Realizing The Promise by The New York Stem Cell Foundation

T H E N EW YO R K ST E M C E L L FO U N DAT I O N R ES E AR C H I N ST I T U T E R EA L I Z I N G T H E P ROM I S E

THE FUTURE IS NOW Stem cells offer the greatest hope for advancing medical research that we have ever known. This new research frontier holds the promise of cures for such diseases as ALS, Parkinson’s, Alzheimer’s, diabetes, multiple sclerosis, heart disease, cancer, macular degeneration, and spinal cord and other traumatic injuries, in addition to a vastly improved platform for the discovery and testing of vital new drugs. NYSCF provides leadership and vital support for the most advanced forms of stem cell research by conducting and funding translational research focused directly on curing diseases. Several of the most important breakthroughs in human stem cell research in the past decade were made possible by NYSCF. Within a year of founding the NYSCF Research Institute, NYSCF scientists created the first patientspecific stem cell lines for ALS (Lou Gehrig’s disease), which has led to several important clinical trials. Since then, NYSCF researchers have pioneered the production of stem cells from adult cells through merging patient DNA with egg cells, created human stem cell models of type 1 diabetes and Parkinson’s disease, generated human bone, and discovered a technique to prevent the inheritance of mitochondrial diseases, deadly and debilitating illnesses. Now, more than ever, NYSCF is a critical catalyst in the world of stem cell research. As we mark ten years of accomplishment, please join with us in continuing to realize the extraordinary promise of stem cell research. With your help, we can ensure that science breaks through to find cures for the diseases affecting those we love.

Sincerely,

Susan L. Solomon CEO & Co-Founder

Roy Geronemus, MD Chairman of the Board

OUR MISSION The mission of The New York Stem Cell Foundation Research Institute is to accelerate cures for the major diseases of our time through stem cell research. The New York Stem Cell Foundation (NYSCF) combines the depth of a highly focused research institute with the breadth of a wide-ranging philanthropic organization. Since its establishment in 2005, NYSCF has invested over $150 million in stem cell research and continues to conduct the most advanced stem cell research internationally, both in its own laboratory in New York City, and also in collaboration with major medical research institutions around the world. As a privately funded, independent stem cell research laboratory, NYSCF is uniquely positioned to help close the gap that exists between basic research and improved treatments for patients by advancing outside research programs and creating new technological platforms for use within the larger research community. Through specific programs focused on using human cells to vastly improve identification of new drug targets and accelerate drug screening, as well as through the development of the NYSCF Global Stem Cell Array technology, NYSCF connects research to cures. The exceptional, global NYSCF Innovator community continues to grow, now comprising over 130 scientists worldwide. In addition to the 45 researchers in the NYSCF Research Institute, the NYSCF Innovators include 52 of todayâ&#x20AC;&#x2122;s brightest young stem cell researchers through the NYSCF Fellowship Program, and an additional 37 of the most talented early career scientists through the NYSCF Investigator Program. In addition, NYSCF educates the public about the promise of stem cell research through public panels and talks, and convenes annually the preeminent international conference on translational stem cell research for the scientific community.

OUR UNIQUE APPROACH With cures as the priority, NYSCF is unique in the world of medical research as both a funder of leading stem cell scientists around the world and also as the operator of its own state-of-the-art private research laboratory which is unencumbered by institutional bureaucracy and government restrictions. By both conducting and also supporting high-risk, high-reward, “tipping point” experiments that are not backed by traditional funding models, NYSCF helps bring the entire field of human stem cell research forward. With the freedom to pursue cutting-edge breakthroughs, NYSCF concentrates on diseases where stem cell research shows the most promise. By setting research initiatives with a clearly focused clinical mandate, NYSCF advances cures and new treatments in such areas as: • Bone engineering • Cancer • Diabetes • Heart disease • Macular degeneration and other forms of eye disease and blindness • Multiple sclerosis • Neurodegenerative diseases, including ALS, Alzheimer’s, and Parkinson’s • Schizophrenia, autism, depression, and other mental health disorders • Spinal cord injury and other traumatic injury

ACCELERATING STEM CELL RESEARCH

NYSCF RESEARCH INSTITUTE The NYSCF Research Institute, NYSCF’s own translational stem cell laboratory, opened its doors on March 1, 2006. Today, 45 researchers conduct the most advanced human stem cell science with a collaborative and translational focus. The integrated NYSCF laboratory and its interactive culture enables scientists to quickly and easily share results and strategies, leading to new thinking and breakthroughs within and across diseases. NYSCF collaborates extensively with disease foundations, government agencies, pharmaceutical, technology, and biotech companies, as well as academic institutions around the world to advance the discovery of new disease treatments. Major NYSCF collaborators include: • The Stanley Center at the Broad Institute of MIT and Harvard • Cure Alzheimer’s Fund • National Institutes of Health (NIH) Undiagnosed Disease Program • Personal Genomes Project In the past seven years, NYSCF’s research has been named the #1 medical breakthrough of the year by Science and twice by Time magazine.

