-profit organization like Sanford-Burnham, one wonders how—or even if—the next generations will participate in philanthropy. Will they be as generous with their time, talent and treasure as their forbearers? Those who are now in college or just entering the workforce have the power to help build a future with more freedom from disease, and there is reason to hope that they recognize this potential.

Carolyn “Lindy” Humber has a promising future ahead of her, both in her budding professional career and as a philanthropist. She graduated last spring, on the Dean’s list, from Boston College, where she studied psychology and neuroscience. Lindy recently toured the Lipton lab at Sanford-Burnham. Stuart Lipton, M.D., Ph.D. is the director of the Del E. Webb Neuroscience, Aging and Stem Cell Research Center and Program Director for Neurodegenerative Diseases.

“Dr. Lipton’s lab blew me away!” Lindy recalls.

Having grown up in a philanthropic family, Lindy’s experience with Dr. Lipton inspired her to make a gift to the lab. “I’ve been incredibly fortunate in that I’m able to donate to foundations and organizations. Giving has been a large part of my life,” she says. Lindy’s parents, Kent and Candace Humber, were so pleased with their daughter’s generosity that they matched her gift through the Rancho Santa Fe Foundation.

Lindy elaborated, “Dr. Lipton is brilliant and the research he’s conducting on Alzheimer’s and autism is a necessity. I trust in his research and know that it is some of the most advanced in the field. It’s criminal when discoveries and innovations as crucial as Dr. Lipton’s are cut short because of funding issues.”

Lindy believes that her peers are no different from people of any age when it comes to giving. “People tend to donate more when they can relate to a cause directly,” she says. If you are interested in getting to know a researcher or exploring Sanford-Burnham firsthand, we can arrange it. Please contact us and be part of the future that will benefit Lindy’s generation and those still to come.

Of the 51 million members of Generation Y in America, more than half are giving to charity, according to a 2010 study by Convio.

PORTAL | www.sanfordburnham.org

TALKING WITH A DONOR

6

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Dr. Freeze is a glycobiologist – he studies sugars and how they alter protein function in the cell. He is especially interested in a group of rare diseases called Congenital Disorders of Glycosylation (CDG). “There was very little training in medical schools for glycobiology,” he recalls, “so when patients started to turn up with these diseases there was no place for these physicians to go. They were forced to come to the basic researchers. That’s pretty different.”

His office is adorned with photos of children. “These are my kids,” he says proudly. In the case of Rocket, Dr. Freeze began a relationship with the child’s family that continues today, bringing hope to other families.

After Dr. Freeze diagnosed Rocket’s CDG, the boy’s parents paid him a visit. “They asked ‘is there a therapy’? and I said ‘no,’ and then they shifted, rather unexpectedly to ‘what are the big questions in the field that need attention?’ So I talked about that, and his mom said, ‘what would it take to get that going?’”

With that, the seeds were planted for what is now The Rocket Fund. At Sanford-Burnham’s recent Rare Disease Symposium,

Dr. Freeze announced the official launch of the expanded Rocket Fund, which will enable donors to direct their gifts to research on rare childhood diseases including CDG.

Caring for children comes naturally to Dr. Freeze. Growing up with a developmentally delayed sister inspired him to help disabled children. “My dad once said to me that I always said I wanted to help kids like my sister, and I said I’d forgotten that, and he said ‘Well, you were eight years old.’ So there was a foundation there for what I do today.”

Over 30 million Americans are affected by “rare” diseases, many of which strike in childhood. When asked to imagine what might be accomplished in rare disease research over the next 35 years, Dr. Freeze answered, “I think we’ll have discovered the causes of most rare diseases. We may not have treatments for all of them, but the origin of the treatments will come from people continuing to explore the basic science.” Understanding the cause of disease is the first major hurdle to treating it, and your support of the Rocket Fund can speed that understanding.

TALKING WITH A SCIENTIST

Basic researchers may understand the inner workings of a human body as well as anyone, but it’s not often they meet the person that inhabits that particular body. Initally, all Dr. Hudson Freeze first knew of John Taylor “Rocket” Williams III was a few cells. The cells were sent to Dr. Freeze by the young boy’s pediatrician, who needed the researcher’s help with a difficult diagnosis.

Making it Personal: Dr. Hudson Freeze

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Sanford-Burnham invests in the future, and nowhere is that illustrated more clearly than at our Lake Nona campus in Orlando, Florida. A recent visit by Florida Governor-elect Rick Scott, and a new partnership among Sanford-Burnham, Florida Hospital and Takeda Pharmaceutical to discover obesity drugs, illustrate how Sanford-Burnham is shaping tomorrow.

