2022 NINDS Nonprofit Forum

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The National Institute of Neurological Disorders and Stroke (NINDS) invites members of interested nonprofit organizations to our annual Nonprofit Forum. The Progress through Partnership Forum provides an opportunity for nonprofit leaders and their membership to network with colleagues and engage in discussions with NINDS staff and leadership.

This year’s virtual meeting is structured around the overarching theme of “How to Work With…” from industry, to research partners, to NIH – including how to find innovative methods to fund research. We would like to thank the 2022 Forum Executive Committee for their work in identifying the goals, topics, and speakers for this year’s event!

The 2022 NINDS Nonprofit Forum was a virtual event that took place July 19-20, 2022 via Zoom. 

Agenda

1:1 and Small-group meetings with Program Staff will be scheduled via Zoom outside of main Agenda timeframe on Monday July 18^th, the lunch break and networking periods on Tuesday July 19^th and Wednesday July 20^th, after 3pm ET on Wednesday July 20^th, and on Thursday July 21^st. All meetings are scheduled based on the availability of our busy program staff. 

Day 1 Meeting Recording

Day 2 Meeting Recording

Presenters

Geraldine Acuña-Sunshine

Geraldine Acuña-Sunshine is President of the Sunshine Care Foundation for Neurological Care and Research and is Co-Founder of the Center for X-Linked Dystonia Parkinsonism at the Massachusetts General Hospital– two international non-profit organizations dedicated to funding neuroscience research and finding innovative ways of delivering free clinical care and humanitarian aid to indigent patients in rural areas of Asia. She was instrumental in establishing free neurological clinics and the first brain bank in the Philippines. Prior to entering the non-profit and social impact sectors, Geraldine practiced corporate law full time as Senior Counsel in alternative investments at Bracebridge Capital in Boston. Geraldine serves on the Harvard University Board of Overseers and on the President’s Council of the Massachusetts General Hospital. She sits on the Visiting Committees for the Harvard Chan School of Public Health and the Harvard Humanitarian Initiative. Geraldine lives in Boston, MA and Manila, Philippines.

Katherine Beaverson, M.S.

Katherine Beaverson, M.S. is Executive Director and Head of Patient Advocacy Lead for Pfizer’s Rare Disease Research Unit (RDRU), leading the strategic planning and implementation of patient engagement activities supporting mutual areas of priority.  She is both external facing, engaging with rare disease patient advocacy groups to integrate their expertise into early medicines research and development and she is also internal facing, helping to facilitate alignment among cross-functional colleagues committed to advancing Pfizer science and programs with rare disease patient communities.  Prior to joining Pfizer Inc., she held similar positions at Boehringer Ingelheim and Amicus Therapeutics.  She is professionally trained as a Genetic Counselor, having, spent 10 years at both New York Hospital- Weill Cornell Medical Center and Memorial Sloan-Kettering Cancer Center before entering industry.   She received her BA from Swarthmore College and her Master of Science in Human Genetics from Sarah Lawrence College.  Katherine is the current Chair of the Companies Constituent Committee of the International Rare Disease Research Consortium (IRDiRC) and a Member of the New York University Pediatric Gene Therapy Medical Ethics Working Group (PGTME).

James Beck, Ph.D.

As Chief Scientific Officer, James Beck, Ph.D., sets the strategic direction for the Parkinson’s Foundation research vision. Dr. Beck oversees a portfolio which has guided more than $400 million in research to explore what causes Parkinson’s disease (PD) and how to improve treatments, working towards a world without Parkinson’s disease. He has played an integral role in positioning the organization as a world’s leader in accelerating Parkinson’s research.

Dr. Beck launched PD GENEration: Mapping the Future of Parkinson’s Disease, a national study started by the Parkinson’s Foundation in 2019 that offers genetic testing for Parkinson’s-related genes and genetic counseling at no-cost for participants with a confirmed PD diagnosis. Additionally, he led the team that updated the estimated prevalence of PD in the U.S. and globally and collaborated with several organizations to develop the economic burden of Parkinson’s disease.

Geraldine Bliss, M.S.