“NYSCF is a unique organization as they not only develop innovative technologies, but also collaborate closely with scientists around the world, including those in my laboratory, to advance medical research in pursuit of cures.” Marc Tessier-Lavigne, PhD President, The Rockefeller University

PIONEERING TECHNOLOGY TO ACCELERATE CURES

NYSCF GLOBAL STEM CELL ARRAY The NYSCF Global Stem Cell Array is a revolutionary robotic technology, developed by NYSCF, that automates and standardizes the production of stem cell lines and adult cell types. The NYSCF Array technology enables scientists to functionalize human genetics for the first time by merging data from the human genome with our own biology—accelerating new therapies and cures for patients using actual human cells affected by disease. This one-of-a-kind automation technology allows NYSCF to: • Standardize and scale up production of identical induced pluripotent stem (iPS) cells, which are stem cells made from an individual’s own skin or blood cells. • Study iPS cells from thousands of patients, reflecting the genetic diversity of the global population and enabling scientists to detect patterns that are only discernable when examining a large population of patients, and which show how different genetic mutations impact individual disease symptoms. • Conduct clinical trials in a petri dish using the actual human cells that get sick — from patients rather than from surrogate models (e.g. animals). • Develop more effective, safer, and less expensive new drugs to target the causes of disease by factoring in genetic differences during the development process. This revolutionary approach will help “de-risk” projects that pharmaceutical companies would typically avoid.

The NYSCF Global Stem Cell Array fuses big data and the power of stem cell biolog y to bring us closer to achieving precision medicine — ultimately, individually tailored treatments and cures.

A GLOBAL COMMUNITY CHANGING THE WORLD

NYSCF INNOVATORS The NYSCF Innovator community now encompasses more than 130 scientists from around the world. Many of them work at the NYSCF Research Institute, while others are supported by NYSCF through its external funding programs. NYSCF FELLOWSHIP PROGRAM The NYSCF Fellowship Program supports the most talented young scientists at critical junctures in their careers. The competitive Fellowship Program funds scientists at an early stage of their careers, creating and promoting a global community of over 50 of today’s leading stem cell researchers. These are the scientists that are changing the world.

NYSCF INVESTIGATOR PROGRAMS The NYSCF Stem Cell and Neuroscience Investigator Programs provide five years of critical seed funding to outstanding early career scientists, supporting them as they move beyond their postdoctoral training to establish their own independent research laboratories, further accelerating the path of research from bench to bedside. These scientists are the engineers of tomorrow’s breakthroughs.

NYSCF–ROBERTSON STEM CELL PRIZE An annual award honoring the most significant achievement in stem cell research by an early career scientist. The NYSCF–Robertson Stem Cell Prize honors, supports, and encourages the most significant contributions from a young scientist to the field of stem cell research with a cash award to further their research.

Support for the NYSCF Innovators comes from Fiona and Stan Druckenmiller, The Ralph and Ricky Lauren Family Foundation, and The Robertson Foundation.

NYSCF OUTREACH NYSCF brings the latest cutting-edge translational stem cell research to audiences around the world, helping to ensure that scientists, clinicians, policy leaders, and the general public have a forum for straightforward, accurate and unbiased information about human stem cell research and its potential for advancing treatments and cures. ANNUAL CONFERENCE AND SYMPOSIA NYSCF sponsors The NYSCF Conference, an annual translational stem cell research meeting each autumn in New York City that is one of the preeminent global forums for the leading minds in stem cell research. NYSCF also hosts a yearly slate of ongoing panel discussions, online webinars, and symposia that drive new ideas and bring diverse groups together to share new research and address critical ongoing challenges. • 450+ scientific attendees • 30+ leading speakers from around the world • 15+ countries represented

INITIATIVE ON WOMEN IN SCIENCE AND ENGINEERING NYSCF’s Initiative on Women in Science and Engineering (IWISE) helps address gender equality in science, medicine, and engineering. The IWISE working groups bring together a diverse group of women and men representing the leaders in the science, technology, and medical fields, to go beyond simply reflecting on issues of gender representation by outlining a course of action to improve this imbalance. The NYSCF IWISE working group published a forum article in Cell Stem Cell in spring, 2015, entitled “Seven Strategies for Advancing Women in Science, Engineering, and Medicine,” outlining actionable and implementable solutions and strategies to encourage women to enter these fields and foster and support them so that they stay, thrive, succeed, and excel in their careers.

