Genetic Therapies Bootcamp: Foundations, Frontiers, and Clinical Engagement OnDemand
Availability
On-Demand
Release on Mar 23, 2026 12:00 AM Central Daylight Time
Expires on Mar 31, 2028
Cost
ACMG Member: $150.00
Non-Member: $200.00
Postdoc/Trainee (M): $125.00
Postdoc/Trainee (NM): $170.00
Student (M): $100.00
Student (NM): $135.00
Credit Offered
3.5 CME (AMA) Credits
3.5 CME (Other) Credits

 

 

Date of Release: March 23, 2026

Expiration Date: March 31, 2028

ASCLS P.A.C.E.® Expiration Date: March 31, 2027

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

Estimate time of completion: 3.5 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.

Category

Member Fee

Non-Member Fee

Professional

$150

$200

Postdoc/Trainee

$125

$170

Student

$100

$135

 

 

Maya Chopra and Joan Stoler

Welcome and Introduction

Tim Yu and Austin Larsen

Overview of Genetic Therapies (30 min talk + 10 min Q&A)

Toni Pearson

Gene Replacement Therapy (25 min talk + 5 min Q&A)

Rebecca Ahrens

Gene Editing: CPS1 Deficiency (25 min talk + 5 min Q&A)

Tim Yu

Antisense Oligonucleotides (ASOs) (25 min talk + 5 min Q&A)

Liz Berry Kravis

Clinical Trials Training (25 min talk + 5 min Q&A)

PJ Brooks

Academic/Government/Industry Relationships (25 min talk + 5 min Q&A)

Panelist

Panel Discussion with Speakers and Moderators

 

Moderators

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Maya Chopra, MBBS, FRACP

Assistant Professor, Boston Children's Hospital

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Joan Stoler, MD, FACMG

Associate Professor, Boston Children’s Hospital /Harvard Medical School


Presenters

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Austin Larson, MD

Associate Professor, University of Colorado

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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 CreditsTM, 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 3.5 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.® (275-100-26). This activity has been approved for 0.35 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 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.

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