Confirmed Speakers

Orphan Drugs & Rare Diseases Global Congress 2021 Americas

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Barry Ticho, M.D., PhD.,

Chief Medical Officer, Stoke Therapeutics, Inc.

As Chief Medical Officer Dr. Ticho is responsible for Stoke’s efforts to develop first-in-class RNA based disease-modifying medicines to treat severe neurologic genetic diseases. Prior to joining Stoke Barry was Head of R&D for Cardiovascular and Metabolic Diseases at Moderna Therapeutics.  He was previously Head of External R&D Innovation for Cardiovascular and Metabolic Diseases at Pfizer.  Prior to that he was Vice President of Clinical Development at Biogen where he led clinical development for the Tysabri program for MS and led the aducanumab program for Alzheimer’s Disease. Barry obtained his M.D. and Ph.D. degrees from the University of Chicago and completed Pediatrics training at Northwestern University and a Cardiology fellowship at Children’s Hospital in Boston. He was on clinical staff at Harvard Medical School and Massachusetts General Hospital. 

Rahul Ballal, PhD

President & CEO, Imara Inc.

Rahul has served as Imara’s President and Chief Executive Officer and as a member of the board of directors since June 2018. He has more than 15 years of life sciences experience in business development, strategic partnerships, venture capital, and company financing. Rahul joined Imara from Northern Biologics and Versant Ventures, where he served as Chief Business Officer and an Entrepreneur-in-Residence, respectively. At Northern Biologics, he built a first-in-class immuno-oncology portfolio, led a broad strategic partnership with Celgene, and managed several key functional roles at the executive level. At Versant, he negotiated foundational assets for existing portfolio and new companies, sourced several new deals, and participated in investment team activities.

Rahul was previously Vice President, Business Development at Flexion Therapeutics, where he led several business development transactions, supported multiple functions during Flexion’s initial public offering, and completed a key $175 million strategic partnership with Patheon UK. Prior to Flexion, he held business development and leadership positions at Novartis Venture Fund, Broad Institute of MIT and Harvard, and Redmind Inc. Rahul earned a B.S. in biology from Brown University, a M.S. in bioinformatics from Johns Hopkins University, and a Ph.D. in biochemistry from Georgetown University. He is also a Kauffman Fellow.

Andrea C. Furia-Helms, M.P.H.

Director, Patient Affairs Staff, Office of Clinical Policy and Programs, Office of the Commissioner, U.S. Food and Drug Administration

Andrea Furia-Helms is the Director of the Patient Affairs Staff in the Office of Clinical Policy and Programs, Office of the Commissioner. In her role, she collaborates with patient communities, the FDA medical product Centers, and other offices to incorporate patient and caregiver perspectives in cross-cutting regulatory meetings.  Ms. Furia-Helms spent over ten years in the FDA’s Office of Health and Constituent Affairs where she directed the FDA Patient Representative Program and coordinated patient engagement activities for the agency.

Prior to FDA, Ms. Furia-Helms was Director of the Back to Sleep (now Safe to Sleep) campaign, a public-private partnership to educate communities on Sudden Infant Death Syndrome (SIDS), at the National Institutes of Health.  She developed SIDS outreach initiatives for African American, American Indian, and Latino communities.

Ms. Furia-Helms has a B.A. in psychology from Framingham State University, a B.S. degree in community health education from the University of Maryland, and a Master of Public Health degree from The George Washington University.

 
 
 

Christopher Dandrea

Vice President, Global Program Management, uniQure

Chris Dandrea is a 25 year veteran in the Biopharmaceutical Research, Development and Commercialization space.  His current role as uniQure’s Vice President of Global Program Management is focused on scale-up, acceleration and productivity of uniQure’s growing Research & Development Pipeline of CNS/Neuro and Rare Gene Therapy programs.  Prior to uniQure, Chris was Sanofi’s Head of Oncology Project Management tasked to build a next-generation project management organization to support rapid oncology portfolio growth.   Additional experience includes Rare Disease Program Leadership at Genzyme, Life Science Management Consulting, Medical Device Engineering and service as a US Naval Officer.  Education includes Engineering degrees from Duke University and an MBA from Carnegie Mellon University.  