SCIENCE AND TECHNOLOGY EDUCATION PROGRAM The NYSCF Science and Technology Education Program (STEP) has introduced over 1700 high school and middle school students to the exciting world of science and stem cell research via: • NYSCF Academy An ongoing educational seminar series provided to middle and high school students by NYSCF Scientists • NYSCF University The NYSCF internship program that provides young students the opportunity to see and do stem cell research first hand • World Stem Cell Day A nationally recognized day to raise awareness about stem cell science in which NYSCF conducts special educational outreach

A DECADE OF SUCCESS NYSCF has an extraordinary track record for a young organization. Since its founding, NYSCF has already achieved numerous top medical breakthroughs to bring us closer to the cures we need. 2005 NYSCF founded Frustrated by lack of government support and inspired by the potential of stem cells to transform medical research, Susan L. Solomon co-founds NYSCF to encourage private support for the most innovative and promising stem cell science.

2006 NYSCF opens lab doors NYSCF opens first completely privately funded “safe haven” laboratory in New York, free from the restrictions tied to federally funded research.

2008 First-ever patient-specific disease model With NYSCF’s support, the first model of a disease is created using a patient’s own cells; the development of ALS as a “disease in a dish” is named the major scientific breakthrough of the year by both Time and Science magazines.

2011 Cell replacement therapy from patient’s own cells NYSCF’s lab makes another Time “#1 medical breakthrough of the year,” a major advancement toward developing a patient’s own stem cells for cell replacement therapies.

2012 Clinical cure for mitochondrial diseases NYSCF scientists develop a technique to prevent devastating mitochondrial diseases that are passed from mother to child (in 2015, the technique is approved for clinical trials in the United Kingdom).

2012 Robotic technology to produce stem cells for research NYSCF begins test operation of the NYSCF Global Stem Cell Array, a unique robotic technology that automates the production and differentiation of stem cell lines. This allows scientists for the first time to mass-produce high-quality stem cell lines for research and discovery purposes.

2013 New therapeutic approach for diabetes NYSCF scientists discover a compound that restores beta cell function in a genetic form of diabetes, which holds forth the potential of being applicable to all forms of the disease.

2013 First-ever personalized bone from patients own skin cells NYSCF scientists make pieces of bone from stem cells derived from a patientâ&#x20AC;&#x2122;s skin cells.

2014 Accelerating multiple sclerosis research NYSCF generates pluripotent stem cells from MS patients and develops a new method to make the cells affected by the disease that cuts research time in half.

2014 Creating Parkinsonâ&#x20AC;&#x2122;s disease in a dish NYSCF laboratory creates a stem cell model of Parkinsonâ&#x20AC;&#x2122;s disease and identifies potential therapeutic targets for the disease, a first step in the discovery of new drugs.

2014 First-ever patient-specific cells from a patient with Type 1 diabetes NYSCF creates the first patient-specific embryonic stem cells and insulin-producing beta cells from a Type 1 diabetes patient.

2014 Modeling familial and sporadic Alzheimerâ&#x20AC;&#x2122;s disease The NYSCF lab creates a stem cell model of Alzheimerâ&#x20AC;&#x2122;s disease and identifies gene targets to better diagnose and find therapies for the disease.

2015 Stem cells from a few drops of blood NYSCF develops a method for generating pluripotent stem cells from small amounts of frozen umbilical cord and peripheral blood.

2015 Automated, standardized, stem cell production The NYSCF Global Stem Cell Array robotic platform published in Nature Methods shows 12-fold increase in productivity and less variability than previous methods of making iPS cells.

NYSCF CLINICAL TRIALS In 2015, three clinical trials enabled or supported by the New York Stem Cell Foundation began. Each had its roots in NYSCF’s research. • In 2007, NYSCF began supporting research at Harvard University to model ALS, Lou Gehrig’s Disease, using stem cells. NYSCF continued supporting this research and it culminated in the discovery that a drug on the market for epilepsy may be protective of motor neurons, the cells affected in ALS. As of 2015, Massachusetts General Hospital began recruiting ALS patients for clinical trails to test its efficacy. • In 2011, Dr. Pete Coffey was awarded the inaugural NYSCF-Robertson Stem Cell Prize for his work to bring stem cell therapies to patients with macular degeneration. Dr. Coffey announced in September 2015 that the first patient received treatment for “wet” age-related macular degeneration using eye cells made from stem cells. The treatment is the first in a clinical trial exploring the safety and efficacy of the stem cell derived patch transplant. • In 2012, scientists at NYSCF developed a technique to swap the DNA from one woman’s egg to another. This technique, similar to in-vitro fertilization, prevents the mother-to-child transmission of mitochondrial diseases, rare but often fatal diseases. In 2015, this technique was approved for clinical trials in the United Kingdom for the prevention of mitochondrial diseases and NYSCF is working with the FDA and advocacy organizations to get this approved for use in the United States.

