Meet our 2025 Speakers

Alpa Khushalani serves as the Vice President of Client Success & Delivery at Gooseberry Research. She specializes in implementing hybrid trial models that combine traditional and decentralized elements to optimize study execution. She will share insights on operationalizing pediatric research home health services for rare disease trials.

Amy Apostoleris, MBA, is CEO of DFnet and Cofounder of PatientX, bringing over 30 years of leadership in clinical research and biopharma to advance patient-centric trial design and global rare disease data solutions.

Amy Rick, JD, FDA’s inaugural Director of Strategic Coalitions for the Rare Disease Innovation Hub and CDER’s Associate Director for Rare Disease Strategy, brings over two decades of policy, advocacy, and government experience—most recently as Principal Consultant at Leavitt Partners and former President & CEO of the Food and Drug Law Institute—to lead cross-center engagement and external partnerships that accelerate rare disease product development.

Dr. Henry is a Medical Director, Cell and Gene Therapy Center of Excellence at IQVIA. He is a physician‑scientist and clinical development strategist with over a decade of experience leading cell & gene therapy and oncology trials — from early safety through phase IV and global regulatory harmonization.

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—beginning with “Rosiphersen” for his daughter’s ultra-rare condition—while leveraging equity crowdfunding to accelerate rare disease treatments

Dr. Theodore‑Oklota is Vice President, Clinical Outcomes Research at Mahzi Therapeutics and previously with Ultragenyx, where she led real-world evidence and patient burden studies—such as on burosumab and Kabuki syndrome—bridging clinical research and health outcomes science for rare disease therapies.

Christopher Rudolf is the CEO of Volv Global, specializing in leveraging natural language processing and artificial intelligence to enhance real-world data analysis in rare disease clinical trials. He will present on the integration of AI and NLP in analyzing real-world data to improve trial design and patient recruitment in rare diseases.

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. He will discuss how to strategically design rare disease trials that generate robust evidence for payers, ensuring market access considerations, all integrated from the outset.

Craig Lipset is the Co-Chair of the Decentralized Trials and Research Alliance (DTRA). With a background in clinical innovation, he advocates for the integration of decentralized methodologies to enhance trial efficiency and patient access. He will discuss strategies to expedite the adoption of decentralized trials in rare diseases, highlighting technological advancements and regulatory considerations.

Craig Martin is the CEO of the Orphan Therapeutics Accelerator. He brings experience in fostering collaborations between nontraditional sponsors, such as patient advocacy groups, and industry stakeholders to advance rare disease research. He will discuss the increasing role of nontraditional sponsors in rare disease trials, exploring how these entities contribute to innovation and patient-centric research approaches.

David Vulcano is the Vice President of Research Compliance & Integrity at HCA Healthcare, with a focus on integrating AI technologies to improve patient recruitment strategies in clinical research. He will discuss the use of AI in identifying and recruiting patients for rare disease trials, addressing challenges and solutions in this domain.

Dennis Akkaya is Chief Commercial Officer at myTomorrows, where he leads global commercial strategy for patient access—drawing on his extensive background in expanded‑access programs, AI‑enhanced clinical trial matching, and cross‑functional collaborations to improve pre‑approval access and engagement in rare disease research.

Donna Mackey is the Vice President of Clinical Operations at Akouos, part of Eli Lilly, overseeing clinical trial execution across various therapeutic areas. She will share insights on integrating operational strategies to enhance trial efficiency and data quality.

Dr. Warner is the Chief Medical Officer at Tyra Biosciences, bringing extensive experience in clinical development and trial design for rare diseases. He will discuss innovative strategies to overcome common trial design challenges in rare diseases, including patient recruitment and endpoint selection.

Dr. Dashiell-Aje is the Executive Director and Head of Patient-Centered Outcomes Science at BioMarin Pharmaceutical, leading efforts to incorporate patient perspectives into clinical outcome assessments. She will discuss the development and implementation of patient-centered outcome measures in rare disease trials, highlighting the role of digital health technologies.