Rick Scott visited the Lake Nona campus in December 2010, wrapping up a five-day statewide tour to discuss jobs and economic development. Speaking about the power of collaboration and teamwork that helped Florida attract Sanford-Burnham to open its East coast campus in Orlando in 2007, the governor-elect observed “a win-win attitude characterized by people who get things done.” More than 200 business and civic leaders attended the town hall-style meeting in our auditorium. The discussion focused on the job opportunities being created at the emerging life science cluster in Orlando’s Medical City, which is anchored by Sanford-Burnham.

Trustee Jim Jardon said, “We were honored to have Governor-elect Scott choose us as the site from which to address his future plans for the state. It is fitting, since Sanford-Burnham is at the epicenter of the blossoming Medical City in Central Florida, the research leader and a catalyst to future discoveries and cures.”

Dr. Daniel Kelly, scientific director of Sanford-Burnham at Lake Nona, showed the governor-elect around the campus and explained his research on energy metabolism in muscles,

Investing in the FutureA view from Lake Nona

Dr. Dan Kelly showed Governor-elect Rick Scott around the Lake Nona facility.

“Our scientists are world-class at identifying targets for drug discovery, devising creative strategies for tackling diseases, and beginning the process of translating our findings toward clinical applications. But we look to biotechnology and pharmaceutical companies to finish the job.”

— CEO Dr. John Reed

Continued on Page 12

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What impact would you like your planned gift to have on the future?

Planned Giving in Support of Sanford-Burnham

“Their hard work is helping to discover cures with potential to touch us all, either personally or through a family member or friend. My hope is that through my efforts and financial support I can make a difference in improving the health and well being of others, as well as saving people’s lives.” — Alan Gleicher

“I lost the love of my life, Phyllis, to Alzheimer’s disease. I want to keep her memory alive, so I have chosen to leave an estate gift to Sanford-Burnham to support scholarships for young scientists in search of a cure.” — Mel Clause

“We lost my wife Marge this year after an eight- year battle with lung cancer. We established the Morrice Family Innovation Fund at Sanford-Burnham several years ago to support cancer research. We fund it with annual gifts of cash, which will be supplemented by a gift of an IRA account at the end of my life’s journey.” — Bruce Morrice

The Gleicher Family

Phyllis and Mel Clause

Marge and Bruce Morrice To discuss a planned gift please contact Patty Fuller at (858) 795-5231.

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Sanford-Burnham has been busy as usual, welcoming friends and supporters at a variety of events. Here are a few highlights.

Recent Events

Officials from the City of Goleta, CA, where the University of

California, Santa Barbara (UCSB) is based, visited the Center for Nanomedicine. The Center is a collaboration

between Sanford-Burnham and UCSB. Visitors marveled

at a demonstration of 3-D imaging technology inside the

AlloSphere, the only facility of its kind.

Members and residents of Lake Nona

Country Club were invited to an intimate

event at the Institute’s Orlando facility in

February. Dr. Kristiina Vuori spoke

about the work the Institute is doing in

cancer research, and the Florida Breast Cancer Foundation presented grants to

two postdoctoral fellows, Preeti Bharaj, Ph.D.

and Fangfei Li, Ph.D.

Dr. John Reed recapped his State of the Institute address, previously given only to employees, for

members of the President’s Circle on

January 27. He reviewed highlights of

an extraordinarily productive year and

outlined the goals of the Institute for

2011 and beyond.

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The Honorable Eric Shinseki, Secretary, U.S. Department of Veterans Affairs, recently visited

the expansive V.A. construction project at Lake Nona’s “Medical City” in Orlando, Florida. While

visiting the emerging life science hub, the Secretary toured the Sanford-Burnham laboratories.

Recent Events

Forty-seven runners participated in this year’s Walt Disney World Marathon as part of

Team Sanford-Burnham. Each runner was challenged to raise $2,500 for medical research.

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Elizabeth Birlet assumed the position of Vice President, External Relations in December. Based at Sanford-Burnham’s Lake Nona location in Orlando, she leads the Institute’s East Coast philanthropic fundraising operation.

Ms. Birlet brings a strong background in philanthropy and has held senior positions over the past 20 years at academic research institutions across the nation, including the University of California, San Diego, the University of Chicago and the University of Illinois at Urbana-Champaign. Most recently, she was Director of Investor Philanthropy for the Allen Institute for Brain Science in Seattle. She has a master’s degree in journalism with a focus on biomedical science from the University of Illinois.