Geraldine Bliss is the president and co-founder of CureSHANK, a nonprofit organization with the goal of accelerating drug development in Phelan-McDermid Syndrome  (PMS) and related SHANK disorders.  She served on the Phelan-McDermid Syndrome Foundation’s board of directors for two years and as its Research Support Committee chairperson for seven years. In that role she chaired the first scientific strategic planning initiative, successfully applied for grants to further PMS research support initiatives, organized four scientific symposia (2011, 2012, 2014, and 2018), a Banbury meeting (2013), multiple workshops and think-tanks on PMS, the Phelan-McPosium (2016, 2018), family conference research sessions (2010, 2012, and 2014), and established the PMS resource repository partnerships with CIRM, Autism BrainNet, and the NIMH, among other accomplishments. She has served as a lay reviewer for the FDA.  Geraldine has a bachelor’s degree from Trinity University and a master’s in human factors (health and human performance) from the University of Houston. Geraldine’s personal mission is to eventually have cures for PMS. Geraldine and her husband, Brad, of Houston, have two sons, Charles, 24, and Nathan, 21. Charles has PMS caused by a partial deletion of the SHANK3 gene.

Diane Bovenkamp, Ph.D.

Diane Bovenkamp, Ph.D., Vice President of Scientific Affairs, is the chief scientist at BrightFocus Foundation, overseeing global operations of the organization’s research programs. She serves as the scientific liaison in local, national, and international forums, and identifies and develops new research initiatives, partnerships, and funding policies consistent with the mission of BrightFocus.

Dr. Bovenkamp obtained her PhD in Biochemistry from Queen’s University in Kingston, Ontario, Canada, discovering and studying Eph receptors in angiogenesis and neural development in zebrafish and mice. She completed a Postdoctoral Fellowship in the Vascular Biology Program at Boston Children’s Hospital/Harvard Medical School, isolating and characterizing zebrafish neuropilins. Dr. Bovenkamp conducted further research at the Johns Hopkins University Bayview Proteomics Center in the Division of Cardiology at Johns Hopkins School of Medicine in Baltimore, Maryland, using proteomic techniques for biomarker detection in human serum.

Jennifer Brummet, Ph.D.

Jennifer is the Research Manager at the American Epilepsy Society where she supports the Society’s early career grant programs, professional development programs, and other research initiatives.  Jennifer received her bachelor’s degree in Psychology from Illinois Wesleyan University and a MA/PhD in Behavioral Neuroscience at Michigan State University. She has dedicated her career to supporting biomedical researchers, including prior roles as a Social Scientist at the National Science Foundation and in a science policy/outreach role at the Society for Neuroscience. Jennifer volunteers with the Health Research Alliance, the Chicago Association Forum, and the Chicago Council on Science and Technology.

Emily Caporello, Ph.D.

Dr. Emily Caporello is the Program Director of the Small Business Program in the Division of Translational Research (DTR) at the National Institute of Neurological Disorders and Stroke (NINDS). Dr. Caporello has worked at NINDS since 2019, leveraging her extensive experience in translational neuroscience program management as a Scientific Project Manager in the NINDS Small Business Program. Prior to joining NINDS, Dr. Caporello was the Vice President of Research and co-founder of MindX Corporation, a small business focused on brain computer interface technology, where she led research collaborations with academic labs and oversaw investigational approval of a novel neural interface device. Dr. Caporello previously worked as a Senior Neuroscientist supporting neurotechnology development the Biological Technologies Office at the Defense Advanced Research Projects Agency (DARPA), and co-founded CleverPet, a small business focused on automated behavioral training technology. Dr. Caporello received her Ph.D. in Neuroscience from the University of California San Diego, where her research focused on neural mechanisms of auditory processing and selective attention in the songbird model.

The Small Business Program is an integral part of the NINDS portfolio and provides $77M in funding annually to develop solutions in the neurological space. NIH small business funding has a strong national impact as the largest source of non-dilutive seed funding for health-focused small businesses in the U.S. Visit the NINDS Small Business Program for more information.

Penny Dacks, Ph.D.