Dr. Neto is the Global Head of Country and Site Operations and Executive Director at Biogen, specializing in site selection and operational strategy. He will discuss advanced methodologies for site selection that go beyond traditional feasibility surveys to enhance trial efficiency.

Dr. Peay is a Senior Director at Faegre Drinker Consulting, specializing in patient-focused drug development, patient engagement, and bioethics research. She brings extensive experience in integrating patient perspectives into clinical and public health research, with a focus on rare diseases, genomic technologies, and ethical considerations in emerging therapies.

Ilan Ganot, MBA, is the co-founder and former CEO of Solid Biosciences and now CEO of Alesta Therapeutics, drawing on a decade of Wall Street investment-banking experience and personal Duchenne patient advocacy to advance novel oral small-molecule therapies for rare diseases.

Iwona Tongbhoyai is Chief Client Solutions Officer at FutureMeds, where she leads the company’s European decentralized clinical trials strategy—overseeing client services, proposal and feasibility teams, and driving industry-wide efforts to enhance patient diversity, site engagement, and operational excellence. She will discuss her experience with operationalizing hybrid global rare disease trials.

Jake Caines is spearheading commercial strategy, sales, and marketing at Curant Health, with over a decade of experience in neuroscience, renal, gastrointestinal, and regenerative medicine.

Dr. Maynard is the Director of the FDA’s Office of Rare Diseases, Pediatrics, Urologic and Reproductive Medicine at the FDA, where she has led the agency’s efforts in orphan drug, rare pediatric disease, and humanitarian device designation programs—overseeing legislatively mandated grant and designation initiatives and serving as the FDA’s primary liaison with patients, sponsors, and stakeholders in the rare disease field.

Dr. Guptill is the Disease Area Development Lead for late-stage neuromuscular clinical trials at argenx and oversees the late-stage clinical development programs for efgartigimod (FcRn inhibitor) and empasiprubart (C2 complement inhibitor). He has served as a principal investigator for NIH supported clinical trials in academia and currently supports late-stage, global, clinical development programs in neuromuscular diseases at argenx.

Jen Shumsky is a Founder of JLS Consulting and specializes in market access and reimbursement strategies, leveraging her nursing background to bridge clinical and payer perspectives. She will emphasize the importance of early engagement with payers during Phase 2 trials to align clinical outcomes with reimbursement requirements.

Dr. Tanner is an Assistant Professor of Neurology at the Biggs Institute for Alzheimer's and Neurodegenerative Diseases at UT Health San Antonio, specializing in early-onset dementia and chronic traumatic encephalopathy. He is also a father of a child with an ultrarare, randomly-acquired neurodevelopmental condition and a Co-Founder of the Channeling Hope Foundation.

John Hall is the President of Shore Research & Development and Director of Clinical Operations at REGENXBIO, focusing on performance metrics in clinical trials. He is will discuss the development and utilization of KPIs and dashboards to monitor and drive trial performance.

Dr. Lin is Senior Vice President of Clinical Development and Clinical Pharmacology at Travere Therapeutics, leveraging her nephrology and public-health expertise to advance innovative kidney therapies. She will present on the topics of real-world evidence and how to align trials with patient-lived experiences.

Kelly Kilpatrick is Senior Vice President, Head of US Commercial Ocular Gene Therapy at Spark Therapeutics/ Roche, with extensive experience in commercial strategy, market access, and cross-functional leadership. She will share insights on integrating market access considerations early in the clinical development process, emphasizing strategies to align cross-functional teams to ensure successful product launch and patient access.

Ken Harvey is the Director of Clinical Operations at Epicrispr Biotechnologies, with expertise in strategic planning and execution of clinical trials. He will discuss the integration of strategic planning, execution, and accountability measures to enhance trial outcomes in rare diseases.

