Rewriting the Code: Advances and Challenges in Pediatric Gene Therapy

Pediatric gene therapy is rapidly reshaping what is possible for children born with rare genetic diseases. Join Dr. Rachel Abu Taleb, Medical Director, Medical Affairs at Veristat, for a look at the platforms driving clinical progress, including AAV vectors, lentiviral approaches, and CRISPR-based gene editing.

Through examples such as Luxturna, Zolgensma, Casgevy, and Lyfgenia, this webinar will explore what recent approvals have taught the field across retinal, neuromuscular, and hematologic diseases. Dr. Abu Taleb will also discuss the safety, regulatory, long-term follow-up, newborn screening, and access challenges that continue to shape pediatric gene therapy development and delivery.