Genetic Therapies Bootcamp | Medical Genetics in Ophthalmology | Joint Keynote
Date of Release: March 23, 2026
Expiration Date: March 31, 2028
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: 7.5 hours
Maximize your learning — and your savings. When you purchase our two featured courses together as a bundle, you’ll receive 7.5 hours of accredited CME credit and enjoy a reduced package price.
Instead of enrolling in each course separately, bundle them today to:
- Save on registration fees
- Gain comprehensive insights across both courses
Course 1: 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.
Agenda
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Maya Chopra and Joan Stoler
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Welcome and Introduction
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Tim Yu and Austin Larsen
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Overview of Genetic Therapies (30 min talk + 10 min Q&A)
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Toni Pearson
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Gene Replacement Therapy (25 min talk + 5 min Q&A)
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Rebecca Ahrens
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Gene Editing: CPS1 Deficiency (25 min talk + 5 min Q&A)
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Tim Yu
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Antisense Oligonucleotides (ASOs) (25 min talk + 5 min Q&A)
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Liz Berry Kravis
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Clinical Trials Training (25 min talk + 5 min Q&A)
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PJ Brooks
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Academic/Government/Industry Relationships (25 min talk + 5 min Q&A)
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Panelist
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Panel Discussion with Speakers and Moderators
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Course 2: Medical Genetics in Ophthalmology: Through the Lens of Genetics-Informed Therapies
Description
Ophthalmic genetics is a well-established field for management, diagnosis, and counseling of rare genetic eye disorders and genetic systemic disorders with eye manifestations in children and adults. Multiple clinical models and training pathways are available for medical geneticists, ophthalmologists and genetic counselors interested in this career specialty. This workshop aims to provide a description of the clinical landscape of ophthalmic genetics, unique diagnostic and therapeutic challenges, including highlighting the growing field of gene-targeted and gene-agnostic therapies. Participants will gain insight into training, clinical phenotyping, including advanced multimodal imaging, genetic testing, and gene therapy considerations, as well as the opportunity to interact with leaders in ophthalmic genetics clinical and translational research. We will provide an open forum for clinical geneticists and ophthalmologists to present cases which remain unsolved or represent an example of the power of ocular genetics.
Target Audience
ACMG members, medical geneticists, laboratory geneticists, genetic counselors, ophthalmologists with experience in genetics practice, and ophthalmologists with interest in genetics training.
Learning Objectives
At the conclusion of this session, participants should be able to:
- Define key ophthalmic terms used in genetic diagnosis.
- Distinguish between common congenital ocular anomalies and their genetic implications.
- Recognize how precise terminology improves communication between medical geneticists and ophthalmologists.
- Describe current training pathways integrating genetics and ophthalmology.
- Compare educational approaches for ophthalmologists, medical geneticists, and genetic counselors.
- Identify strategies to develop genetics-focused training within ophthalmology programs.
- Explain the essential components of a multidisciplinary ophthalmic genetics clinic.
- Differentiate between genetic testing modalities used in ocular disease diagnosis.
- Evaluate approaches for selecting appropriate clinical, research, or sponsored testing.
- Summarize current and emerging gene therapy options for ocular genetic disease.
- Discuss gene-directed and gene-agnostic therapeutic approaches.
- Examine the patient experience and ethical considerations surrounding ocular gene therapy.
Agenda
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Topic / Session
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Opening Remarks & Session 1 — The Importance of Proper Eye Terminology in Genetic Diagnosis
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Session 2 — Pathways for Genetics Training and Education in Ophthalmology
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Session 3- The Role of Multidisciplinary Clinics in the Gene Therapy Era
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Session 4 - Therapeutic Options for Ocular Genetic Disease and the Medical Geneticist
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Joint Closing Address: The Evolution of Gene Therapy — Milestones, Challenges, and Future Directions:
Presenter: Eric Pierce, MD, PhD
This on-demand joint closing session is the recorded presentation from the live event and serves as the last session where attendees from both the Gene Therapy Bootcamp and the Ophthalmology Symposium come together. The session explores the evolution of gene therapy, highlighting key milestones, ongoing challenges, and future directions in the field.