Understanding Cell and Gene Therapy Trials for Pediatric Rare Diseases: Join the Upcoming Xtalks Webinar
Cell and gene therapy (CGT) has emerged as a promising treatment for rare diseases, particularly for pediatric populations where few treatment options exist. The increasing number of clinical trials utilizing CGT for rare diseases presents significant breakthroughs in medical science. However, these trials come with unique challenges—especially when the trials involve neonates and young children who suffer from rare, life-altering conditions. To address these challenges, Xtalks is hosting an informative, free webinar on Cell and Gene Therapy Trials in Pediatric Rare Disease Populations, scheduled for Monday, February 24, 2025, at 10 am EST (4 pm CET).
The webinar will explore the recent scientific advances in CGT, offering insights from leading experts in the field of rare disease drug development. Attendees will learn about the hurdles encountered when conducting clinical trials for rare pediatric diseases, including complex ethical considerations, safety monitoring, and long-term follow-up. The speakers will highlight the importance of incorporating the voices of patients and families into trial planning and execution. Additionally, a real-world example will be shared on how to educate pediatric patients participating in CGT trials, emphasizing how these trials can lead to potentially curative treatments.
Addressing the Need for Pediatric Rare Disease Treatments
Rare diseases, while often overlooked, affect millions of people worldwide. With over 8,000 identified rare diseases, most of which are genetic, treatment options remain limited. Of these, approximately 80% are monogenic diseases, where a single genetic mutation causes the disease. Many of these rare diseases manifest during childhood, making them particularly devastating for families. For these children, CGT presents a potential curative treatment option, offering hope for a better future.
Since many rare diseases in children are progressive and do not have adult counterparts, there is an urgent need for early intervention. Recent clinical trials have shown that early treatment in these diseases significantly improves outcomes, highlighting the importance of conducting CGT trials for neonates and young children. In fact, many of these children are involved in first-in-human trials, making it all the more critical to address the scientific, operational, and ethical challenges unique to these studies.
Challenges of Conducting CGT Trials in Pediatric Populations
Cell and gene therapy trials for pediatric rare diseases present a myriad of challenges. These trials involve high levels of complexity in both product development and trial execution. Key challenges in product development include selecting the right viral vector, determining the appropriate dose, and ensuring the safe administration of CGT products. Additionally, there is a need to account for the unique safety monitoring required for neonates and children, as their bodies may react differently to treatments compared to adults.
The ethical considerations surrounding pediatric CGT trials are especially delicate. Children cannot provide informed consent themselves, so parental consent and the child’s assent (when appropriate) are critical components of these trials. Furthermore, long-term follow-up is mandated by regulators to monitor potential long-term adverse effects, which can pose significant logistical challenges for both clinical trial teams and families.
Incorporating the Patient and Family Voice into Trial Planning
One of the most important aspects of conducting CGT trials in pediatric populations is understanding the perspectives of the patients and their families. The webinar will discuss how to incorporate the patient and family voice into trial design and execution. Listening to the attitudes and concerns of caregivers is essential to ensure the trials are patient-centered, focusing on both the scientific outcomes and the emotional well-being of the families involved.
The expert speakers will also share insights on educating pediatric patients participating in CGT trials. Effective communication strategies are crucial in helping children understand the treatment process, even at a young age, while also managing their expectations and addressing their fears. By focusing on both scientific education and emotional support, these trials can be more effective in achieving positive outcomes for children and their families.

Real-World Insights from a Rare Disease Patient and Family
In addition to expert speakers, the webinar will feature a rare disease patient and their mother, who will share their personal experience navigating CGT trials. Their perspectives will provide valuable insight into the realities of living with a rare disease, the challenges of participating in clinical trials, and the hope CGT brings to families.
This personal story will help the audience better understand the emotional and psychological aspects of rare disease trials and highlight the importance of family-centered care in the clinical trial process. By hearing from real patients and their families, attendees can gain a deeper appreciation for the human side of CGT research.
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Webinar Speakers and Key Takeaways
The expert panel of speakers includes:
- Julie Maher, Senior Director of Rare Diseases, Advanced Therapies, and Pediatrics Team (RAPT) at Fortrea, who will moderate the session.
- Dr. Susanne Schmidt, Senior Medical Director at Fortrea, who will provide insights into the medical aspects of rare disease drug development.
- Darby Thomas, PhD, Scientific Director at Fortrea, who will delve into the scientific advancements in CGT.
- Dr. Chris Evans, MD, Founder & CEO of Little Journey, who will share practical examples of educating pediatric populations on CGT.
- Michelle Burgess and Michael, rare disease patients and advocates, who will provide personal insights into their experience with CGT trials.
This informative session will cover:
- The latest scientific breakthroughs in CGT for pediatric rare diseases.
- Ethical considerations, including patient consent and the role of families in clinical trials.
- Key operational challenges in conducting CGT trials for young children.
- How early treatment can significantly improve outcomes for pediatric rare disease patients.
- The importance of integrating the patient and family voice into trial planning and execution.
Register for the Webinar
This upcoming Xtalks webinar is an invaluable opportunity for medical professionals, researchers, caregivers, and anyone interested in the future of cell and gene therapy for pediatric rare diseases. By attending, you will gain critical insights into overcoming the unique challenges of CGT trials and contribute to improving outcomes for children with rare, life-threatening conditions.
To register for the live webinar on Monday, February 24, 2025, at 10 am EST (4 pm CET), click here and join the discussion on advancing CGT trials and treatment options for pediatric rare diseases.
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