Alison Lawton, BSc Hons

Chair, Dianthus Therapeutics

Pitch Track 3: Immune & Connective Tissue Function

Alison is Chair of the Board at Dianthus & TrimTech Therapeutics and a seasoned biotech executive with deep experience in orphan disease drug development. Over more than 20 years at Genzyme, she advanced therapies for underserved communities by leading global regulatory, policy, market access, and commercial teams. She later served as COO of X4 Pharma & Aura Biosciences and then as CEO of the publicly traded Kaleido Biosciences, all of which were pioneering new therapies for orphan diseases.

Amy Comstock Rick, J.D.

Director, Rare Disease Innovation Hub at the FDA

Session 4: Regulatory Update: Innovative Paths to Therapy Development & Scalability

Amy Comstock Rick, J.D., is the Director of FDA’s Rare Disease Innovation Hub (the Hub). She serves in a cross-cutting role across FDA’s Center for Drug Evaluations and Research (CDER), Center for Devices and Radiological Health (CDRH), and Center for Biologics Evaluation and Research (CBER) to facilitate implementation of the Hub. The Hub is a cross-center FDA initiative that promotes internal and external engagement for the purpose of advancing rare disease medical product development.

Ankit Malhotra Ph.D.

Global Head of Genomics, Strategy and Solutions, Amazon Web Services

Pitch Session – Main Stage

Ankit Malhotra, Ph.D., is Global Head of Genomics Strategy and Solutions at Amazon Web Services. He advises health systems, biobanks, and rare disease research programs on the cloud and AI infrastructure required to accelerate genomic diagnosis and therapeutic development at population scale — from newborn screening pipelines to federated data architectures that enable cross-institutional collaboration without compromising patient privacy.

Ashley Winslow, Ph.D.

CEO, CSO at Odylia Therapeutics

Session 1: Build it and They Will Come: Datasets that De-Risk Therapeutic Development & Attract Investment

Ashley Winslow, Ph.D., is CEO and Chief Scientific Officer of Odylia Therapeutics, a nonprofit biotech advancing treatments for rare diseases through partnerships with patient groups, researchers, CROs, and industry. Previously, she led portfolio development and translational research at the Orphan Disease Center (U. of Pennsylvania) and held roles at Pfizer, MGH and Harvard. She earned a Ph.D. in Medical Genetics from the University of Cambridge and serves on multiple rare disease advisory boards.

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Brett Abrahams, Ph.D.

President & Founder at Heppinn Biosciences

Pitch Track 1 Judge: Neurodevelopmental Disorders & Epilepsy

Dr. Abrahams is founder and President of Heppinn Biosciences, a consulting practice launched to support venture investors, biotechs, and foundations on scientific strategy and asset development. He’s also the CSO of the AUTS2 Research Collaborative and an advisory board member for Autism Speaks, CureShank, CombinedBrain, FAST, and Accelerator Life Science Partners. He has led R&D at public and private biotechs, and his peer-reviewed research has been cited more than 15,000 times.

Clark Paramore, MSPH

Head of Global Value Evidence Strategy at Biogen

Session 5: Market Access for Rare and Utrarare Communities: We Can’t Wait Until the Approval Comes

L. Clark Paramore, MSPH, is Global Head of Value Evidence Strategy at Biogen, Inc, based in Cambridge, MA. Clark leads a team of health economists focused on developing and executing on value demonstration strategies for Biogen’s pipeline products. Target therapeutic areas include Alzheimer’s Disease, rare neuromuscular diseases, lupus and nephrology. Clark is actively engaged in assessing the relevance and application of existing value frameworks for innovative therapies.

Dan Ollendorf, Ph.D., MPH

Chief Scientific Officer and Director of HTA Methods & Engagement at the Institute for Clinical and Economic Review

Session 5: Market Access for Rare and Ultrarare Communities: We Can’t Wait Until the Approval Comes

Dan Ollendorf is Chief Scientific Officer and Director of Health Technology Assessment (HTA) Methods and Engagement at the Institute for Clinical and Economic Review (ICER), with responsibility for managing the organization’s internal health economics capacity and program of external collaboration, as well as collaboration with international HTA bodies and other stakeholders on key process and methods topics. He also maintains a part-time faculty appointment at CEVR at Tufts Medical Center.

