Aki Ko has served as Chief Executive Officer and Chair of the Board of Directors since May 2017, when he co-founded the company. Previously, Mr. Ko served as Chief Operating Officer for Elixirgen, LLC, the parent company of Elixirgen Therapeutics, and President of Elixirgen Scientific, LLC, a provider of stem cell reagent kits and related services. In his previous roles, Mr. Ko was responsible for operations and business development at Elixirgen, LLC and Elixirgen Scientific. Mr. Ko earned a Bachelor of Science degree from the University of Virginia, where he cultivated his interest in biology.

Jenifer Ngo Waldrop joined the Black Women’s Health Imperative (BWHI) as Executive Director of the Rare Disease Diversity Coalition (RDDC) in October 2022. Before joining the leadership team of RDDC, she developed models and programs for diversity, equity, and inclusion initiatives to support multiple business units with Fortune 500 companies and organizations in Asia, Canada, and the US.

Jenifer has led initiatives at non-profit organizations where she recruited local community members to participate in numerous health-related fundraising events and developed programs to target disparities and underrepresented groups. In addition to facilitating development and fundraising, Jenifer developed a reputation for successful coalition building.

Jenifer received her undergraduate degree from Colby College and her Master of Human Resources Development from Villanova. As the Professional Development Director of the National Association of Asian-American Professionals (NAAAP) of Colorado, Jenifer continues her outreach in society. Through this organization, she mentors formally and informally.

After being diagnosed with a meningioma and p1RCC (a rare kidney cancer) in 2014, Bill co-founded rarekidneycancer.org with Dr. James Hsieh in 2016.  In 2018 and 2020, with Pete Kane, he brought together 17 teams of more than 100 researchers who used his medical data to determine “genes of interest” related to his disease.  He applied ensemble reasoning to evaluate the results of these hackathons (which Bill calls “gamified tumor boards” ) to discover better ways to do cancer research.  Bill is currently automating these “gamified tumor boards” using Large Language Models (LLMs).  Bill is also involved in several patient organizations and is a CDMRP reviewer.

Prior to rarekidneycancer.org, Bill worked with Sabrina Paseman to use non-invasive Blood fluorescence to detect iron deficiency anemia (Patent 8,306,594) and with Katherine Paseman to expand Pulse oximetry technology to do non-invasive measurement of Hemoglobin and Hematocrit.  

Bill’s 50 year career in AI started at age 18, when he worked with Howie Johnson at Rice University using n-grams (like chatGPT) to do classical music composition.  He continued his music work at MIT in Steve Ward’s lab exploring generative grammars and constraints.  He then applied his Symbolic AI knowledge to Electrical Design Automation as the 16th employee at Daisy Systems. Bill next founded Atherton Technology (which failed) and then Calico Commerce (which IPOed), where he repurposed his music composition system to do sales configuration over the internet (Patent 5,745,765).  After Bill took Calico public, he retired and, as discussed above, has dedicated his time to rarekidneycancer.org, patient advocacy and working with his daughters on non-invasive blood analysis.

Kartik Ramkumar is the Vice President of Diversity, Equity, and Inclusion at Leapcure, where he champions efforts to increase participation among underrepresented and marginalized communities in clinical research and develops programs to support DEI internally at Leapcure as well. He has worked on Diversity, Equity and Inclusion initiatives since university, supporting company teams in leading training and helping teams execute DEI programs. He is thrilled to discuss how DEI can be championed within clinical trials and how to achieve greater diversity and inclusion within rare disease clinical trials. Kartik currently lives in Washington D.C. where he enjoys being outside, exploring coffee shops, and volunteering outside of work.

Amanda Moore has served as the CEO of the Angelman Syndrome Foundation since 2019. With a background in nonprofit leadership and development, Amanda brings a wealth of experience to her role, having previously worked extensively with the YMCA.

