Conference Speakers

Dr. Donald B. Kohn

Dr. Donald B. Kohn is a physician-scientist specializing in gene therapy for blood disorders. He earned his M.D. and trained in pediatrics at the University of Wisconsin and performed a fellowship in pediatric immunology at the National Cancer Institute.

For over three decades, he has advanced gene therapy for immune deficiencies and hemoglobin disorders, leading research at UCLA since 2009. A founding member and past president of the American Society of Gene and Cell Therapy, Dr. Kohn has pioneered clinical trials and mentored future scientists.

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Dr. Paul Ayoub

Dr. Paul Ayoub is the Chief Executive Officer of Rarity PBC, a company dedicated to developing gene therapies for rare diseases. He holds a Ph.D. in Medical and Molecular Pharmacology and an MBA from UCLA Anderson School of Management.

Under his leadership, Rarity PBC focuses on addressing ultra-rare diseases, aiming to bring attention and innovative treatments to these often-overlooked conditions. Dr. Ayoub is particularly interested in the Public Benefit Corporation (PBC) model as a means of balancing financial sustainability with patient-first priorities, ensuring that life-saving treatments remain accessible rather than being driven solely by profit incentives. He actively engages in discussions about the impact of Priority Review Voucher (PRV) programs on rare disease research and therapeutic accessibility, as well as the role of alternative pricing models in ensuring long-term viability for gene therapies. His commitment to advancing gene therapy solutions reflects a deep dedication to improving the lives of patients with rare diseases.

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Dr. Arjan Lankester

Professor Arjan C. Lankester is a leading pediatrician-immunologist specializing in stem cell transplantation for inherited immune disorders. He earned his medical degree from Leiden University and his Ph.D. from the University of Amsterdam, focusing on B cell receptor signaling.

Since 2009, he has led the pediatric stem cell transplantation program at Leiden University Medical Center (LUMC), where he is also a professor of Pediatrics and Stem Cell Transplantation.

Professor Lankester’s research focuses on improving stem cell therapy efficacy and safety, particularly in optimizing conditioning regimens and immune reconstitution. He is the principal investigator of the RECOMB trial on lentiviral gene therapy for RAG1 SCID and has led numerous investigator-initiated trials. A co-founder of the AGORA consortium, he is committed to expanding access to gene therapies. He has also chaired key European working groups, shaping advancements in transplantation and immune deficiency care.

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Dr. Svenja Espenhahn

Svenja Espenhahn (PhD) is the manager of the Canadian Rare Disease Network (CRDN), a growing pan-Canadian network focused on uniting Canada’s clinical, scientific, and patient expertise to address the unique challenges faced by rare disease patients and their families.

Svenja provides strategic leadership and oversees operations of CRDN and its diverse activities for the benefit of the rare disease community. She is passionate about innovations that bridge the gap between research and care, and fostering collaboration across healthcare, academia and patient communities to build a more equitable and effective rare disease ecosystem in Canada.

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Dr. Charlotte Moore Hepburn

Dr. Charlotte Moore Hepburn is a faculty paediatrician in the Division of Paediatric Medicine at the Hospital for Sick Children, an Associate Professor of Paediatrics in the Department of Paediatrics at the University of Toronto School of Medicine and an Adjunct Professor at the Institute of Health Policy, Management and Evaluation (iHPME) at the University of Toronto.

Dedicated to a career combining clinical care with health system advocacy, she has held several leadership roles both in government and at national non-governmental organizations in both Canada and the United States. She now proudly serves as the inaugural Medical Lead for the Child Health Policy Accelerator at the Hospital for Sick Children, Canada’s first hospital-based applied child and youth health and social policy initiative dedicated to closing the gap between what we know is effective in optimizing paediatric outcomes and what we do to attend to the needs of young people in the public policy space.

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Dr. Thierry Lacaze-Masmonteil

Dr. Thierry Lacaze-Masmonteil is the RareKids-CAN Nominated Principal Investigator and the Scientific Director of MICYRN since 2018 and Clinical Professor of Pediatrics at the University of Calgary (UC).

He has been the inaugural Scientific Director of the Women and Children Health Research Institute (WCHRI, U of A) from 2006 to 2010, and the Scientific Director of the Clinical Research Unit at the Children's Hospital of Eastern Ontario (CHEO) from 2011 to 2015. Thierry is a Clinical Trialist Neonatologist. Under his tenure, MICYRN has acquired the expertise to function as a de-centralized Academic Research Organization (ARO), a first in Canada for the non-oncology pediatric clinical trial space. MICYRN currently provides ARO services to  more than 15 teams of CIHR-funded investigators. 

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Emma Iverson

Emma completed her Master of Science in Epidemiology at the University of Ottawa in 2024, with a focus on ophthalmic epidemiology and visual impairment health outcomes in Canada.

Throughout her graduate studies, she had the opportunity to work with INFORM RARE, a Canadian research network that is co-designed by patients and families, healthcare providers, policy-makers, methodologists, and research ethicists to improve clinical care, outcomes, and health policy for children with rare genetic diseases. This experience inspired her interest in rare disease research and family and patient engagement in research. Currently, Emma is a research coordinator at the University of Ottawa, contributing to the Registry Sub-Platform for the RareKids-CAN Pediatric Rare Disease Clinical Trials and Treatment Network. Her work aims to advance the role of patient registries in the generation and use of evidence to inform decision-making about rare disease therapies, particularly in pediatric rare disease clinical trials. 

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Tomasz Czarny

Tomasz Czarny is the Executive Director of the Precision Child Health Initiative at SickKids  and the Azrieli co-director of the Precision Child Health Partnership between SickKids and CHU Sainte-Justine.

