Patient Access
Panel Discussion: Patient Access to CRISPR Medicine
Innovative CRISPR-based therapies hold tremendous promise for addressing a range of diseases, including rare disorders and diseases with unmet medical needs. However, ensuring equitable patient access remains a significant challenge. This one-hour panel discussion will examine key issues involved in the availability and accessibility of CRISPR-based medicines. By bringing together diverse stakeholders, the discussion aims to provide a comprehensive perspective on overcoming barriers to access and ensuring these transformative therapies reach those who need them most. Audience participation is highly encouraged.
Confirmed participants:
Session co-organiser and moderator: Daniël Warmerdam
Amsterdam UMC, Netherlands
Dr. Daniël Warmerdam is an Assistant Professor at the Emma Center for Personalized Medicine and the Department of Human Genetics at Amsterdam UMC. He specialises in molecular therapy development for rare neurometabolic diseases and leads efforts to create innovative therapeutic strategies for unmet medical needs, with a particular focus on gene-editing technologies and advanced cell-based models.
With a strong dedication to translational research, Dr. Warmerdam strives to bridge the gap between pre-clinical discoveries and clinical applications, ensuring that cutting-edge molecular advancements are translated into meaningful patient outcomes. His work places a strong emphasis on the availability, accessibility, and equity of advanced therapies, addressing the challenges these factors pose in the development of rare disease treatments.
Dr. Warmerdam’s background is rooted in molecular biology, where he focused on understanding cellular responses to DNA damage and establishing academic expertise centers for CRISPR technology. He also gained extensive experience working within government- and policy-related organisations involved in therapy development. In his current role, he aims to further advance the development of personalised molecular therapies for rare diseases.
Panelist: Alvin Luk
HuidaGene Therapeutics
Dr. Alvin Luk, Co-founder and CEO of HuidaGene Therapeutics, a clinical-stage company that is developing a broad pipeline of RNA- and DNA-editing therapeutics. He has more than 30 years of global drug development experience, specialising in rare diseases. He has held executive roles at multiple companies including Spark Therapeutics and Biogen, contributing to the approval of LUXTURNA®, the first AAV gene therapy. Dr. Luk has made over 250 regulatory submissions globally and served on the US-FDA Rare Disease Clinical Design Committee. He has contributed to 21 approved products and co-authored more than 100 scientific papers in prestigious journals. Dr. Luk holds an MBA from Harvard and a Ph.D. in Neuroscience from UC San Francisco Medical School.
Alvin Luk will also give an oral presentation as an invited speaker in the CRISPRMED25 in-person conference programme.
Panelist: Cecilia Jimenez-Mallebrera
Institut de Recerca Sant Joan de Déu. Hospital Sant Joan de Déu Barcelona and CIBERER
Cecilia Jimenez-Mallebrera, based at Barcelona's Sant Joan de Déu Pediatric Hospital, specialises in translational research in neuromuscular diseases. Her work integrates advanced therapies and biomarker discovery for paediatric neuromuscular conditions. Following a Biology degree from the University of Navarra and a PhD in Genetics from University College London, studying the utrophin gene, she completed postdoctoral work at the Dubowitz Neuromuscular Centre in London, focusing on congenital muscular dystrophies.
Since establishing her research group in 2010, she has pioneered CRISPR/Cas9 applications for muscular dystrophies and implemented serum GDF-15 as a biomarker for mitochondrial diseases. Her research group participates in various clinical trials for Duchenne Muscular Dystrophy, Spinal Muscular Atrophy, Pompe´s Disease and other muscle diseases and contributes to several research networks such as the EU ERDERA consortium and the COST Action on Gene Editing, GenE-HumDi, as well as the Spanish Biomedical Research Network on Rare Diseases, CIBERER.
Panelist: Jimi Olaghere
Patient Advocate
Jimi Olaghere is a CASGEVY recipient who has developed a keen interest in the advancements of cell and gene therapies, since his transformative participation in the groundbreaking clinical trial. Jimi is passionate about the positive impact that these therapies can have on patients in need and has become a staunch advocate for increased accessibility of gene therapies for sickle cell disease patients worldwide. At CRISPRMED25, Jimi will share his story about living with sickle cell disease, before and after treatment with the recently approved CRISPR therapy CASGEVY.
Follow Jimi on LinkedIn
Jimi Olaghere will also share his story in an oral presentation in the CRISPRMED25 in-person conference programme.
Panelist: Kevin Holden
Synthego
Kevin Holden is VP of Science at Synthego.
Panelist: Liselotte Wesley Andersen
Rare Diseases Denmark
Liselotte Wesley Andersen is the Acting Chair of Sjældne Diagnoser (Rare Diseases Denmark) since August 2024. She has been actively involved in the organisation for many years, serving as Vice Chair from 2009 to 2024 and as a volunteer for Sjældne Diagnosers Helpline since 2016. Additionally, she held leadership roles in the Danish Association for Tuberous Sclerosis from 1999 to 2018.
Liselotte holds a Cand. Scient. degree and a Ph.D. Her professional career includes positions as a Senior Researcher at the Institute for Ecoscience (2000-2021), a Research Assistant at Greenland Institute of Natural Resources (1998-2000), and a Postdoctoral Fellow at Aarhus University’s Department of Biology (1993-1994).