Christine Von Raesfeld

Founder & CEO, People with Empathy

As Founder/CEO of People with Empathy, Christine Von Raesfeld is a leader in bringing a critically needed patient perspective to cutting-edge medical innovations. Committed to providing patients with chronic and rare diseases with the support they need, Christine works with patient advocacy organizations, industry representatives, and individual patients and their loved ones. Living with many rare and chronic diseases, she believes that the only way to ensure health equity is to leverage one’s own data. As a patient involved in Stanford’s Humanwide program, she speaks on the benefits and advantages of precision medicine, with a special interest in pharmacogenomics. 

Christine’s drive to make lives better for patients has been nationally recognized. In 2019, she spoke on stage at the Startup Health Festival as an invited guest of Sanguine Biosciences. In 2018, Christine served on the Team of Patient Advisors for PatientsLikeMe. She has been nationally recognized for her work in advocacy and when several awards including Silicon Valley Business Journal’s100 Women of Influence, 2019 and a 2020 Community Hero by Assemblyman Kansen Chu. In addition, Christine serves as a Virtual Advisor Team member for the All of Us research program, a Technical Expert Panelists for CMS and a patient advisor/consultant or multiple pharmaceuticals off branded campaigns and startup ventures.

Haya Taitel

Senior Vice President, Chief Commercial Officer, Kadmon

Ms. Taitel is Senior Vice President, Chief Commercial Officer at Kadmon, where she is responsible for the strategy, tactical execution and forecasting of in-line promoted products, portfolio contracting strategies and the launch readiness of pipeline assets.

Haya leads commercial operation disciplines including marketing, sales, national accounts and contracting strategies across several therapeutic areas. She also oversees the commercial operation infrastructure, including the business analytics, market segmentation and customer service units.

Prior to joining Kadmon, Haya led the U.S. commercial franchises of both the Pharma and Device sectors of Johnson & Johnson. In her 25-year tenure with J&J, she launched new chemical entities in several therapeutic areas including neurology, analgesia, peripheral diabetic neuropathy, women’s health, and urology, with focus on interstitial cystitis.

Cherry T. Thomas, M.D.,

SVP Clinical Development, Caribou Biosciences, Inc.

Coming Soon

Deborah Collyar

President, Patient Advocates In Research (PAIR)

Coming Soon 

Monica L Weldon

President/CEO/Founder Bridge the Gap - SYNGAP Education and Research Foundation

In November of 2012, Monica’s twin son, Beckett, was the first to be diagnosed at Texas Children’s Genetics Clinic with the gene mutation SYNGAP1 (6p21.3). When Beckett was 4 months old, she noticed he was not meeting the same milestones as his twin sister. Monica then began a journey to find answers to help her son. She started to blog about his progress and this led to building a community of parents and caregivers that are now a strong support group. She is the Founder and President/CEO of Bridge the Gap – SYNGAP Education and Research Foundation. It is her passion to help support these families by raising awareness and creating a strong foundation that will accelerate a path to better therapies.

She retired in 2016 after 23 years in education teaching secondary science. Her new focus is on building the programs and mission of Bridge the Gap – SYNGAP Education and Research Foundation. She is the Primary Investigator on the SYNGAP1 (MRD5) Registry and Natural History Study. She is a life member of the Worldwide Association of Female Professionals and a member of the first class of 2017 Illumina Ambassadors established in the United States. In addition to leading the foundation, she is an author, public speaker, consultant on rare disease business strategies and advocates for rare disease legislation at both the federal and state levels. Several of her authored scientific publications include Nature Neuroscience, The Journal of Neurodevelopmental Disorders, and The Journal of Pediatrics. She has authored a book about her son Beckett’s diagnostic journey called “Slow Moving Stream – My Special Boy”. She is a graduate from East Texas Baptist University with a Bachelor’s of Science in Biology/Psychology (1991) and Secondary Certification in Education (1995). She will be attending Northwestern University Pritzker School of Law in the Fall of 2020 to earn her Masters in Science Law.  She has five beautiful children, Haleigh (28), Taylor, USMC (25), Sawyer (23), and the twins Beckett & Pyper (12). She was a Global Genes 2015 & 2016 RARE Champion in Advocacy Award nominee for her work in the rare disease community and WeGo Health Awards Nominee for years 2015, 2017, 2018, 2019, & 2020. She was named 2020 Global Shakers Rare Disease Champion.