Penny Dacks, Ph.D. is Senior Director of Scientific Initiatives at AFTD where she leads strategy for research initiatives to fund research, recruit and retain talent, advance collaboration, and engage the community with lived experience. On behalf of AFTD, she serves as President of the FTD Disorders Registry LLC, responsible for partnerships and sustainability of this organization affiliate. Previously, she worked at the American Epilepsy Society, overseeing mission-related programs in research, medical education, and clinical activities, and the Alzheimer’s Drug Discovery Foundation, leading development of CognitiveVitality.org and the Aging & Alzheimer’s Prevention Program to source and evaluate potential therapies. She trained in neuroscience with a PhD and postdoctoral fellowship at the University of Arizona and the Icahn School of Medicine, Mount Sinai.

Kenneth Fischbeck, M.D.

Dr. Fischbeck received A.B. and A.M. degrees from Harvard University and an M.D. degree from Johns Hopkins. After a medical internship at Case Western Reserve University and a neurology residency at the University of California in San Francisco, he did postdoctoral research on muscular dystrophy at the University of Pennsylvania. In 1982 he joined the faculty in the Neurology Department at the University of Pennsylvania Medical School. In 1998 he came to the NINDS as Chief of the Neurogenetics Branch. He received the Cotzias Award from the American Academy of Neurology and the Jacoby Award from the American Neurological Association, and he was elected to the National Academy of Medicine. His research group is identifying the causes and studying the mechanisms of hereditary neurological and neuromuscular diseases with the goal of developing effective treatment for these disorders.

Julie Louise Gerberding, M.D.

Dr. Julie Louise Gerberding is the Chief Executive Officer of the Foundation for the National Institutes of Health (FNIH).  The FNIH creates and manages research alliances with public and private institutions in support of the mission of the NIH to enhance health, lengthen life, and reduce illness and disability.  The FNIH works with its partners to accelerate biomedical research that addresses some of the most pressing health challenges in the United States and across the globe.

Formerly, Dr. Gerberding was Executive Vice President and Chief Patient Officer at Merck & Co., Inc., where she was responsible for patient engagement, strategic communications, global public policy, population health, and corporate responsibility. She joined Merck in 2010 as President of Merck Vaccines to help increase global access to important vaccines among those who need them most.

Dr. Gerberding was Director of the United States CDC from 2002-2009, where she led the agency through more than 40 emergency responses to public health crises, including SARS in 2003.  She currently co-chairs the CSIS Commission on Strengthening America’s Health Security and serves as a board director for Cerner Corporation, HilleVax, Afternext Health Tech, and SummerBio.

Dr. Gerberding has received more than 50 awards and honors, including the United States Department of Health and Human Services (DHHS) Distinguished Service Award for her leadership in responses to anthrax bioterrorism and the September 11, 2001 attacks.  In 2018, she was selected as the Healthcare Businesswomen Association’s Woman of the Year.

Dr. Gerberding received her undergraduate and M.D. degrees from Case Western Reserve University (CWRU).  She completed her internship and residency in Internal Medicine and fellowship in Clinical Pharmacology and Infectious Diseases at the University of California, San Francisco (UCSF) and is board certified in Internal Medicine and Infectious Diseases. She served as a tenured member of the infectious diseases faculty at UCSF and the hospital epidemiologist at San Francisco General Hospital and is currently an Adjunct Associate Professor of Medicine at UCSF and an Adjunct Professor of Medicine at CWRU.  Dr. Gerberding received a Masters of Public Health at the University of California, Berkeley.  She is a member of the National Academy of Medicine, a member of the Council on Foreign Relations, and a fellow of the Infectious Diseases Society of America and the American College of Physicians.

Paul Gross

Paul Gross is a driving force in accelerating clinical and translational research in neuroscience for cerebral palsy and hydrocephalus.  He is the founder and chair of the Cerebral Palsy Research Network – a twenty-eight center effort in North American to conduct high quality clinical research for CP.  He is the co-founder of the Hydrocephalus Clinical Research Network and the Hydrocephalus Association’s Network for Discovery Science.  He spent four years as an advisor to the National Institute of Neurological Disorders and Stroke. Prior to ramping up his focus on advancing medical research, he was CEO of a web startup, a Senior Vice President with the Microsoft Corporation and with Borland International.  In his personal life, he is married and the father of two children, one of whom has hydrocephalus and cerebral palsy.  He is also an avid mountain biker.

