2026 Speakers

Dr. Granados is Global Head of Rare Disease Medical Scientific Advocacy & Insights at Sanofi, where she focuses on rare disease evidence generation, HTA strategy, and patient-centered evidence frameworks in regulatory and access decision-making.

Amit Gulwadi is Vice President and Head of Digital and Transformation at Alexion Pharmaceuticals, where he leads efforts to modernize clinical development through data, technology, and operational innovation.

Angi Robinson is Senior Vice President, Specialty Areas at Premier Research, where she brings more than 24 years of experience leading rare disease, pediatric, and other specialty clinical development programs globally.

Annie Kennedy is Chief Mission Officer at the EveryLife Foundation for Rare Diseases, where she advances evidence-based policy, advocacy, and patient-engagement initiatives to make rare diseases a national public health priority.

Barrett Tenbarge is a partner in Faegre Drinker’s FDA and regulatory group and a former Senate HELP Committee general counsel, advising life sciences clients on FDA regulation, compliance, product development, public policy, and congressional strategy.

Dr. Henry is Founder and Chief Executive and Medical Officer of Aurelix Bio. He is a physician‑scientist and clinical development strategist with over a decade of experience leading cell & gene therapy trials.

Dr. Meltzer is Vice President of Clinical Research at Alexion Pharmaceuticals, where he leads clinical development efforts focused on advancing therapies for rare diseases.

Casey McPherson is the Founder & CEO of AlphaRose Therapeutics, a public-benefit biotech spun out from the To Cure A Rose Foundation, pioneering parent-led precision genomic medicine to develop ASO therapies, while leveraging equity crowdfunding to accelerate rare disease treatments.

Chris Garabedian is CEO of Xontogeny and Portfolio Manager of Perceptive Xontogeny Venture Fund, bringing deep rare disease and biotech operating experience from his leadership of Sarepta Therapeutics and his work backing early-stage life sciences companies.

Clark Paramore is the Head of Global Value Evidence Strategy at Biogen and a seasoned health economics and outcomes research expert with deep expertise in value demonstration for innovative therapies such as gene and rare disease treatments.

Craig Lipset is an advisor, advocate, and educator in applied innovation for medicine developement, currently serving as Co-Chair of the Digital Trials & Research Alliance, as Clinical Innovation Lead for the Buffalo Initiative, and on the boards or advisory boards of organizations dedicated to transforming medicine development.

Danielle Dong is a rare disease medical affairs and scientific advocacy leader with global experience across patient care, research, and patient-centered evidence generation, currently serving as Scientific Advocacy Lead, Global Medical Affairs, Rare Disease at Sanofi.

Dennis Akkaya is Chief Commercial Officer at myTomorrows, where he leads the company’s global commercial strategy, accelerating patient access to treatments through clinical trial matching, pre-approval access, and stakeholder engagement in rare disease research.

Dr. Pichard, Chief Science Officer at the International Rett Syndrome Foundation, is a physician-scientist and rare disease parent-advocate advancing Rett syndrome therapeutic development, with prior rare disease leadership at NIH/NCATS.

Effie Parks is the host of Once Upon a Gene, a rare disease podcast sharing stories from families, advocates, clinicians, and researchers, and is a parent advocate whose work is rooted in her family’s experience with CTNNB1 syndrome.

Dr. Duke is Senior Vice President, Drug and Biological Therapies at Canal Row Advisors, a pediatric neuro-oncologist and former clinical reviewer in oncology who brings deep prior experience from FDA review of pediatric and rare cancer therapies.

Holly Fernandez Lynch is an Associate Professor of Medical Ethics and Law at the University of Pennsylvania, whose work focuses on FDA policy, clinical research ethics, and access to investigational therapies.

Dr. Peay is a Senior Director at Faegre Drinker Consulting, specializing in patient-focused drug development, patient engagement, and bioethics research.

Drawing from her rare disease journey, previously captured in A Rare Journey, Hulda Björk Svansdóttir joins director Lainey Moseley for a live conversation on how mindset and purpose can help sustain joy and resilience, even when life feels difficult.

Dr. Caro is a globally recognized expert affiliated with LSE, McGill University, NUS, and Evidera known for pioneering innovations in health technology assessment, evidence integration, disease modeling, and healthcare value measurement.

Jake Caines is Chief Revenue Officer at Curant Health, where he leads commercial strategy and growth across employer, health plan, and life sciences markets, driving adoption of outcomes-based patient engagement solutions.

Dr. Berry is Chief, Division of ALS and Motor Neuron Diseases, Mass General Brigham, Associate Professor of Neurology at Harvard Medical School, and clinical researcher focused on ALS biomarkers and therapeutic development.

Dr. Woodcock is a renowned former FDA Acting Commissioner and longtime Director of the Center for Drug Evaluation and Research (CDER) who shaped modern drug regulation over four decades, including leading therapeutics for Operation Warp Speed.

Dr. Khushalani is National Lead, HEOR – Epilepsy & Rare Syndromes at UCB, a physician-scientist focused on real-world evidence and evidence strategy in neurology and rare diseases.

Dr. McKenzie is a Senior Clinical Program Leader at Boehringer Ingelheim and pediatric nephrologist focused on advancing clinical development and pediatric trial design in kidney and rare diseases.