Dr. Neena Nizar

Founder and Executive Director, Jansen's Foundation

Dr. Nizar serves as Founder and Executive Director of the Jansen’s Foundation. She is a rare disease advocate and Change Leader in the rare disease community, specifically in the field of ultra-rare skeletal dysplasias. Dr. Nizar has a Doctoral degree in Educational Leadership from Creighton University, Nebraska, is a TEDx speaker, a blogger and passionate voice for the special needs community. She is a Trailblazer for the Rare Advocacy Movement (RAM) and Nebraska’s Mother of The Year, 2018. Dr. Nizar is also a rare disease patient and a mother to two boys with Jansen’s Metaphyseal Chondrodysplasia – a disease that affects less than 30 people worldwide.

Melinda Bachini

Director of Advocacy, Cholangiocarcinoma Foundation

Melinda Bachini, is an 11-year survivor of cholangiocarcinoma. She was treated at the National Cancer Institute starting in 2012 with a clinical trial using Adoptive Cell Therapy. Due to her exceptional and breakthrough response to treatment at NIH, her story was published in the New York Times and other media. 

Melinda is married and the mother of six children, grandmother to one. She is passionate about patient advocacy. Her personal experience with previous and current treatments allows for great insight to patients participating in clinical trials.  Her position as Director of Advocacy for the Cholangiocarcinoma Foundation, allows Melinda to interact, support and educate others touched by this cancer.  She shares her story with as many cancer patients as possible in hopes of giving courage to those in need.

Melinda had previously volunteered for seven years with the Cholangiocarcinoma Foundation and in August of 2017 came on as staff. She also worked for over 15 years in the Emergency Medical Field as an EMT Paramedic.

Melinda serves as patient advocate for the NCI Hepatobiliary Task Force, the NCI Patient Advocate Steering Committee (PASC), ECOG-ACRIN GI Committee, ECOG-ACRIN Cancer Research Advocacy Committee, and the NCRA.  Melinda attended the 2016, 2017, and 2018 NCCS, CPAT Symposium and advocated on Capitol Hill. She participated as a panelist in the Cancer Moonshot – Community Oncology Event at the White House, told her personal story and was introduced by her state Senator for the Innovation and Access in Quality Cancer Care at the US Capitol. In June of 2017 she spoke with nine US Senators at NIH to share her personal story of how research has prolonged her life.  Melinda has served as a Consumer Reviewer for the Department of Defense for several years.

Luke Rosen

VP, Accelerated Development & Community Engagement, Ovid Therapeutics

Luke Rosen is Co-Founder of KIF1A.ORG, a non-profit organization working to discover treatment for children living with KIF1A Associated Neurological Disorder, a rare genetic disorder affecting his daughter, Susannah. Started in 2016, KIF1A.ORG works to forge efficient collaborations within the scientific and patient communities to accelerate development of therapeutics for KIF1A. 

Luke also serves as VP, Patient Engagement and Government Affairs at Ovid Therapeutics, a biotech working to develop treatments for rare neurological disorders. In his role at Ovid, Luke works closely with members of the rare disease community to encourage efficient and impactful collaboration among all stakeholders. Working closely with every function at Ovid, Luke ensures the patient and caregiver voice is central in decision making, clinical trial readiness and public policy.

Co-Executive Director, Bensfriends.org

Co-Executive Director, Bensfriends.org

Clasina is a retired Canadian teacher and school counselor who lives near Ottawa, ON. She puts her teaching and counseling skills to good use in managing the Member Experience side of Ben’s Friends. She guides community moderators, deals with members who are having difficulties on the sites, and takes responsibility for the overall experience of Ben’s Friends members. Having a rare disease herself, when she’s not working on Ben’s Friends, Clasina enjoys being home, but she also loves going away and indulging in international travel.