Ms. Birlet has had a busy first few months getting to know many of Sanford-Burnham’s supporters and becoming acquainted with leaders in Orlando’s civic and business communities. If you have the opportunity to visit the Lake Nona campus or attend any of the Institute’s Orlando events, please say hello to her! She is an avid tennis player and gardener and looks forward to doing both in Florida.

and how this research is yielding insights into the obesity problem that currently plagues our nation.

On December 28, Sanford-Burnham announced a partnership with Florida Hospital and Takeda Pharmaceutical to investigate obesity, a growing worldwide health problem. The partnership leverages the three organizations’ strengths in basic biomedical research, clinical research, and drug development to identify obesity-related biomarkers and other targets with therapeutic potential.

Only through collaboration can Sanford-Burnham’s groundbreaking research be translated into new therapies. As CEO Dr. John Reed said in a recent interview with The San Diego Union-Tribune, “Our scientists are world-class at identifying targets for drug discovery, devising creative strategies for tackling diseases and beginning the process of translating our findings toward clinical applications. But we

look to biotechnology and pharmaceutical companies to finish the job.”

The partnership is the first corporate-sponsored research undertaken by Sanford-Burnham at Lake Nona. “This research partnership is a collaborative model that capitalizes on the synergistic expertise of each group” said Dr. Kelly.

Takeda Pharmaceutical officials presented Sanford-Burnham and Florida Hospital scientists with a “Daruma,” a traditional Japanese doll that symbolizes good luck and perseverance, to commemorate their newly formed research partnership.

Continued from Page 8

Investing in the Future

External Relations Profile: Elizabeth Birlet

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Dr. Hansen is a Principal Investigator—that means she runs her own lab—who has been working at Sanford-Burnham for three years. She works with a team of scientists from all over the world. Canada, Germany, Denmark, Taiwan, China, and the U.S. are all represented in Dr. Hansen’s lab.

Together, the team studies aging. You probably know that when people get older, their bodies change and they are more likely to get diseases like cancer or Alzheimer’s disease. Worms get old too, only they do it much faster. C. elegans only live two to three weeks. That means the scientists can more quickly watch a lot of worms go through their whole life cycle. Even more convenient, the worms are transparent. So, under the microscope, the scientists can see what is going on inside them.

Dr. Hansen and her team make changes, or mutations, to certain genes in the worm. When they change these specific genes, the worms’ aging process works differently, and sometimes they live up to twice as long!

SCIENTISTS OF TOMORROW

Mutant Worms

Each issue of Portal will include an article written for middle school to high school-aged students. Please share this article with the youth in your life, and help us encourage the scientists of tomorrow.

What if you got to work with worms every day? Malene Hansen, Ph.D., a scientist at Sanford-Burnham not only gets to work with them, she mutates them. That might sound a little strange, but she and her team could discover something in a tiny worm called C. elegans that could teach us something about human beings.

The proteins Dr. Hansen studies in these worms are similar to proteins in people. By studying C. elegans, researchers can potentially teach us more about how people age. Scientists and medical doctors might one day use this knowledge to help people stay healthier as they get older, and maybe even live longer.

When you work in a research lab, you learn and explore little by little, day by day. “The implications are huge, but the steps we take toward them are usually small,” Dr. Hansen tells us. It is important to keep asking questions and sharing ideas. Dr. Hansen says, “Arguably, the most important trait for a scientist is curiosity.”

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Nonprofit OrganizationU.S. Postage

PAIDSanford-BurnhamMedical Research Institute

10901 N. Torrey Pines Road, La Jolla, CA 92037

San Diego businessman and philanthropist Art Brody pledged $1 million to help support Sanford-Burnham’s efforts to translate basic research discoveries into new medicines, creating the Arthur Brody Innovation Fund.

Dr. Chris Hassig, Director, Drug Discovery and Dr. Michael Jackson, Vice President of Drug Discovery & Development, recently met with Mr. Brody in the Conrad Prebys Center for Chemical Genomics.

This facility offers access to advanced drug discovery technologies. By testing hundreds of thousands of chemicals using the Center’s robots, researchers discover promising new medicines very quickly. Mr. Brody’s support will help leverage these early-stage discoveries to develop the next generation of innovative new medicines.

PARTNERS IN SCIENCE

Art Brody, Dr. Chris Hassig & Dr. Michael Jackson