Tish Hevel

Tish Hevel is the Founder/CEO of The Brain Donor Project, a unique non-profit that exclusively supports the brain banks of the National Institutes of Health (NIH), the NeuroBioBank. The Brain Donor Project was established in late 2016 to raise awareness of the critical need for donated brain tissue to advance the science of brain disease and to simplify the process of becoming a brain donor. To date, more than 15,000 people have started the process of becoming a brain donor when the time comes. They represent more than 160 categories of brain disease…and about half have healthy (or control) brains. The Brain Donor Project was inspired by the death of Tish’s dad, Gene, who died from Lewy Bodies Dementia and was a brain donor himself.

Tish is an Emmy award-winning broadcast journalist who worked internationally as a PR professional and served as Chief Communication Officer for a 44-county region of the American Red Cross.

Janet Hieshetter

Janet Hieshetter serves as the Executive Director of the Dystonia Medical Research Foundation (DMRF). She joined the DMRF in November 20104. Ms. Hieshetter serves on the National Institute of Neurological Diseases and Stroke Advisory Council and also serves as the Chairman of the Board for the American Brain Coalition. She also served on the Illinois Women’s Health Task Force, Massachusetts Bone Health Task Force, Tennessee’s Task Force on Bone Health, The Texas Task Force on Women’s Health and the Pennsylvania Task Force on Women’s Health. Ms. Hieshetter is a graduate of Kalamazoo College and lives in Chicago, IL.

Agustin Ibanez, Ph.D.

Agustin Ibanez is a neuroscientist interested in global approaches to dementia and social, cognitive, and affective neuroscience. He is the Director of Latin American Brain Health Institute (BrainLat) at Universidad Adolfo Ibáñez (UAI). Also, He holds international positions from the USA/Ireland [Senior Atlantic Fellow, Global Brain Health Institute-GBHI-University of California San Francisco and Trinity College Dublin)] and Argentina [Cognitive Neuroscience Center]. Agustin holds a track record with +120 publications in the last five years, including top-ten journals (e.g., Lancet Neurology, World Psychiatry, Nature Reviews Neurology, Nature Human Behavior, JAMA Neurology, Alzheimer’s & Dementia, Brain). He has received funding from the Inter-American Development Bank (IDB), ANID (Chile), COLCIENCIAS (Colombia), DAAD (Germany), MRC (United Kingdom), CONICET (Argentina) and Alzheimer’s Association, Tau Consortium, GBHI, Takeda, and NIH/NIA (USA). He is the founder of the critical regional initiatives, such as the Multi-partner consortium to expand dementia research in Latin America (ReDLat) and the Latin American and Caribbean Consortium on Dementia (LAC-CD). His work has been highlighted in the BBC, Nature, Nature News, Discovery Channel, Popular Science, Daily Mail, Newsweek, Le Monde, and Oxford University Press, among others.

David D. Limbrick, M.D., Ph.D.

David Limbrick, MD, PhD, is neurosurgeon whose areas of interest include pediatric neurosurgery, epilepsy surgery, neuro-endoscopy, hydrocephalus, pediatric brain tumors, deep brain stimulation, radiosurgery and pediatric spine.

Laura Mitic

Laura Mitic, PhD, is the Chief Scientific Officer at the Bluefield Project to Cure Frontotemporal Dementia. Dr. Mitic holds a BA degree from Northwestern University and a doctorate degree in cell biology from Yale University. She completed postdoctoral studies at the University of California, San Francisco.

Eric Morrow, Ph.D.

Eric M. Morrow received his Ph.D. in genetics and neurodevelopment at Harvard University. He received his MD degree from the Health Science Training (HST) Program at Massachusetts Institute of Technology and Harvard Medical School. He conducted further clinical and scientific training in neurology and psychiatry at Harvard Medical School. Dr. Morrow's research focuses on normal molecular mechanisms of brain development, and genetic perturbations that underlie disorders of human cognitive development, including autism and intellectual disability.

