Running Paediatric Gene Therapy Studies: Operations and Oversight in Practice

Gene therapy is changing what is possible in paediatric rare disease, but the distance between a sound protocol and a study that delivers for patients is closed through execution. Patient populations are small and scattered, product handling allows no margin for error, dosing frequently happens once with no chance to correct a logistical failure and follow-up then runs for years in children who are still growing. The standard clinical trial playbook was never built for any of it.

This webinar provides a comprehensive overview of practical considerations for running paediatric gene therapy studies.

Solomon Kamal-Uddin, Medical Advisor, and Lakshmi Guduri, Senior Director, Project Management at TFS HealthScience, have supported these programmes from start-up through long-term follow-up—Solomon from medical oversight and safety, and Lakshmi from operational delivery. Much of what they have learned sits where those two responsibilities meet.

Join them in this webinar as they share best practices across:

• Study start-up and execution
• Site readiness
• Patient journey pathway management
• Safety oversight and medical monitoring
• Cross-functional collaboration
• Long-term follow-up

The session will focus on the decisions that optimise recruitment and study conduct while protecting patient safety and regulatory compliance, including what could be approached differently in future studies.

Register for this webinar to learn how Paediatric Gene Therapy studies can improve operational execution, safety oversight and long-term follow-up.

Speakers

Solomon Kamal-Uddin, Medical Advisor, TFS HealthScience

Solomon Kamal-Uddin is a board certified Paediatric and Paediatric Emergency Medicine Consultant with more than 15 years of clinical experience and seven years in the life sciences industry. His work spans paediatrics, rare disease, neuroscience, cardiology, immunology, oncology, dermatology and endocrinology, including medical support on over 100 clinical trials and participation as a Principal Investigator and across numerous Delphi studies.

Solomon has advised across the drug lifecycle, from preclinical through post marketing, with a global footprint that includes direct engagement with regulatory authorities and state healthcare buyers. His cross-sector background spans clinical practice, CRO consultancy and biotech medical affairs, including therapies for lysosomal storage disorders and real-world evidence generation for large pharmaceutical companies. He also serves on the board of directors for the rare disease charity Usher Syndrome Ireland, an active disease area of activity for potential gene therapies.

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Lakshmi Guduri, Senior Director of Project Management, TFS HealthScience

Lakshmi Guduri is a clinical research leader with 18 years of experience across the pharma, biotech and CRO sectors, including more than a decade of global leadership in clinical project and program management. Lakshmi works closely with cross-functional teams to operationalize CDPs, shape protocol execution strategies and lead end-to-end delivery of complex multinational clinical programs across North America and Europe. Expertise spans strategic planning, governance, portfolio oversight, resource forecasting, budget management, risk mitigation, quality oversight, inspection readiness and operational excellence throughout the clinical development lifecycle.

Lakshmi’s therapeutic expertise spans across numerous therapeutic areas, including ATMPs and cell and gene therapies, including AAV-based gene therapies and tissue-engineered regenerative medicine, alongside rare paediatric, neuromuscular and neurodegenerative disorders, immunology and chronic inflammatory disease, dermatology, cardiovascular and metabolic disorders and endocrinology. Lakshmi works cross-functionally to shape protocol execution strategy and leads multinational programs across North America and Europe.

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