Jennifer Panagoulias is Chief Operating Officer of AS2 Bio and Head of Regulatory and Policy at the Foundation for Angelman Syndrome Therapeutics (FAST), with more than 20 years of experience advancing global development and regulatory strategies for rare neurological disease therapies.

Julia Taravella is Founder and Executive Director of the Rare Trait Hope Fund, where she created an ultra-rare AGU AAV9 gene therapy program from parent-led advocacy into an FDA-cleared clinical trial, bringing unusual firsthand experience in patient-led drug development

After her daughter Mila was diagnosed with a rare genetic disease, Julia Vitarello drove an unprecedented collaboration to develop the first-ever drug tailored to a single person, and now leads an international movement to advance individualized medicines.

Kim Quaintance-Lunn is Vice President, Head of Regulatory Science & Execution at Alexion, where she brings 30+ years of pharmaceutical and regulatory leadership experience across FDA, Eisai, Bayer, and Alexion to advance rare disease therapy development.

Kris O’Brien is Director Program Strategy Rare Diseases at ERGOMED, focused on rare disease and oncology trials, specializing in operational strategy, patient-centric design, and trial execution in complex studies.

Lainey Moseley is an award-winning producer, director, and rare disease advocate whose documentary RARE: A Rare Disease Revolution spotlights families navigating the race to develop treatments for ultra-rare conditions.

Lauren Morgenroth is the CEO and co-founder of TRiNDS, a CRO specializing in neuromuscular and rare disease clinical trials, with deep expertise in multicenter trial operations and patient-focused research.

Marc O'Connor is a principal owner and Chief Business Officer for Curant Health, and a nationally recognized thought leader on health care delivery, collaboration, and alignment.

Dr. Binks is the Chief Medical Officer at Capricor Therapeutics, bringing over 25 years of experience in global clinical development and translational research, with a focus on immunology and rare diseases.

Michele M. Oshman is Chief Patient Advocate and Senior Vice President, Alliance Development at the Biotechnology Innovation Organization (BIO), where she leads patient advocacy efforts to elevate patient voice in biotech policy, partnerships, and access discussions.

Dr. Mellion is a rare disease clinical development leader who will speak at RTS on platform trials, drawing on her experience helping design and advance one of the earliest commercial rare disease platform trials in the UK.

Dr. Pastrnak is Chief Operating Officer of Gooseberry Research, where he helps advance patient-centered solutions for rare disease and pediatric clinical trials.

Molly Painter, is Vice President of Sales & Strategic Accounts at Takeda, where she leads U.S. Plasma-Derived Therapies commercial strategy and brings senior leadership experience spanning rare disease, neuroscience, biotech launch, and patient/community engagement.

Dr. Kreher is the Chief Medical Officer at Alltrna and Board member at Rezolute Bio; she is a pediatric endocrinologist and has spent her entire career dedicated to rare disease drug development.

Paula Orandash is Director, Patient Engagement at ERGOMED, advancing patient-centered strategies in rare disease clinical trials, including community engagement, recruitment, and integrating the patient voice into study design.

Rachel Smith is Vice President and Global Head of Rare Disease at Parexel, providing expertise on effective, expedited, and robust clinical development strategies for rare therapies to accelerate these much-needed options to rare patients as safely as possible.

Dr. Pryluk is CEO and co-founder of PhaseV, a health-tech company developing causal machine learning–based platforms for adaptive clinical trial design and execution.

Dr. Huml is an executive leader in the healthcare and biopharmaceutical industries, focused on enhancing patient access, engagement and insights, with a focus on rare diseases.

Ron Bartek is Co-Founder and President of the Friedreich’s Ataxia Research Alliance (FARA), a nationally recognized rare disease advocacy leader whose work spans patient-led research, policy, regulatory engagement, and coalition-building across the rare disease ecosystem.

Sarah Rhee, Vice President and Head of Regulatory Affairs at Alexion, is a global rare disease regulatory leader with experience spanning North America, Japan, and Asia-Pacific markets.

Dr. Batchelor is a pediatrician and Principal Investigator at Rare Disease Research, with a focus on expanding access to clinical trials and investigational therapies for patients with rare diseases.

Dr. Messahel is Chief Operating Officer at Aurelix Bio and a rare disease clinical development leader with deep experience in pediatric neurology, gene therapy, and patient-centered research, including earlier work in Rett syndrome and autism.

Stacey Frisk is the Executive Director of the Rare Disease Company Coalition and a seasoned policy and regulatory affairs professional with more than 14 years of experience spanning industry, patient organizations, insurers, and clinical research.

Tim Hunt is CEO of the Alliance for Regenerative Medicine, where he brings more than 20 years of biotechnology leadership experience to advancing cell and gene therapies, regulatory policy, patient access, and the broader rare disease treatment ecosystem.

Wendy Erler leads Patient Affairs at Sarepta across approved gene therapy and PMO RNA-targeted therapies, as well as its emerging siRNA pipeline, bringing a patient-centered, enterprise-focused approach to integrating patient insight into strategy, governance, and portfolio decision-making.

Dr. Yan is Senior Director of the Center for Genetic Surgery at the Broad Institute, Founding President of the N=1 Collaborative and a physician-scientist focused on advancing genetic medicines for patients with rare diseases underserved by traditional drug development.