The laboratory investigates mechanisms in neuronal organelles, such as endosomes, lysosomes and mitochondria, including also neurometabolism. Dr. Morrow and his colleagues have focused on novel human neurogenetic syndromes. Increasingly study of these syndromes, such as Christianson Syndrome and GPT2 Deficiency, have also led his laboratory to investigate mechanisms in selective neuronal vulnerability and neurodegeneration. His research has been funded by NIMH, NINDS and NIA.

Indu Navar, M.S.

Over the past 20 years, Indu has experience in being Co-founder/CEO of software companies and taking them through successful exits, strategic partnerships and M&A. 

She is an Investor and Strategic Advisor in many startups. She is currently CEO and Founder of EverythingALS. She co-founded geek.ly and Xendota. Prior to that, Indu was Managing Director at Woodside Capital Partners where she advised software companies on strategic financing transactions and M&A. She was Founder and CEO of Serus Corporation, provider of SAAS platform for companies with outsourced manufacturing. The company was acquired by E2OPEN (Nasdaq:EOPN). Prior to Serus, Indu has been on the ground floor of several successful technology companies including Healtheon (WebMD), Silicon Graphics, Black and White Software (Segue). She started her career at NASA in Moffett Field, California.

Indu has B.S Electrical Engineering from Bangalore University, India and M.S. in Computer Science from California State University, Chico.

Kevin O'Connor, Ph.D.

Dr. Kevin C. O’Connor is an Associate Professor of Neurology and Immunobiology at Yale School of Medicine. He earned a Bachelor of Science degree in Chemistry from the University of Massachusetts at Amherst and his Ph.D. in Biochemistry at Tufts Medical School. He took his post-doctoral training in Immunology at Harvard Medical School where he also spent several years on the faculty as an Assistant Professor. His investigative interests are in human translational immunology and neurology. He and his group are specifically interested in defining the mechanisms by which B cells, and the antibodies they produce, affect tissue damage in autoimmunity. To this end they are engaged in understanding how particular B cell subsets initiate and sustain autoimmunity. He and his team were among the first to characterize tertiary lymphoid tissue and the adaptive immune response in germ cell tumors and meningiomas. They also described the molecular characteristics of B cells and plasma cells that populate the muscle tissue of patients with myositis. They refined the role of Epstein-Barr virus in the multiple sclerosis (MS) brain and have further defined the role of humoral immunity in children with MS. Recently, he and his team identified a network of B cells and autoantibodies that populate the MS central nervous system. His current research focus includes further defining the immunopathology of myasthenia gravis (MG). He and his team demonstrated that B cell depletion therapy has sustained efficacy in MG. They were the first to show that MG-derived AChR antigen specific T cells belong to the pro-inflammatory Th17 subset. They also determined that MG subjects harbor defects in B cell tolerance checkpoints that correlate with abnormalities in the naïve B cells repertoire. They most recently identified the autoantibody-producing cells in MuSK MG. Their current focus is on further defining the mechanisms of autoantibody production in MG with the aim of improving therapeutic approaches.

Daniel Okobi

Daniel Ebele Okobi, Jr., M.D., Ph.D. is a highly experienced, board-certified neurologist. After graduating cum laude from Harvard University for his undergraduate degree in Chemical and Physical Biology, Dr. Okobi earned his medical degree and his Ph.D. from New York University. He completed an internship in internal medicine at Jackson Memorial Hospital and the University of Miami, then went on to complete his residency in neurology from the University of California, Los Angeles. Most recently, Dr. Okobi completed a behavioral neurology and neuropsychiatry fellowship at UCLA and the West Los Angeles Veterans Affairs Medical Center.

During a distinguished career at Harvard, NYU, and UCLA Dr. Okobi contributed to numerous publications in respected scientific and medical journals and has presented at the annual meetings of the Society for Neuroscience and the American Academy of Neurology, among others.

Sabrina Paganoni, M.D, Ph.D.