He has over 10 years of experience leading cross-functional teams in developing, implementing, and leading high-impact projects, platforms, and operational programs within health research and clinical care. He first joined SickKids in 2016. Tomasz holds an MBA from the Schulich School of Business & a PhD in biomedical sciences from McMaster University. 

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Jason Arsenault

Jason is a parent to three children, with the middle child diagnosed with Dravet Syndrome, a rare form of severe epilepsy. This syndrome drastically changed his life as a parent and severely limited his family’s ability to thrive.

He has witnessed his partner advocate for diagnosis, treatment options, trial participation, and inclusion in everyday life for their child. Jason is excited about the opportunity RareKids-CAN will provide to patients, families, and those working in the industry. He looks forward to sharing his lived experience as a parent and leveraging his skills as a regulatory compliance professional for the Health Canada Relations & Advocacy sub-committee.
Jason enjoys playing hockey and coaching his youngest daughter, and he is a big fan of softball for his oldest daughter. He has a passion for various kinds of fishing, home projects, and lifelong learning.

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Breanne Stewart

Breanne Stewart (BSc, BScN, RN) serves as the Network Director for RareKids-CAN: Pediatric Rare Disease Clinical Trials and Treatment Network- she provides strategic leadership, oversees operations, and drives collaboration within the network.

With a focus on shaping RareKids-CAN's direction, Breanne is dedicated to fostering partnerships and ensuring the delivery of high-quality clinical trials and innovative research initiatives for individuals affected by rare diseases. Having joined MICYRN in 2018 as the inaugural Associate Director of Clinical Trials, Breanne is deeply passionate about optimizing research processes in Canada to ensure timely and efficient treatment and access to therapies for patients and their families.

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Alicia Hilderley

Alicia Hilderley partners with families and children to create meaningful patient-oriented research across the clinical trial pipeline.

She is a Facilitator of Patient & Family Engagement with RareKids-CAN, Instructor with CanChild's Family Engagement in Research program, postdoctoral fellow at the University of Calgary, and Patient Partner at The Ottawa Hospital. Alicia has trained in Rehabilitation Sciences & Neuroscience (PhD) and Kinesiology (MSc, BScHon). Her research focuses on the design, evaluation, and implementation of child-centred neurorehabilitation interventions in collaboration with family partners and multi-disciplinary teams.

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Beth Potter

Beth Potter is a Professor of Epidemiology and Public Health and the University Research Chair in Health Services for Children with Rare Diseases at the University of Ottawa; and an Affiliate Investigator at the Children’s Hospital of Eastern Ontario Research Institute.

Her research aims to produce evidence to improve health care and outcomes for children with rare genetic diseases. She is Principal Investigator for the INFORM RARE research network, which brings together patients and caregivers, clinical providers, and methodologists in the design of patient registries and clinical trials for specific inherited metabolic and neurologic diseases. She also leads the Registry sub-platform for the RareKids-CAN Pediatric Rare Disease Clinical Trials and Treatment Network.

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Sara Pot

Sara is an educator, facilitator, and parent of four, including two fantastic teens who require constant care specific to their rare diagnosis.

She is a Facilitator of Patient & Family Engagement with RareKids-CAN and a Coordinator with CanChild's Family Engagement in Research program. In addition to several volunteer advisor roles, she is a writer and presenter on the topic of faith, disability & belonging and a long time learner of implementation science.

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Kelly Sandhu

Kelly Sandhu (MSc, BSc) is the project manager for RareKids-CAN, providing management leadership. She has a Master's in Health Psychology and has over ten years of experience in academic and clinical research across Europe and Canada.

She has worked with multiple speciality teams in pediatric research, such as rheumatology, neurology, endocrinology and renal. Kelly has also managed and delivered research in pediatric rare diseases, such as lupus, vasculitis and ataxia-telangiectasia.
With a strong passion for research and evidenced based practice, Kelly was awarded the Edward Jenner Leadership Award from the National Health Service in the UK. This has contributed to her leadership approach when training and mentoring research staff, students, and volunteers.

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Nicola Wright

Dr. Nicola Wright completed fellowship training in Pediatric Hematology/Oncology with a focus on Immunology at Cincinnati Children’s Hospital Medical Center and Stanford University, following which she returned to her hometown of Calgary to practice a hybrid of benign hematology and clinical immunology at the Alberta Children’s Hospital. 

She holds the Barb Ibbotson Chair in Pediatric Hematology.
Her research interests include developing a platform for gene editing for blood and immune disorders. She is excited to be collaborating with Dr. Don Kohn, University of California, Los Angeles, on a Canadian CIHR funded gene therapy clinical trial for CD3delta severe combined immune deficiency, one of the more common types of SCID in Alberta. She is advocating for increased gene therapy access and clinical trials for pediatric rare disorders in Canada.

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Eric P. Hoffman, PhD

Dr. Hoffman is a resident of Halifax Nova Scotia, with current positions as CEO and Co-founder of AGADA Biosciences, CEO and Co-founder of ReveraGen Biopharm, and Professor of Pharmaceutical Sciences and Associate Dean for Research, Binghamton University – SUNY where he helped establish a new School of Pharmacy and Pharmaceutical Sciences (2016-present).

Dr. Hoffman received his PhD in Drosophila molecular genetics from Johns Hopkins University and transitioned to human molecular genetics as post-doctoral fellow with Louis Kunkel at Boston Children’s Hospital and Harvard Medical School working on the identification of the Duchenne muscular dystrophy gene and dystrophin protein. He has held faculty positions at Harvard Medical School (1988-90), University of Pittsburgh (1990-1998), George Washington University and Children’s National Medical Center (1998-2016). He founded the Cooperative International Neuromuscular Research Group (CINRG), is an inventor on over 20 patents and has authored over 500 publications.

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