“The New York Stem Cell Foundation is an important accelerator of scientific research in New York and around the world, connecting academic, industry, and patient groups. We are proud to partner with the Foundation in its efforts to push the field towards the cures that patients need.” Laurie H. Glimcher, M.D. Stephen and Suzanne Weiss Dean, Weill Cornell Medicine 21

URGENT TODAY, EVEN MORE SO TOMORROW WHY IS PRIVATE SUPPORT STILL NEEDED FOR STEM CELL RESEARCH? Private philanthropy can be nimble in ways that governments or large institutions cannot. Government support, even when it is available, is slow and encumbered by bureaucracy. Because of its private philanthropic support, NYSCF is free to put science first, supporting the most urgent high-risk/high-reward research. Finding cures is our only priority. NYSCF aims to enable the most innovative research, and to ensure that it takes place now.

WHY SHOULD I SUPPORT NYSCF IF I ALREADY GIVE TO A MAJOR MEDICAL RESEARCH INSTITUTION? A gift to NYSCF leverages your existing commitments to other medical research and academic organizations. NYSCF not only enables investigators from partner institutions to do specialized human stem cell work in our laboratories, it also creates cutting-edge techniques that accelerate all translational research in the field. NYSCF is like the â&#x20AC;&#x153;special operationsâ&#x20AC;? unit for other medical research institutions, helping them to move ahead faster, more effectively, and more cost-efficiently.

HAS THE CURRENT ADMINISTRATION PAVED THE WAY FOR FEDERAL FUNDING OF STEM CELL RESEARCH? Key political and administrative hurdles remain that prevent critical federal dollars from being spent on the most advanced stem cell science. Important congressional and judicial obstacles remain, and will likely continue indefinitely. Private funding is essential to ensure that the most innovative and promising human stem cell research continues notwithstanding the politics of the day.

A CRITICAL PHILANTHROPIC OPPORTUNITY NYSCF is a catalyst for essential, cutting-edge scientific research with a remarkable record of achievement. In its first ten years, NYSCF has enabled some of the most important discoveries in the field and has set the agenda for innovative stem cell research, bringing us closer to the necessary treatments and cures for a range of diseases. NYSCF is the best opportunity to assure that your investment in human stem cell research will be directed where it is most urgently needed: toward cures for the devastating diseases that touch all of our lives.

“I truly believe the work being undertaken at the New York Stem Cell Foundation can help all mankind. I believe in the organization and its mission and feel it’s incumbent upon people to come together and get behind them.” Stephen M. Ross Chairman & Founder, Related Companies

For more information on how you can support NYSCF, please contact Christine M. Lin, Vice President of External Relations, at cmlin@nyscf.org or 212.365.7434 NYSCF is a 501(c)(3) non-profit organization. Tax ID #20-2905531 25

OUR LEADERSHIP BOARD OF DIRECTORS NYSCFâ&#x20AC;&#x2122;s Board of Directors is made up of leaders in medicine, the arts, and business. Their talents and experience, combined with an unwavering commitment to advancing stem cell research, guide the Foundation in realizing its mission. Roy Geronemus, MD, Chairman Susan L. Solomon, CEO & Co-founder Margo Alexander

David A. Carmel

George Lazarus, MD

Peggy Bewkes

Alan M. Cohen

Richard J. Massey, PhD

Marilyn G. Breslow

Paul Goldberger

David K. Mitnick

Karen E. Burke, MD, PhD

Zach W. Hall, PhD

Stuart Smith, DPhil

LEADERSHIP COUNCIL NYSCF Leadership Council members are our valued ambassadors who are committed to funding and promoting the Foundationâ&#x20AC;&#x2122;s mission to accelerate stem cell research. Katherine Bristor and William Priest

Lawrence E. Golub and Karen Finerman

Carol Roaman Julian Robertson

Russell L. Carson

April Gornik

Eric A. Rose, MD

Chuck Close

Marlene Hess

Jack Rudin

Shirley Cook

Richard D. Kaplan

Stephanie and Fred Shuman

Fiona Druckenmiller

Dorothy Lichtenstein

Alice Shure

Jodie and John Eastman

Sandra Lloyd

Barbara Stovall Smith

Frank Gehry

Stephen Meringoff

Noelle and Dick Wolf

Nancy and Fred Poses

â&#x20AC;&#x153;The scientists supported by the New York Stem Cell Foundation envision a society transformed by their work. These are people that change the world. These are the people that know we can do better.â&#x20AC;? Michael R. Bloomberg Former Mayor of New York City