New Frontiers in Gene Therapy: From the Lab to Cures for Kids

Saturday, April 26, 2025

10:00am – 11:30am HST

Location: Convention Center: 318 B

CE: 1.5

Selected By: SPR
Co-sponsored By: PIDS, CHTRC

Chair(s)

Ann Chahroudi, MD, PhD (she/her/hers)

Professor of Pediatrics
Emory University School of Medicine / Children's Healthcare of Atlanta, United States
Katherine Luzuriaga, MD (she/her/hers)

Professor, Vice Provost Research
UMass Chan Medical School, United States

Disclosure(s):

Ann Chahroudi, MD, PhD: ViiV Healthcare, Inc (Ongoing) (Products/Services: Advisory Committee)

Katherine Luzuriaga, MD: Moderna (Ongoing) (Products/Services: Research Support including clinical trials & the principal or named investigator); Pfizer (Terminated, November 15, 2023) (Products/Services: Research Support including clinical trials & the principal or named investigator); Sanofi (Terminated, July 12, 2024) (Products/Services: Consultant)

Gene therapy is transforming the landscape of pediatric medicine, offering hope for patients with devastating diseases. This session will explore cutting-edge advances in gene-based treatments for childhood diseases. Bringing together leading experts in the field, the session will focus on FDA-approved therapies as well as innovative clinical and preclinical research.

The session will include an overview from the Chairs, followed by three presentations focused on specific disorders (sickle cell disease, cystic fibrosis, and HIV). Each presentation will cover the latest scientific breakthroughs, detailing the translational steps from laboratory research to clinical trials, and ultimately, to treatment of pediatric patients. With a focus on state-of-the-art technologies, including viral vector-based therapies and gene editing techniques, this session will offer a comprehensive overview of the progress being made across multiple disease areas.

By addressing a diverse range of diseases, this session is designed to appeal to a broad audience, from basic scientists and clinical researchers to pediatricians and subspecialists seeking insight into future therapeutic strategies. Attendees will gain an understanding of the complex challenges of gene therapy, as well as the exciting potential for curative treatments in childhood diseases.

Engaging and forward-looking, this session will not only provide an overview of the current state of the field but will also inspire discussion about the next frontiers in pediatric gene therapy. Topics such as access, cost and ethics will be discussed.

Learning Objectives:

Explain recent advancements in gene therapy technologies and their application in developing curative treatments for childhood diseases.
Describe and compare the mechanisms of gene therapy approaches for sickle cell disease, cystic fibrosis, and HIV infection.
Discuss the challenges and successes in translating gene therapy from research into clinical practice, and its potential impact on pediatric patient outcomes.

Presentations:

10:00am – 10:05am HST

Introduction to Session
Speaker: Ann Chahroudi, MD, PhD (she/her/hers) – Emory University School of Medicine / Children's Healthcare of Atlanta
10:05am – 10:15am HST

Introduction of Topic & Background
Speaker: Katherine Luzuriaga, MD (she/her/hers) – UMass Chan Medical School
10:15am – 10:25am HST

Overview of Fda-approved Gene Therapies in Pediatrics
Speaker: Katherine Luzuriaga, MD (she/her/hers) – UMass Chan Medical School
10:25am – 10:45am HST

Gene Therapy for Sickle Cell Disease
Speaker: Vivien A. Sheehan, MD, PhD (she/her/hers) – Emory University School of Medicine
10:45am – 11:05am HST

Gene Therapy for Cystic Fibrosis
Speaker: Matthew H. Porteus, MD, PhD (he/him/his) – Stanford University School of Medicine
11:05am – 11:25am HST

Gene Therapy for Pediatric HIV Cure
Speaker: Mauricio Martins, PhD (he/him/his) – University of Florida
11:25am – 11:30am HST

Q & A