OasisLMS

Title: 2026 Gene Therapy Bootcamp – Application in Neurodevelopmental Disorders

Translating Genes Into Health Series

Sponsored by The Education and Professional Development Committee

Tuesday, January 20 | 2:00pm-3:00pm ET

1.0 AMA PRA Category 1 Credits™, P.A.C.E.® - Educational Credits Claim by February 20

Explore the evolving landscape of gene therapy, including gene replacement, gene editing, and other nucleic-acid–based approaches. This session will provide a practical, clinically oriented update on the latest developments and their implications for medical genetics practice in 2026. Speakers will highlight applications in neurodevelopmental disorders and showcase the FDA-approved gene therapy ladocagene exuparvovec-tneq, while addressing key considerations such as underlying genetic mechanisms, dosage windows, preclinical modeling, clinical endpoints, biomarkers, and risk-benefit assessment.

Agenda

Target Audience: Clinical and laboratory geneticists, genetic counselors, researchers of all levels.

Learning Objectives

At the conclusion of this session, participants should be able to:

1. Summarize the current landscape of gene therapies for neurodevelopmental disorders
2. Describe the building blocks for therapeutic development for rare monogenic disorders including genetic mechanism, and preclinical, clinical and ethical considerations. 
3. Illustrate the concepts behind the application of gene therapy to a neurodevelopmental inborn error of metabolism

Planners

Henry J. Mroczkowski, MD, PhD, FACMG (Chair)

University of TN Health Science Ctr

Mari Mori, MD, MS, FACMG

The Ohio State University

Georgianne L. Arnold, MD, FACMG

University of Pittsburgh

Roya M. Mostafavi, MGC

University of Washington School of Medicine

Catherine A. Ziats, MD, FACMG

Cook Children's

Damara Ortiz, MD, FACMG

Children's Hospital of Pittsburgh of UPMC

Daniel Schecter, MD, MS

Mount Sinai School of Medicine

Staff

Jane Radford, MHA, CHCP

Director of Education 

Claudia Barnett

Senior Program Coordinator

	Moderator:  Mari Mori, MD, FACMG The Ohio State University
	Presenter: Maya Chopra, MD Harvard Medical School

Presenter: Toni Pearson, MD Nationwide Children’s Hospital

Accredited Continuing Education Information:

AMA PRA Category 1 Credit^TM, ASCLS P.A.C.E.^®

Accreditation

The American College of Medical Genetics and Genomics is accredited by the Accreditation Council for Continuing Medical Education (ACCME) to provide continuing medical education for physicians.

Credit Designation
The American College of Medical Genetics and Genomics designates this live activity for a maximum of 1.0 AMA PRA Category 1 Credits™. Physicians should claim only the credit commensurate with the extent of their participation in the activity.

Claiming your Educational Credits

Complete the activity and carefully complete the evaluation form. The deadline to claim educational credits is within 30 days from the date of the activity. Educational credit requests after this date will not be accepted.

Learner Data Consent

ACMG Education reports learner data to respective agency boards and you will be asked for consent during the evaluation process. Your compliance with deadlines and completing evaluations are part of the process in meeting learner needs and ACMG’s education mission.

Technical Support:
You can reach us by email at education@acmg.net or call 301-718-9603.
Support Center Hours: Monday – Friday, 9:00 AM – 5:00 PM Eastern Time.

Accredited Continuing Education Financial Disclosure

The American College of Medical Genetics and Genomics (ACMG) is accredited by the Accreditation Council for Continuing Medical Education (ACCME) to provide Accredited Continuing Education (ACE) for physicians. ACMG is an organization committed to improvement of patient care and general health by the incorporation of genetics and genomics into clinical practice.

ACMG has implemented the following procedures to ensure the independence of ACE activities from commercial influence/promotional bias, the Accreditation Council for Continuing Medical Education (ACCME) requires that providers (ACMG) must be able to demonstrate that: 1) everyone in a position to control the content of an ACE activity has disclosed all financial relationships that they have had in the past 24 months with ineligible* companies; 2) ACMG has implemented a mechanism to mitigate relevant financial relationships; and 3) all relevant financial relationships with ineligible companies are disclosed to the learners before the beginning of the educational activity. The learners must also be informed if no relevant financial relationships exist.
*Ineligible companies are defined as those whose primary business is producing, marketing, selling, re-selling, or distributing healthcare products used by or on patients.

ACMG Education Policies

Please review the policies below regarding the ACMG Education program

• Financial Disclosures and Mitigation Procedure
• ACMG Content Validation Policy
• ACMG Content Branding Policy

All of the relevant financial relationships listed for these individuals have been mitigated.

Disclaimer

ACMG educational programs are designed primarily as an educational tool for healthcare professionals who wish to increase their understanding of the application of genomic technologies to patient care. The ACMG does not endorse or recommend the use of this educational program to make patient diagnoses, particular by individuals not trained in medical genetics. Adherence to the information provided in these programs does not necessarily ensure a successful diagnostic outcome. The program should not be considered inclusive of all proper procedures and or exclusive of other procedures and that are reasonably directed at obtaining the same results. In determining the propriety of any specific procedure or, a healthcare professional should apply his or her own professional judgment to the specific clinical circumstances presented by the individual patient or specimen.

Questions regarding CE credit should be directed to education@acmg.net