Pioneering Progress for Rare Lives: Cell and Gene Therapy Trials in Pediatric Rare Disease Populations
Webinar Overview
Ground-breaking scientific advances have been made in the field of cell and gene therapy (CGT) in the past few decades, which is evidenced by an increasing number of CGT-based clinical trials.
While there are more than 8000 known rare diseases, treatment options are limited and only available for a few of them. As about 80 percent of rare diseases are monogenic diseases and many of them manifest during childhood, CGT is a potentially curative treatment for children and their families living with a rare life-altering disease.
For rare diseases presenting early in childhood and for which no adult disease exists due to rapid progression, young children and neonates will be increasingly involved in early CGT trials and even in first-in-human trials. There is also an urgent need in treating these children early as recent trials have shown that early treatment can improve outcomes.
These highly complex trials hold a myriad of challenges ranging from product development (choice of vector, determination of dose and administration of product) to conduct of the trial (ethical considerations, safety monitoring of potential toxicities and treatment of adverse events) and the long-term follow-up required by regulators.
This webinar aims to highlight some of the recent scientific developments relevant to studies in young pediatric patients with rare diseases. The expert speakers will further discuss some of the operational challenges that they have encountered so far when conducting these studies.
They will also focus on the patient voice and caregiver by exploring their attitudes towards CGT and discuss incorporating these insights into the training of pediatric patients on CGT. The expert speakers will be joined by a rare disease patient and his mother giving their very own perspectives on CGT trials and available CGT treatments.
Register for this webinar to gain insights on improving outcomes through early treatment and addressing regulatory requirements for pediatric rare diseases.
Learnings
- Some of the recent scientific advances in the field
- Obstacles, from ethical considerations to long-term follow-up, faced in CGT trials for neonates and young children with rare diseases
- The need to include the voice of the patients and families when planning and conducting these trials
- A practical example on the education of a pediatric population participating in a CGT trial
Speakers
Julie Maher is a Senior Director of Fortrea's Rare Diseases, Advanced Therapies and Pediatrics Team (RAPT). She has more than 29 years of clinical research experience, which includes 28 years of industry experience at Labcorp (now Fortrea). She has extensive project management expertise in late phase oncology as well as operational, strategy and planning expertise across complex Phase I-IV oncology, rare disease and pediatric trials.
Julie started her career in pediatrics at Sheffield Children's Hospital as a respiratory laboratory technician, where her duties included the study coordinator role for all pediatric asthma trials before she joined the industry side as a CRA for pediatric asthma trials. Her treatment experience includes immunotherapy, cell and gene therapy, vaccines and radiopharmaceuticals.
Message Presenter
Dr. Susanne Schmidt, MD, PhD, is a Senior Medical Director in the Rare Diseases, Advanced Therapies and Pediatrics Team (RAPT) at Fortrea. She is a board-certified pediatrician with 14+ years of clinical experience, including rare diseases. She joined the CRO industry more than 10 years ago and has supported numerous pediatric and rare disease projects, including gene therapy trials.
Message Presenter
Darby Thomas, PhD, is the Scientific Director of Fortrea's Rare Diseases, Advanced Therapies and Pediatrics Team (RAPT). She has more than 25 years of experience in virology, cell and molecular biology, gene and cell therapy and biotechnology. Her experience includes broad experience in adeno-associated virus (AAV) gene therapy with roles in CMC, discovery, translational development, and patient advocacy for several gene therapy companies with an emphasis on rare pediatric diseases and ophthalmology.
Message Presenter
Dr. Chris Evans is the CEO and co-founder of Little Journey, a health-tech company revolutionising paediatric healthcare and clinical trials with its patient experience and engagement platform.
With over 12 years of clinical experience as an anaesthetic doctor and researcher, Chris is currently pursuing a PhD focused on reducing pre-procedural anxiety and supporting families through paediatric medical care. His mission is to promote better health for all children through data-driven, personalised care and family-centered clinical trials.
Little Journey has recently partnered with the LEGO Foundation to create innovative solutions that support neurodiverse children, ensuring inclusive and accessible care for all. The platform is also used globally in pediatric clinical trials, serving as an integrated participant support system.
Message Presenter
A passionate advocate for patient-centric care and a mother of four, including a teenage son with sickle cell disease. Michelle's mission is to support parents of children with disabilities and emphasize the critical importance of self-care.
Learn more about Michelle’s story:
The Importance of Prioritizing Self-Care for Parents of Children with Disabilities | Jumo Health
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