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Dana Layo-Carris, Ph.D.

Senior Study Director, The Jackson Laboratory

Interactive Breakout Session: Do I have the right models to Advance my Development

Dana Layo-Carris is a Senior Study Director at the Rare Disease Translational Center at The Jackson Laboratory, where she leads translational research programs focused on advancing therapies for rare genetic diseases. Her expertise spans disease modeling, preclinical development, ASOs, gene therapy, and gene editing. She is passionate about building partnerships across the rare disease community and accelerating the translation of scientific discoveries into meaningful treatment opportunities.

Erica Cox, Ph.D.

Executive Director, Head of Clinical Regulatory at Denali Therapeutics

Session 3: Successes and Lessons Learned from Recent Rare Disease Drug Approvals and Clinical Trials

Erica Cox, Ph.D., Executive Director, is the Head of Clinical Regulatory at Denali Therapeutics, where she leads regulatory strategy for therapies for rare and neurodegenerative diseases. She has over fourteen years of experience in clinical regulatory, including most recently leading the filing for a rare pediatric therapy under accelerated approval. She is passionate about partnering with patients, advocates, clinicians, and regulators to bring novel therapies to patients.

Hélène Dassule, Ph.D.

Head of Excellence, Scientific Communications, Alexion AstraZeneca Rare Disease

Interactive Breakout Session: From Community Insight to Scientific Impact: Publishing and Amplifying Rare Disease Evidence

Hélène Dassule, Ph.D, is the Head of Strategy and Excellence for Global Scientific Communications at Alexion, AstraZeneca Rare Disease. She leads strategies that support the delivery of meaningful, ethical, and transparent publications and scientific communications to healthcare professionals. Her career spans roles in scientific communications at Bayer and Parexel, as well as independent consulting in publications. She earned her Ph.D. in Molecular and Cellular Biology from Harvard University.

Jennifer Panagoulias, RAC

Head of Regulatory and Policy, Co-director of ABOM at FAST

Session 2: Therapeutic Timeline Deep Dive: Synergies and Handoffs that Matter Most

Jennifer has worked in drug development for over 20 yrs, primarily focused on advancing global development programs for the treatment of rare neurological diseases. She is the co-director of the Angelman Syndrome Outcome Measure and Biomarker Consortium (ABOM) and has recently taken on the role of Chief Operating Officer for AS²Bio, Inc., focused on accelerating drug development for individuals living with Angelman syndrome. She has a niece who lives with Angelman syndrome.

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Jill Weimer, Ph.D.

Senior Director, Therapeutic Development at Sanford Research

Session 2: Therapeutic Timeline Deep Dive: Synergies and Handoffs that Matter Most

Dr. Jill Weimer is a developmental neuroscientist and Senior Director of Therapeutic Development at Sanford Research. She bridges basic and clinical research to advance bench-to-bedside science and fosters partnerships with biotech and pharma. She earned her PhD from the University of Rochester and completed postdoctoral training at UNC before joining Sanford Research.

Joe Katakowski, Ph.D.

Director, Research at RTW Foundation

Session 1: Build it and They Will Come: Dataset that De-Risk Therapeutic Development and Attract Investment

Expert Office Hours

Pitch Track Judge: Neurodevelopmental Disorders & Epilepsy

Joe leads development strategy and R&D for RTW Foundation Rare Disease Advisory Program partners. He previously led gene therapy and AAV work at Regeneron and advanced multiple immuno-oncology programs to IND at Pfizer across diverse modalities. Earlier, he was a senior scientist at Innovimmune. Joe earned a PhD in immunology from Albert Einstein College of Medicine, with multiple first-author publications.

Krista Vasi, M.P.A.

Executive Director, Usher Syndrome Coalition

Pitch Track 2: Adult Neurologic & Vision-Related Disorders

Krista Vasi is Executive Director of the Usher Syndrome Coalition, a global nonprofit dedicated to Usher syndrome, the most common genetic cause of deafblindness. The Coalition bridges the gap between researchers, industry and a trial-ready community through the USH Trust, the world’s largest Usher syndrome contact database, and Global Genes’ RARE-X Data Collection Program. With the Coalition since its first year, Krista leads industry, advocacy and community partnerships worldwide.