Sabrina Paganoni, M.D., Ph.D., is the Co-Director of the Neurological Research Institute at the Massachusetts General Hospital, Assistant Professor at Harvard Medical School and physician investigator at the Healey & AMG Center for ALS at Mass General. Dr Paganoni’s research focuses on clinical trials and therapy development for ALS. She has served as Principal Investigator of several ALS clinical trials including the CENTAUR trial (a trial of AMX0035 in ALS). She is also the co-Principal Investigator of the HEALEY ALS Platform Trial, the first platform trial for ALS in the world. Her research has been funded by the NIH, non-profits, and industry; she published more than 100 peer-reviewed manuscripts and received several awards for her work including the 2021 Top 10 Clinical Research Achievement Award for the CENTAUR trial. She is also passionate about clinical care innovation and access to research for people with ALS. She co-chairs the Upper Motor Neuron Task Force, the Technology Committee and the Recruitment/Retention/Experience Committee at NEALS.

Ken Schaner

In his more than 40 years of private practice, Ken Schaner has represented many for-profit and nonprofit entities in the corporate and tax aspects of a wide variety of agreements, transactions, financings, licenses, mergers and acquisitions.

Schaner began his career at the Internal Revenue Service’s (IRS) legislative and regulations division. During his time with the IRS, Schaner worked on the 1969 Tax Reform Act and was one of the principal drafters of the new private foundation provisions.

In 1982, Schaner co-founded Swidler Berlin, LLP. While a partner in that firm, he also served as managing member and chair of the corporate group. After Swidler Berlin’s merger with Bingham McCutchen, LLP in 2006, Schaner remained a partner until 2008, when he formed a new firm to focus on representing tax-exempt organizations.

Since 1983, Schaner has served as general counsel to the Cystic Fibrosis Foundation (CFF). In that capacity, Schaner represented CFF in its first venture philanthropy transaction with Aurora Biosciences Corporation (now Vertex). Since then, Schaner has represented numerous clients in venture philanthropy transactions and related legal matters. Schaner also serves as general and outside counsel to many nonprofits. He advises on the full range of issues faced by Section 501(c)(3), (c)(4) and (c)(6) organizations, including board governance, business and tax-exempt compliance issues.

He holds a bachelor’s degree and Juris Doctor from the University of Illinois.

Nina F. Schor, M.D., Ph.D.

Nina F. Schor, M.D., Ph.D. former Deputy Director of the NINDS and a Senior Faculty Associate at the University of Rochester.  Dr. Schor graduated cum laude from Yale University with a B.S. degree in Molecular Biophysics and Biochemistry and as a Scholar of the House in Chemistry Research in 1975. She received her PhD in Medical Biochemistry from Rockefeller University and the laboratory of Dr. Anthony Cerami in 1980 and her MD from Cornell University Medical College in 1981.  Dr. Schor pursued residency training in Pediatrics at Boston Children’s Hospital (1981-1983) under Dr. Mary Ellen Avery and Child Neurology at the Longwood Area-Harvard Neurology Program (1983-1986) under Dr. Charles Barlow. During residency, she also pursued a postdoctoral fellowship in the laboratory of Dr. Manfred Karnofsky at Harvard. During this time, she began her studies of neuroblastoma, aimed at understanding the neurobiology of this tumor and exploiting this understanding to design and test in preclinical models novel strategies for the therapy of chemoresistant neuroblastoma.  For the next 20 years, Dr. Schor rose through the academic and administrative ranks at the University of Pittsburgh, ultimately becoming the Carol Ann Craumer Professor of Pediatric Research, Chief of the Division of Child Neurology in the Department of Pediatrics, and Associate Dean for Medical Student Research at the medical school. In 2006, Dr. Schor became the William H. Eilinger Chair of the Department of Pediatrics, and Pediatrician-in-Chief of the Golisano Children’s Hospital at the University of Rochester, posts she held until January 2018, when she became Deputy Director of the NINDS.

Todd Schwedt, M.D.

