OasisLMS

Date of Release: March 23, 2026

Expiration Date: March 31, 2028

Credits offered: AMA PRA Category 1 Credits™, ASCLS P.A.C.E.®, NSGC (Pending)

Estimate time of completion: 4.25 hours

Genetic Therapies Bootcamp: Foundations, Frontiers, and Clinical Engagement

Description

Explore the transformative world of gene therapy at your own pace with this comprehensive on-demand course, designed for clinical geneticists, laboratory geneticists, and genetic counselors. Gain an insightful overview of both FDA-approved and emerging gene therapies across a variety of conditions.

Learn from leading experts about the principles of clinical trials, discover the toolkit of cutting-edge therapeutic strategies, and understand the collaborative relationships shaping the field. Delve into discussions on ethical considerations and acquire practical knowledge to enhance patient care and advance your professional growth in the rapidly evolving landscape of gene therapy.

Target Audience

Clinical and Laboratory geneticists, trainees, genetic counselors.

Learning Objectives

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

• Evaluate the current state of gene therapy for rare disorder, including successes, challenges, future directions, citing recent case studies.
• Explain the principles of clinical trial design as applicable to rare disorders, including phases, regulatory considerations, n=1 studies and good clinical practice.
• Describe the different therapeutic strategies in gene therapy for monogenic disorders, including gene replacement, antisense oligonucleotides (ASO), and gene editing.
• Apply knowledge of gene therapy therapeutic strategies and clinical trials to inform their own clinical practice, research endeavors and career pathway opportunities.
• Explore the potential future role of medical geneticists in the therapeutic era, including in clinical trials and in the long-term management of patients treated with gene therapy.
• Recognize ethical considerations in genetic therapies, including consent, access and long-term impacts.
• Compare limitations, risks and benefits of different genetic therapy approaches.
• Analyze interdisciplinary collaborations among academia, industry and healthcare teams
• Apply case studies to illustrate the diversity of approaches and outcomes in genetic therapy applications
• Recognize opportunities for future ongoing education in genetic therapies for professional development.

Moderators

	Maya Chopra, MBBS, FRACP Assistant Professor, Boston Children's Hospital
	Joan Stoler, MD, FACMG Associate Professor, Boston Children’s Hospital /Harvard Medical School

Presenters

	Austin Larson, MD Associate Professor, University of Colorado
	Elizabeth Berry-Kravis, MD, PhD Professor of Pediatrics and Neurological Sciences, Rush University Medical Center
	PJ Brooks  Acting Director of NCATS’ Division of Rare Diseases Research Innovation
	Rebecca Ahrens-Nicklas, MD, PhD, FACMG Assistant Professor, The Children’s Hospital of Philadelphia
	Timothy Yu, MD, PhD Neurologist, Havard Medical School
	Toni Pearson, MD Neurologist, Nationwide Children’s Hospital

Accredited Continuing Education Information:

CME AMA PRA Category 1 Credits^TM, ASCLS P.A.C.E.®, NSGC (Pending)

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 enduring activity for a maximum of 4.5 AMA PRA Category 1 Credits™. Physicians should claim only the credit commensurate with the extent of their participation in the activity.

The American Medical Association (AMA) defines physicians as those individuals who have obtained an MD, DO, or equivalent medical degree from another country. Non-physicians may request a certificate of attendance for their participation.

ASCLS P.A.C.E.® CEU's 

ACMG is approved as a provider of continuing education programs in the clinical laboratory sciences by the American Society for Clinical Laboratory Science (ASCLS) Professional Acknowledgment for Continuing Education (P.A.C.E.®) Program.

ACMG is approved by the Florida Board of Clinical Laboratory Personnel as CE Provider (50-11878). This course is registered with CEBroker #20-1332029. ACMG is approved by the California Department of Health Services through the ASCLS P.A.C.E.® (854327). This activity has been approved for 0.45 P.A.C.E.® CEU's.

National Society of Genetic Counselors (NSGC):

This event has been submitted to the National Society of Genetic Counselors (NSGC) for approval of Category 1 CEUs. The American Board of Genetic Counseling (ABGC) accepts CEUs approved by NSGC for purposes of recertification. Approval for the requested CEUs and contact hours is currently pending.