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Lindsay Jesteadt, Ph.D.

CEO at Sleep Consortium, Inc.

Session 1: Build It and They Will Come: Datasets that De-risk Therapeutic Development and Attract Investment

Lindsay Jesteadt, Ph.D., is Co-Founder and CEO of Sleep Consortium, a nonprofit organization dedicated to accelerating sleep research through patient-centered data and collaboration. Following her son’s diagnosis with Type 1 Narcolepsy, she became a leading advocate for advancing sleep research, patient engagement, and innovation. She works globally with researchers, clinicians, industry, and patient communities to improve outcomes for individuals living with sleep disorders.

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Lindsay Marjoram, Ph.D.

Chief Scientific Officer at the Barth Syndrome Foundation

Session 2: Therapeutic Timeline Deep Dive: Synergies and Handoffs that Matter Most

Lindsay Marjoram, Ph.D. is the Chief Scientific Officer for the Barth Syndrome Foundation, which is dedicated to advocacy and advancing research for the development of treatments and cures for this ultra-rare disease. Dr. Marjoram has a background in developmental biology and a vested interest in rare disease research. Dr. Marjoram previously worked in biotech where she gained extensive experience in cutting edge gene therapies and rare diseases impacting vision loss.

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Marc Patterson, M.D.

Chief Medical Officer, IntraBio

Session 2: Therapeutic Timeline Deep Dive: Synergies and Handoffs that Matter Most

Marc Patterson was born in Australia and graduated in medicine there in 1981. He trained in child neurology at the University of Queensland and Mayo Clinic and in neurometabolic disease at NIH, under Roscoe Brady, MD. His research and practice have focused on rare inherited metabolic disorders, particularly NPC, LSDs and CDG. He became Emeritus Professor of Neurology, Pediatrics and Medical Genetics at Mayo Clinic in December 2024, and Chief Medical Officer at IntraBio, Inc in January 2025.

Maya Chopra, MBBS, FRACP

Clinical Geneticist at Boston Children’s Hospital and Assistant Professor at Harvard Medical School

Pitch Session – Main Stage Judge

Dr. Maya Chopra, MBBS, FRACP, is a Clinical Geneticist at Boston Children’s Hospital and Assistant Professor at Harvard Medical School. She is Division Chief of Neurogenetics and Neurodevelopment and Director of Translational Genomic Medicine at the Translational Neuroscience Center. Dr. Chopra’s research is focused on clinical trial readiness and therapeutic development for rare monogenic neurodevelopmental disorders.

Megan Abbott, M.D.

Pediatric Epileptologist at University of Colorado

Session 1: Build It and They Will Come: Datasets that De-risk Therapeutic Development and Attract Investment

Dr. Megan Abbott is a pediatric epileptologist at Children’s Hospital Colorado, embarking on a career in clinical research focused on outcome measures in developmental and epileptic encephalopathies (DEEs). She is working to establish multi-disciplinary clinics, generate gene-specific datasets, and devise outcome measures applicable to rare disease. She is excited to help advance the field further towards clinical trial readiness and disease modifying therapies for DEEs and other rare diseases

Michael Sherman, M.D., M.B.A., M.S.

Chief Strategy Officer at GeneSprout

Session 5: Market Access for Rare and Ultrarare Communities: We Can’t Wait Until the Approval Comes

Dr. Michael Sherman serves as Chief Strategy Officer for GeneSprout. which offers affordable Whole Exome and Whole Genome Screening together with best-in-class navigation and support services to consumers, employers, and health plans. access to cutting edge precision medicine.

Before pursuing an M.B.A. at Harvard Business School, Dr. Sherman received his M.D. from Yale and holds B.A. and M.S. degrees from the University of Pennsylvania.

Michelle Davis

Executive Director, International FOP Association (IFOPA)

Session 6: Reimagining Industry–Advocacy Collaboration in Drug Development

The majority of Michelle’s career has been spent working in patient advocacy—the national headquarters of the National Kidney Foundation and Polycystic Kidney Disease Foundation and 10 years leading the IFOPA, an ultra-rare bone disease. At the IFOPA, Michelle leads the staff team and partners with the Board of Directors to execute the organization’s strategic plan. She provides strategic leadership for fundraising activities, global outreach and manages the IFOPA’s relationships with industry.