In 2015, Amanda and her husband Adam were blessed with the joyous addition of twin boys, Jackson and Baden. Their world changed in 2016 when Jackson was diagnosed with Angelman Syndrome, sparking Amanda's unwavering commitment to advocacy and support for individuals like her son.

Driven by her personal experiences and a deep desire to make a difference, Amanda immersed herself in patient advocacy. She served on the Angelman Syndrome Board for one year before assuming her current position as CEO, where she continues to lead the foundation with passion, dedication, and a relentless pursuit of advancing the lives of those affected by Angelman Syndrome. Her advocacy efforts extend beyond her professional role, as she actively engages in initiatives aimed at raising awareness, promoting research, and advocating for improved care and support for individuals with Angelman Syndrome and their families.

Ms. Lane has over 25 years of experience in the Pharma/Biotech industry in clinical operations and project management. She has served as VP of Clinical Operations at Adverum Biotechnologies and has held leadership positions at Genentech, Avalanche, Geron, Pfizer and Sanofi. Her extensive experience in Clinical Operations of phase II-IV trials includes preparation of IB, ICF, CSR, annual safety update reports, eDC as well as IND,BLA, CTA filings. She has vast experience in CRO Management, Training of Clinical Project Leaders and Clinical Research Site Managers (CRSMs).

Michelle Carpenter, J.D., RAC has extensive work experience in regulatory affairs and project management. Michelle is currently serving as the EVP of Regulatory Affairs and Project Management at Star Therapeutics since January 2020. Prior to this, they worked at RPSpharma as the President since September 2013. Michelle has also worked as an instructor at UCSC Extension Silicon Valley from January 2014 to August 2023. Michelle has held leadership positions at True North, Afferent Pharmaceuticals, Acucela Inc., Dow Pharmaceuticals, Oculus Innovative Sciences, Dey Labs, and Santen, Inc. Michelle has successfully led various regulatory and clinical strategies, obtained regulatory approvals, and negotiated development programs and clinical studies throughout their career.

Michelle Carpenter, J.D., RAC has a comprehensive education history. Michelle earned their J.D. degree in health law from Golden Gate University, School of Law, attending from 1987 to 1991. Prior to that, they attended UC Santa Barbara, where they pursued a B.A. degree in sociology with a focus on biology, chemistry, and sociology. However, specific start and end years for their undergraduate studies are unavailable at this time.

Dr. Maida is the Chief Clinical Officer for Oncotelic, Inc. and its joint venture, SapuBioscience, LLC, focused on immunotherapy of cancer and infectious disease. He has over 30 years of experience in clinical research in oncology and has worked with many Center of Excellence and Key Opinion Leaders. He is responsible for overseeing the operations of the company and has experience in finance, operations, research, and commercial clinical and scientific development, regulatory, and manufacturing for various therapeutic modalities. Previously, he served as Vice President of Clinical Research and General Manager, Oncology, World-wide for PharmaNet, Inc., and President and CEO of Replicon NeuroTherapeutics. He raised or assisted in financings in excess of $200 million for start-up and emerging biotechnology companies and negotiated licensing agreements with various centers of excellence and premier pharmaceutical firms. Over the past 18 years, he has served in various executive roles, including Chairman, CEO, COO, CSO, CFO, and business development. He also served as Senior Financial Controller of Lockheed Missiles, a $1.7 billion division.

Nicole Miller, PhD is the Vice President of Molecular Diagnostics at Ultragenyx, a patient-focused biotech company led by Emil Kakkis that develops therapies for rare genetic disease. She focuses on advancing biochemical and genetic testing in support of reducing the diagnostic odyssey for rare disease patients. She has partnered to develop and launch ten gene locus specific databases and published several variant reviews for rare genetic disorders.  Prior to Ultragenyx, Nicole developed and led a similar function at BioMarin for about 9 years, focusing on rare disease patient diagnosis. Since 2010, Nicole has worked closely with the newborn screening and sequencing community in support of improving newborn screening for rare disease.  Prior to working in the rare disease field, she led a molecular pharmacology research team at Exelixis and led a biomarker program there for about a decade supporting research through clinical development. Nicole has a bachelor’s degree in genetics from U.C. Davis, a PhD in oncology from U.W. Madison and her postdoc was at UCSF. She sits on the board of the Professional Science Master’s program at the University of San Francisco since the program’s inception.