Angela Ramirez Holmes

Founder/President, Cal Rare

Angela Ramirez Holmes started her advocacy in rare diseases in 2013 when her then 8 year old son Andrew was diagnosed with a rare brain condition, an arteriovenous malformation or AVM. Angela is the founder and president of Cal Rare, a non-profit coalition of rare disease stakeholders dedicated to improving the lives of rare disease patients in California. Cal Rare started the California Rare Disease Legislative Caucus and was able to secure $2 million in the 2018 budget for a Whole Genome Sequencing Pilot Project for undiagnosed pediatric patients. In 2018, Angela was the recipient of the Rare Voice Award for State Advocacy from the EveryLife Foundation. She is the Principal of Alliance Campaign Strategies, a political consulting and public affairs firm working on local campaigns and issues. Angela has nearly twenty years of political and legislative experience on the national, state, and local levels. She is also an elected official, currently serving her second term on the Board of the Zone 7 Water Agency. Angela graduated from Saint Mary’s College of California with a B.A. in Politics and U.S. History.

Tom James Lambdin

Co-Executive Director, Bensfriends.org

Tom James, after a wide and varied career connected to research, medicine and business, looks after the technical and business side of Ben’s Friends. With his background in many aspects of medicine, and his long list of friends and contacts, he is also the Ben’s Friends medical fact checker.  Tom, a rare disease patient himself, lives in the western USA and if he ever had any spare time, he’d be fly fishing.

Femida Gwadry Sridhar

CEO and Founder, Pulse Inforframe Inc.

Femida is a pharmacist, clinical epidemiologist, and methodologist with over 25 years of experience in clinical trials, patient registries, knowledge translation, health analytics, and clinical disease outcomes. Femida has received more than $10 million in research funding as a principal investigator or co-investigator. She was awarded a career scientist award in 2006 from the Canadian Institute of Health Research and through her work founded the first knowledge translation health informatics lab in North America, creating a collaborative ecosystem for interdisciplinary research. Recently recognized as a lifetime fellow of CIHR for her contributions to global research with impact. Femida has published over 200 manuscripts and abstracts in top-tier journals and actively continues her professional interests in enabling technologies for better provider workflow and patient engagement. 

Femida is a founding member of the International Society for Pharmacoeconomics and Outcomes Research medication adherence group, which has provided an early glimpse into how to conduct research in adherence. She is an expert reviewer in North America and Europe on large-scale research network grants and a frequent international speaker on patient engagement, patient registries, innovation, health informatics, and data visualization. The recipient of numerous nominations, awards, and positive press, Femida has joined Springboard Enterprises as a female entrepreneur in digital health.

David A Pearce, PhD

President of Innovation, Research, & World Clinic, Sanford Health

David Pearce is President of Innovation, Research, & World Clinic for Sanford Health.  He completed his undergraduate Bachelor of Science Degree with honors in biological sciences at Wolverhampton Polytechnic in 1986.  He gained his PhD in 1990 at the University of Bath, UK, and did postdoctoral training at the University of Rochester, U.S., and Oxford University, UK.

Dr. Pearce heads the leading lab in Juvenile Batten disease research.  He has been researching Juvenile Neuronal Ceroid Lipofuscinosis (Batten disease) since 1997.  His research has led to the first clinical trial for Juvenile Batten disease.  He has published over 100 research papers on Batten disease. He also oversees a national registry for rare diseases known as the Coordination of Rare Diseases at Sanford (CoRDS).   He has served on numerous NIH review committees, has organized rare disease workshops for the National Institute for Neurological Disorders and Stroke (NINDS) arm of the National Institutes of Health (NIH) and is currently the vice-chair of the consortium assembly for the International Rare Diseases Research Consortium

(IRDiRC). 

As President of Innovation, Research, & World Clinic at Sanford, he oversees the development of research programs, including more than 450 researchers, eight research centers and more than 300 ongoing clinical trials.