Dr. Musunuru is the Barry J. Gertz Professor at the University of Pennsylvania and co-founder of Verve Therapeutics, specializing in gene editing therapies for cardiovascular and metabolic diseases. He is will discuss the application of gene editing technologies in platform trials for inherited cardiovascular diseases.

Kristi Clark, Senior Vice President of Clinical Operations & Data Management at Avidity Biosciences, channels over 30 years of global rare-disease trial leadership—including co-founding Agility Clinical and building operations in Europe—into relentlessly patient-centric development strategies. She will share her experience operationalizing decentralized and hybrid rare disease trials.

Dr. Pisani is Vice President of Clinical Development at Taysha Gene Therapies, with a background in medical genetics and extensive experience leading gene therapy programs for rare neurological disorders. She will discuss common trial design challenges specific to gene therapy study protocols.

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. She will share strategies for designing and implementing KPIs and cross-functional dashboards that improve transparency, collaboration, and decision-making in rare disease clinical trial operations.

Leila Cupersmith is the CEO of Choice ClinOps, with extensive experience in global oncology and rare disease clinical trial operations. She will discuss best practices for operational excellence in complex, global rare disease trials.

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. Marc has experience in the pharmacy supply chain component of the commercialization of biopharmaceuticals, as well a deep history in patient support and engagement.

Mandi Walters serves as Executive Vice President at Sciensus, focusing on patient-centric strategies and the implementation of precision medicine in clinical trials. She will discuss operational aspects of precision medicine trials, emphasizing patient engagement and data management.

Marcella Lynch is Head of Pricing, Forecasting, and Contracting for the Americas Market Access Rare Disease division at Chiesi USA, bringing over 15 years of experience driving strategic pricing and access decisions for rare disease therapies across the biotech and pharmaceutical landscape. She will share practical strategies for initiating meaningful pricing and access conversations with payers during Phase 2.

Dr. Hames is the CEO of BioLogic Pharma Solutions, bringing expertise in strategic planning and commercialization of pharmaceutical products. She will discuss challenges and solutions encountered during the commercialization of rare disease therapies, drawing from her consultancy experience.

Dr. Pletcher is the Chief Scientific Officer at Weaver Biosciences, with a background in leading gene therapy programs at Astellas, Roche, and Pfizer. He will share insights on the development and implementation of platform trials across multiple rare diseases.

Megan Parisi is the Patient Solutions Lead at Syneos Health, specializing in the integration of digital health technologies to enhance patient engagement in clinical trials. She will explore the use of digital tools to collect patient-reported outcomes and improve trial efficiency.

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. He will lead a discussion on pragmatic strategies to overcome trial design challenges, including patient heterogeneity, endpoint selection, and global site feasibility.

Mindy Cameron is the President of AdvocacyWorks, bringing extensive experience in patient advocacy and ethical considerations in clinical research. She specializes in developing frameworks for transparent communication between researchers and participants. She will explore ethical strategies for returning individual patient data, emphasizing the importance of transparency and trust in the patient-researcher relationship.

Dr. Trifillis is the Senior Vice President of Medical Affairs & Scientific Communications at Rapid Commercialization Partners, with prior experience at PTC Therapeutics. She specializes in strategic planning and communication in rare disease clinical development. She will discuss common pitfalls in rare disease trials and strategies to mitigate risks, ensuring successful trial outcomes.

Pat Furlong is the Founding President and CEO of Parent Project Muscular Dystrophy (PPMD), the largest U.S. nonprofit dedicated to Duchenne muscular dystrophy, which she established in 1994 after losing her two sons to the disease, and she is recognized globally as a leading advocate and authority in rare disease research, policy, and patient-centered care.

Paul Kidwell is a seasoned public relations and patient advocacy consultant in the life science industry, founder and co-host of the In Sickness and Openly Rare podcasts, and a passionate rare disease caregiver whose writing—like the Openly Caring column in Rare Revolution Magazine—amplifies caregiver voices and integrates family perspectives into drug development and policy discussions.