Stephen has been in biopharma for over 25 years and has worked in neurology with Elan Pharmaceuticals, gene therapy for rare diseases with Audentes Therapeutics and Encoded Therapeutics, virology with Gilead Sciences, and oncology with Genentech, Medivation, Clovis Oncology, and 23andme, and, currently, Corcept Therapeutics.  I started my career in clinical operations where I led company-sponsored studies and oversaw investigator-initiated studies.  I made the move to project/program management in 2014 to work on all phases of drug development from discovery through post-marketing activities and have assumed business development and alliance management responsibilities as needed for my programs.  I earned an MBA through the Gilead Sciences MBA cohort program, an MA in East Asian Art from the Sotheby’s Institute of Art in London, and an MS in Project Management.  I am a team leader dedicated to bringing new drugs to patients. I believe in life long learning, appreciate the here and now, strive to treat everyone with compassion and dignity, and lead with integrity and drive to meet corporate goals.

Katrina "Kat" Paz is a Director of Clinical Programming from Denali Therapeutics.  Serving as a programmer for over two decades, Katrina has worked at Genentech, Gilead Sciences and Nektar Therapeutics.  She has worked as a Data Manager and Statistical Programmer but found her niche in Clinical Programming, creating outputs for data cleaning and monitoring.  Kat's joys are leading technical teams with a focus in mentoring and finding efficiencies via tools and processes.

Nasser Al-Ali is currently a senior Clinical Programmer at Denali Therapeutics and a PhD student in Statistics and Data Science at the University of Nevada, Reno. He is deeply interested in programming, particularly in automating processes to enhance efficiency and effectiveness. His focus extends to the utilization of machine learning in data cleaning, aiming to elevate data quality through innovative, real-time cleaning strategies.

Head of Clinical Operations for Epic Bio, where he oversees all Clinical Operations activities in support of Epic's clinical trials. He has a background in patient care setting, as well as research-focused clinics on the ground level of clinical trials before moving into research management. For the last 20+ years he has led cross-functional teams carrying out global multicenter trials - in all phases of drug development - and multiple therapeutics areas including Neuro, Oncology and Immunology. He has experience with both large and small molecules therapeutics as well as gene therapies and now epigenetic therapies with Epic Bio.

For the last decade his focus has been on building capabilities for startup Biotechs to tackle rare and ultra-rare diseases and has launched first-in-human therapeutics for companies like Genentech, Audentes, Cytokinetics, Jasper Therapeutics and Kezar Life Sciences.

Ken has an International Executive MBA in Business and Finance from the International School of Management (ISM) in Paris, France.

Hayley Mason MPA, is a dedicated professional with a fervent commitment to promoting effective governance and public policy. Holding a Master of Public Administration (MPA) degree from American University in Washington, DC, and a Bachelor's degree in Global Affairs from George Mason University, Hayley brings a comprehensive understanding of governmental structures, policy analysis, and program evaluation to her advocacy work for individuals with rare diseases. Throughout her career, Hayley has focused on enhancing public service delivery and driving positive societal change.

Hayley's journey began as an intern for the United States Senate and then at the Ability Center of Virginia, a nonprofit organization dedicated to supporting children and adults with disabilities lead productive lives in Virginia Beach, Virginia.

