Board of Directors
The N1C Board of Directors plays a pivotal role in guiding the organization’s mission to make individualized medicine safe and rapidly accessible to patients worldwide. Drawing from a diverse group of internationally acclaimed experts, the board ensures that our efforts in advancing individualized treatments remain focused on knowledge sharing, safety, and collaboration.
Nicole Boice
Co-Founder, SEE RARE™
Founder, Global Genes & RARE-X
Nicole Boice is a committed champion of families affected by rare disease. She founded Global Genes in 2008 and grew it into one of the most notable rare disease advocacy organizations focused on educating and empowering patients/advocates throughout their journey. Impact includes a membership of over 750 patient organizations, collaborations with over 100 biopharma and academic research partners, numerous support programs and educational events. RARE-X grew out of the need to provide more tools and resources for families needing to become better equipped data stewards and participants in research. A board member of several patient organizations and advisory roles within Biopharma, Nicole remains an important voice for rare disease patients globally.
Holm Graessner
Managing Director, Centre for Rare Diseases, University Hospital Tübingen, Germany
Holm Graessner has graduated in Biomedical Engineering, Cybernetics, Electrical Engineering, German Language and Literature, Philosophy as well as Business Administration. He received his PhD “Summa cum laude” in 2004 and, then, he obtained his MBA degree in 2008.
He has been Managing Director of the Rare Disease Centre, since 2010, at the University and University Hospital Tübingen, Germany. www.zse-tuebingen.de He is Coordinator (Lead PI) of the European Reference Network for Rare Neurological Diseases (ERN-RND, www.ern-rnd.eu). Together with Olaf Riess, he was leading the H2020 Solve-RD project on “Solving the unsolved rare diseases” (www.solve-rd.eu) and is currently co-leading the Clinical Research Network – that is the in-house research and development – of European Rare Disease Research Alliance (www.erdera.org) that has started in Sept 2024. He is a founding member of the European 1 Mutation 1 Medicine initiative.
Since 2020, as a fellow of the European Academy of Neurology (EAN) he has been a member of the management teams of the Neurogenetics Panel and the Rare Neurological Disease Coordinating Panel of the EAN.
Brian Tseng
As a board-certified M.D., Ph.D. pediatric neurologist/neuromuscular physician–scientist, is currently the CEO for The POLG Foundation, young non-profit rare disease research advocacy organization.
Mission – The POLG Foundation
Brian has had a highly productive career in both academia and biopharma industry. He has led large cross-functional teams (Novartis, AveXis, Vertex) to develop and bring forward many new medicines approved for use in infants and children with rare disorders, including genetic disorders. He has had deep experience in translational and clinical development of novel molecules (small, biologic, cell-based, AAV genetic replacement, and CRISPR-mediated gene editing) for children. His past clinical/academic practice has been at Massachusetts General Hospital/Harvard Medical School and earlier at Children’s Hospital Colorado. His undergraduate degree is from University of California Berkeley and he has a Ph.D. in molecular biology from MD Anderson Cancer Center/University of Texas at Houston McGovern Medical School. His pediatric residency was conducted at Children’s Hospital in Colorado and child neurology training at UCSF. While based in Colorado, he is currently a Parkmedic NOLS Wilderness Medicine first responder for Search and Rescue in the Rocky Mountain National Park.
Julia Vitarello
Mila’s Mom
Mila’s Miracle Foundation
julia@stopbatten.org
In December 2016, Julia founded Mila’s Miracle Foundation (MMF) upon learning that her seemingly healthy six-year-old daughter Mila had Batten disease, a devastating fatal genetic condition. In a race against time, Julia raised the funds to support the science in grass-roots efforts, from over 7000 supporters in 60 countries, while at the same time being a mother and caregiver. In an unprecedented collaboration led by Julia and Dr. Timothy Yu from Boston Children’s Hospital, Mila became the first person in the world to receive a drug tailored to just one person – named milasen. Driven by a sense of hope and obligation, Julia is on a mission to open up the new field of individualized medicine and make it accessible to children across all rare diseases who otherwise would have no hope.
Through MMF, Julia also initiated the work toward a novel gene replacement therapy targeting the MFSD8/Batten CLN7 gene which began in 2021, launched the Batten and Neurodegenerative Disease Clinic at Children’s Hospital Colorado, and funds basic science projects in the US and Europe. She co-hosts the weekly show, Gene-Fixers on Clubhouse, launched the photo journal This is Today and speaks at scientific meetings and conferences across the country.
Winston Yan, M.D., Ph.D.
Founding President, N=1 Collaborative
Director, Center for Genetic Surgery (CGS), Broad Institute
Winston Yan is a physician-scientist, genome engineer, and organization builder motivated to help the many patients with rare genetic diseases for whom no treatments exist. He is the Founding President of the N=1 Collaborative, working as a volunteer to build the N1C since its inception in 2021.
He co-founded and directs the Center for Genetic Surgery (CGS) at the Broad Institute, which was established by the Broad in collaboration with partners across hospitals, industry, research institutes, and patient advocacy groups to advance programmable genetic therapies for patient populations that are too rare or too challenging for traditional commercial development. Previously, Winston co-founded Arbor Biotechnologies, with firsthand experience taking Arbor’s lead therapeutic program from discovery to dosing patients. Over nearly nine years at Arbor, he helped lead Arbor’s early strategy and fundraising, build close-knit teams and organizational culture, led scientific platform development and translational strategy, authored clinical and regulatory documents towards IND and CTA acceptance, and served as clinical lead of a global first-in-human CRISPR gene editing trial.
Winston completed his undergraduate degree in physics magna cum laude at Harvard College, his M.D. from Harvard Medical School through the Health Sciences & Technology (HST) program, and his Ph.D. from Harvard University, working at the Broad Institute on early CRISPR genome editing technology development. Winston aspires to bring together the scientific, strategic, and operational parts of his background to help build the N1C with the care and urgency that rare disease patients need.
Tim, Yu, M.D., Ph.D.
Boston Children’s Hospital
Harvard Medical School
Dr. Yu completed his undergraduate degree in biochemistry and molecular biology at Harvard College, his M.D. and Ph.D. degree (in Neuroscience) from the University of California at San Francisco, clinical neurology training at Massachusetts General Hospital and Brigham and Women’s Hospital, and a fellowship in neurodevelopmental genetics at MGH and Boston Children’s Hospital. He joined the faculty in the Division of Genetics and Genomics as Instructor in 2010 and Associate Professor in 2013.