Dr. Brooks is the Deputy Director of the Division of Rare Diseases Research Innovation at NCATS NIH, focusing on developing platform approaches to expedite gene therapy trials. He will discuss collaborative frameworks and regulatory considerations for platform trials in gene therapy.

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. She will discuss operational excellence in global, multisite rare disease trials.

Dr. Huml is Vice President of Rare Disease Strategy at Sciensus, with over 30 years in biopharma and a focus on rare disease commercialization and patient engagement. He is a published author and former Global Head of the Rare Disease Consortium at Syneos Health. He will explore how rare disease trials are evolving with decentralized models, real-world evidence, and digital health tools, and how global commercialization strategies must adapt to ensure patient access.

Renata Shih, MD is a pediatric cardiologist with over a decade of expertise in pediatric heart failure, transplantation, and clinical research, now serving as Principal Investigator on multiple neuromuscular gene therapy trials at Rare Disease Research in Atlanta.

Richie Kahn is the Co-Founder & COO of Canary Advisors, focusing on patient engagement and recruitment strategies in clinical trials. He will share insights on effective patient recruitment methodologies and engagement tactics across diverse populations.

Dr. Paganoni is an Associate Professor in the Department of Physical Medicine and Rehabilitation at Harvard Medical School and a physician at Massachusetts General Hospital. She specializes in ALS research, with an emphasis on clinical trials and biomarker development. She will share insights on precision medicine approaches in rare disease trials, highlighting the integration of biomarkers to enhance trial efficiency and outcomes.

Dr. Kassim is Chief Technology Officer of Genetic Medicines at Danaher. He is a translational research and manufacturing expert and has contributed to 15 first in human trials and three BLAs in the field of genetic medicines.

Dr. Hopkins serves as Senior Vice President of Therapeutics at AskBio, a subsidiary of Bayer AG, where he leads gene therapy programs targeting rare neurological disorders. He will explore the application of gene therapy in rare disease trials, focusing on design considerations unique to these therapeutic approaches.

Seth Rotberg is the Senior Manager of Patient Advocacy at Solid Biosciences. As a patient advocate living with Huntington's disease, he leverages his personal experience to inform patient-centric approaches in clinical trial design. He will lead discussions on incorporating patient-lived experiences into trial design, ensuring that studies are aligned with the needs and preferences of participants.

Dr. Patel is a Field Medical Director specializing in Rare Disease Cardiology at Pfizer and a parent of a child with Metachromatic Leukodystrophy. Her dual perspective as a medical professional and caregiver informs her advocacy for patient-centered research. She will share insights on the experiences of patients and caregivers in rare disease trials, emphasizing the importance of empathy and support throughout the research process.

Dr. Babu is an Associate Professor of Neurology at Harvard Medical School and a neurologist at Massachusetts General Hospital. Her research focuses on neuromuscular disorders and clinical trial design, particularly in ALS. Dr. Babu will discuss the implementation of adaptive trial designs in neuromuscular disorders, emphasizing the benefits of platform trials in accelerating therapeutic development for rare diseases.

Dr. Sparks is a rare disease clinical development consultant with prior experience at Sanofi-Genzyme, focusing on translating clinical insights into successful commercialization strategies. She will share real-world examples of rare disease product launches, emphasizing the integration of clinical development and market access planning.

Suzanne Gaglianone serves as patient representative at Reveragen BioPharma and is the founder of Patient Focused Travel. She brings considerable experience with patient clinical trial travel and patient focused trial design. She will discuss how trial sponsors can share individual patient data.

Dr. Defay is the Deputy Head of Diagnostics at Alexion AstraZeneca, contributing to the development of precision medicine approaches in rare disease treatment. He will share insights on implementing precision medicine strategies in clinical trials, including biomarker integration and patient stratification.

Tim Mooney is Senior Director and Program Leader for ALN‑APP and ALN‑SOD at Alnylam Pharmaceuticals, with extensive commercialization and market access expertise in advancing RNA interference therapies for rare indications. He will discuss successful case studies of commercializing rare disease therapies.