Rewriting The Code: Advances And Challenges In Pediatric Gene Therapy

In this webinar, Dr. Rachel Abu Taleb examines the three platforms now driving clinical progress for pediatric gene therapy: AAV vectors for in vivo gene replacement, lentiviral vectors for ex vivo hematopoietic stem cell modification, and CRISPR-based gene editing for precise correction without viral integration. The session concludes with an honest look at the current challenges and opportunities, including platform-specific safety considerations, evolving regulatory pathways, long-term follow-up requirements, and the real-world barriers that still prevent many children from accessing approved therapies. As the field shifts from proving efficacy to optimizing treatment timing and delivery, pediatric gene therapy continues to redefine what is possible for children and families living with rare genetic diseases.