Todd Schwedt, M.D., MSCI is a Professor of Neurology and Chair of Neurology Research at the Mayo Clinic Arizona.  In addition to his clinical duties in the Mayo Headache Clinic, he leads an active headache research portfolio.  He is a principal investigator of the Neuroimaging of Headache Laboratory at Mayo Clinic, of the American Registry for Migraine Research (ARMR), the Medication Overuse Treatment Strategy Trial (MOTS), and he is the Medical Director of the Mayo Clinic Arizona Clinical Studies Unit. He was a recipient of the 2015 Harold G. Wolff Award from the American Headache Society, an award presented annually to a researcher who has most significantly contributed to the understanding of headache. Dr. Schwedt serves on the Executive Committee for the Board of Directors for the American Headache Society, is Co-Chair for the 2021 International Headache Congress, is a member of the American Headache Society scientific meeting planning committee, is the chairperson of the post-traumatic headache working group for the International Classification of Headache Disorders, and is an associate editor for Cephalalgia, Headache, and Headache Currents. Dr. Schwedt is a frequent lecturer and has authored over 115 peer-reviewed publications.…

Miguel Sena-Esteves, Ph.D.

Miguel Sena-Esteves, PhD, is associate professor of neurology and a member of the Horae Gene Therapy Center. He received his PhD in biomedical sciences from the University of Porto, Portugal, where he started his work in gene therapy.

His laboratory at UMass Chan focuses on the development of adeno-associated virus (AAV) based gene therapies for lysosomal storage diseases, Huntington’s disease, ALS, FSHD and brain tumors. His laboratory develops novel AAV capsids to improve targeting, distribution and potent transduction of target tissues in these diseases, with a particular focus on the central nervous system.

Dr. Sena-Esteves is a member of the Tay-Sachs Gene Therapy Consortium, which focuses on developing AAV gene therapies. In 2011 he received the Outstanding New Investigator Award from the American Society of Gene & Cell Therapy (ASGCT) for his contributions to the field of gene and cell therapy. He has authored 88+ peer-reviewed publications and is inventor on several patent applications.

Pilar Trelles, Ph.D.

Pilar Trelles, MD, is a former Medical Expert Board member with Verywell. She is a board-certified child and adolescent psychiatrist. Her area of clinical expertise is in autism spectrum disorder (ASD) and related neurodevelopmental disorders (NDDs).

Heather Watkins

Heather Watkins is a disability advocate, author, blogger, mother, speaker, and consultant who was born with muscular dystrophy. A disabled Black woman living in Boston, Massachusetts, she graduated from Emerson College with a B.S. in Mass Communications.

Yael Weiss, M.D., Ph.D.

Yael Weiss is currently CEO of Mahzi Therapeutics, a company focused on the development of therapies for ultra-rare genetic neurodevelopmental disorders. Mahzi works closely with patient foundations to support their journey towards drug development and bring programs into Mahzi once pre-clinical proof of concept is established. Yael completed her M.D. at Hadassah Medical School at the Hebrew University in Jerusalem and her Ph.D. at the Weizmann Institute of Science in Rehovot, Israel. She has over 20 years of industry experience in medical-clinical and business development roles at Genzyme, Merck and Ultragenyx. Yael is a member of the NIH driven Bespoke Gene Therapy (BCTG) consortium, ASGCT translational committee, N1 collaborative and is a 2022 Termeer Fellow Board member and advisor to ADNP and FOXG1 foundations.

Jill Wood

Jill Wood graduated from Oregon State University with a BS in retail management and apparel design. Jill is a rare disease patient advocate as her son Jonah was diagnosed with Sanfilippo syndrome. She co-founded Jonah’s Just Begun (JJB) with her husband in 2010, a non-profit to raise awareness and support those with Sanfilippo. She built a scientific advisory board, created a comprehensive social media strategy and raised funds to support the research into Sanfilippo syndrome. In 2012 she co-founded Phoenix Nest with the aim to develop treatments for Sanfilippo Syndrome. Jill frequently lobbies on Capitol Hill with the RDLA in support of rare disease initiatives. Jill is also a speaker at major conferences where she tells her story from mom to CEO of a rare disease organization.