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Nasha Fitter, MBA

CBO, Citizen Health

Opening Fireside Chat

Nasha Fitter is dedicated to transforming rare disease drug development. After her daughter’s diagnosis, she co-founded the FOXG1 Research Foundation, pioneering faster, more cost-effective paths to clinical trials. She is also Co-Founder and Chief Business Officer of Citizen Health and serves on the board of the Buffalo Initiative. A Harvard Business School MBA, Nasha is committed to accelerating treatments and cures for rare diseases.

Reenie McCarthy, J.D.

CEO at Stealth BioTherapeutics

Session 3: Successes and Lessons Learned from Recent Rare Disease Drug Approvals and Clinical Trials

Reenie McCarthy is CEO and a Board member of Stealth BioTherapeutics, a clinical-stage biopharmaceutical company developing investigational therapies for rare and age-related diseases linked to mitochondrial dysfunction. She previously led the U.S. investment team at Morningside Ventures and serves on the Board of Biotechnology Innovation Organization. Reenie earned a J.D. from the University of Pennsylvania Carey School of Law and a B.A. from Bates College.

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Rohita Sharma, Ph.D.

Global Head – Strategic Alliances & Patient Advocacy, Alexion AstraZeneca Rare Disease

Interactive Breakout Session: From Community Insight to Scientific Impact: Publishing and Amplifying Rare Disease Evidence

Rohita Sharma is Head of Global Strategic Alliances & Advocacy at Alexion, AstraZeneca Rare Disease. She leads global partnerships across the rare disease ecosystem to advance patient-centered innovation, evidence generation, and equitable access to care. With 15+ years of experience spanning medical affairs, patient experience, and corporate functions, she brings deep expertise in rare disease strategy, stakeholder engagement, and patient-centered healthcare. She holds a PhD in Neuroscience.

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Sean Kassen, Ph.D.

Director of the Ara Parseghian Medical Research Fund, University of Notre Dame

Session 2: Therapeutic Timeline Deep Dive: Synergies and Handoffs that Matter Most

Sean Kassen, director, is responsible for administering all of the functions of the Parseghian Fund which includes advancing Niemann-Pick Type C (NPC) disease research, fundraising, managing the grants program, and raising awareness of NPC disease. He is well known for taking all of the credit for the accomplishments of the APMRF, though he knows it is his staff and team of volunteers that actually do the work!

Tai Pasquini, Ph.D., MPA

Chief Research Officer at Congenital Hyperinsulinism International

Session 6: Reimagining Industry–Advocacy Collaboration in Drug Development

Tai Pasquini is the Chief Research Officer at Congenital Hyperinsulinism International, where she leads the HI Global Registry, the Centers of Excellence program, and the Collaborative Research Network. A rare disease advocate and researcher, she holds a PhD in Public Health from UMass Amherst and a Master’s in Public Administration from American University. She previously worked at NORD and serves as chair of the research subcommittee of the Massachusetts Rare Disease Advisory Council.

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Wendy Chung, M.D., Ph.D.

Physician in Chief at Boston Children’s Hospital

Pitch Track Judge: Neurodevelopmental Disorders & Epilepsy

Dr. Chung is a clinical and molecular geneticist. She directs NIH-funded research in human genetics of autism, pulmonary hypertension, breast cancer, obesity, diabetes, and birth defects—including congenital diaphragmatic hernia and congenital heart disease. Dr. Chung has identified the genetic basis of more than 60 rare diseases. She leads the GUARDIAN study to expand newborn screenings for genetic disorders and advance development of personalized therapies for children with these conditions.

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

CEO & Founder, Mahzi Therapeutics

Day 3 Kickoff: De-Risking Your Disease to Attract and Sustain Investment

Pitch Session Judge – Main Stage

Yael Weiss founded Mahzi Therapeutics to develop therapies for ultra-rare genetic neurodevelopmental disorders. Mahzi partners directly with patient foundations — supporting their path toward drug development and bringing programs in-house once pre-clinical proof of concept is established.

Yael earned her MD from Hadassah Medical School at the Hebrew University in Jerusalem and her PhD from the Weizmann Institute of Science in Rehovot, Israel.

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