Drawing from these diverse experiences, Hayley currently serves as a Policy Analyst with the National Organization for Rare Disorders. In this role, she collaborates with advocates, Congress, and government officials to champion increased treatments, access, and inclusion for individuals living with rare diseases. Driven by a profound desire to effect meaningful societal change, Hayley is committed to inspiring others to pursue excellence in public service and harnessing the power of effective governance for the betterment of all

Vij Senthilnathan is currently working at PTC Therapeutics, a US pharmaceutical company as Associate Director. She is a PTC leader in clinical operations working on the small molecule therapy PTC743 (vatiquinone), now in a Phase 3 global clinical trial for patients with mitochondrial disease and associated refractory epilepsy. Her involvement in rare diseases has been since 2014 starting with her role in clinical operations at BioElectron Technology Corporation (previously known as Edison Pharmaceuticals) where her responsibilities included running global clinical trials for Leigh Syndrome, Pearson Syndrome, Friedreich Ataxia, Amyotrophic lateral sclerosis (ALS), Parkinson Disease and Rett Syndrome. As a global lead and point of contact for clinical operations and strategy, she ensures effective execution of the clinical studies, strategies and mitigation plan for key regulatory submissions keeping patients first. She has been a strong advocate for patient’s voice and believes that patient advocates are an integral part of drug development process from start to finish. She works closely with different patient advocacy groups to understand their perspective for a clinical trial and ensures that the patients get asked the question they wish others would ask.

She holds a master’s degree in Molecular Medicine from University of Sheffield, UK and Bachelor’s of Technology in Biotechnology from India. She currently lives in San Francisco Bay Area, California. Her work: https://www.ptcbio.com/2021/02/08/working-to-treat-mitochondrial-epilepsy-one-seizure-at-a-time/

Revati Tatake, PhD, is the Global Head of Pharma Research, Analysis and Competitive Intelligence at GlobalData Healthcare.  Revati has over 25 years of multidisciplinary experience in academic research and the healthcare industry, both on the client and agency sides of the business. Previously she worked as a VP of Databases and Analytics at GlobalData, where she made significant contributions to Pharma Intelligence Center. Before joining GlobalData, Revati worked at Citeline/Informa, where she was involved in competitive intelligence and analytics of clinical trials and drugs in the areas of Autoimmune/Inflammation, Central Nervous System, and Ophthalmology.  Earlier, Revati worked at Boehringer Ingelheim Pharmaceuticals for over 12 years as Senior Scientist to Senior Principal Scientist, where she led drug discovery projects involving traditional high-throughput screening, as well as innovative approaches for gene and cell therapies. She also worked at Boehringer Ingelheim as a Director of Competitive Intelligence for projects in cardiometabolic space and research beyond borders. She is a co-inventor on many issued US patents related cell and gene therapies and has several publications in peer reviewed scientific journals. Revati holds a PhD in Tumor Immunology from the Tata Cancer Research Institute in Mumbai, India. She did her postdoctoral fellowship at the University of Connecticut Health Center and Boehringer Ingelheim.

Yssa DeWoody, PhD, is the new Global Site Engagement and Enrollment Lead at Longboard Pharmaceuticals. She started her career as a research mathematician working across diverse fields of study in academia and industry. This course changed dramatically after the birth of her youngest daughter who was diagnosed with a rare chromosomal disorder. Since that shift, she has acquired more than 15 years of experience in the rare epilepsy and neurodevelopmental space working closely with researchers, clinicians, patient advocates, and caregivers to drive positive change. In 2011, Yssa co-founded Ring14 USA, a non-profit committed to enhancing the quality of life for children impacted by rare neurodevelopmental disorders on the 14^th chromosome, like Ring 14, and currently serves as Director of Research. Her passion for collaboration and commitment to serve is epitomized by her involvement in several formidable consortiums, including Ring14 International (Cofounder and Past President), the Epilepsy Leadership Council, the Rare Epilepsy Network (ex officio Chair), and the Commission on Novel Technologies for Complex Copy Number Variants (founding partner). Yssa brings a unique perspective and personal passion for rare diseases to effective site engagement and thoughtful patient recruitment strategies.