Claiming your Educational Credits

This activity consists of: View content, take a post-test, the test may be taken as often as necessary to achieve a passing score of 80% or better is required to receive credit.  If you do not achieve a passing score, the program will identify which questions you answered incorrectly so that you can review the module and try again. Complete the overall evaluation form.

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.

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

Financial Disclosures

Faculty

Maya Chopra – Moderator
No Relevant Financial Relationships

Joan Stoler – Moderator
Consultant: Ultragenyx Pharmaceuticals
Research: Zevra Therapeutics

Timothy Yu – CE Presenter
Research: EveryONE Medicines

Austin Larson – CE Presenter
Consultant: UCB Pharma, Stealth Biotherapeutics, Tisento, Saol, Abliva, Pretzel

Toni Pearson – CE Presenter
No Relevant Financial Relationships

Rebecca Ahrens-Nicklas – CE Presenter
Scientific Medical Advisor: LatusBio
Consultant: AskBio

Liz Berry-Kravis – CE Presenter
Grant/Research Support: Tetra Therapeutics, Zynerba, Taysha Gene Therapies, Ultragenyx, Roche, Ionis, Zevra, Neuren, BioMarin
Scientific Medical Advisor: Roche, Zevra, Neurogene, Neuren, Novartis, Encoded, Ionis

PJ Brooks – CE Presenter
No Relevant Financial Relationships

Program Committee and Planning Staff

Ross A. Rowsey, PhD, FACMG – Chair, Program Committee
No Relevant Financial Relationship

Nathan D. Kopp, PhD – Vice Chair, Program Committee
No Relevant Financial Relationship

Jodi D. Hoffman, MD, FACMG – Immediate Past Chair, Program Committee
No Relevant Financial Relationship

Shweta Dhar, MD, MS, FACMG – VP Education, Program Committee
No Relevant Financial Relationship

Mira Irons, MD, FACMG – ACMG President, Program Committee
No Relevant Financial Relationship

Kevin M. Bowling, PhD, MS – Member, Program Committee
No Relevant Financial Relationship

Lauren B. Carter, MD, FACMG – Education and PD Committee Liaison
No Relevant Financial Relationship

Matthew Deardorff, MD, PhD, FACMG – Member, Program Committee
No Relevant Financial Relationship

Rajarshi Ghosh, PhD, MS, FACMG – Member, Program Committee
No Relevant Financial Relationship

Anna C. Hurst, MD, MS, FACMG – Member, Program Committee
No Relevant Financial Relationship

Jaeseung Kim, PhD, MS – Member, Program Committee
No Relevant Financial Relationship

Alaa Koleilat, PhD, MS, FACMG – Member, Program Committee
No Relevant Financial Relationship

Murugu Manickam, MD – Member, Program Committee
No Relevant Financial Relationship

Shivarajan Manickavasagam Amudhavalli, MBBS, MD, FACMG – Member, Program Committee
No Relevant Financial Relationship

Teri A. Manolio, MD, PhD – Member, Program Committee
No Relevant Financial Relationship

Andres A. Morales Corado, MD, FACMG – Therapeutics Committee Liaison
Research Grant/Contract: Phoenix-Nest, Inc.

Anna Nagy, MGC – Member, Program Committee
No Relevant Financial Relationship

Chanika Phornphutkul, MD, FACMG – Member, Program Committee
No Relevant Financial Relationship

Andrea J. Schelhaas, MS – Former Education and PD Committee Liaison
Consultant: BioMarin Pharmaceutical Inc., Tyra Biosciences

Panieh Terraf, PhD, FACMG – Member, Program Committee
No Relevant Financial Relationship

Katarzyna Thompson, PhD, FACMG – Member, Program Committee
No Relevant Financial Relationship

Neeta L. Vora, MD, FACMG – Member, Program Committee
No Relevant Financial Relationship

Jeffrey Nelson Weitzel, MD, FACMG – Member, Program Committee
Consultant: Cancer IQ; Natera; MyOme

Xinming Zhuo, PhD – Trainee Member, Program Committee
Stock: FUTU Holdings Limited; GE Healthcare; Gilead Sciences, Inc.

Jane Radford, MHA, CHCP – Staff
No Relevant Financial Relationship