Genetic Therapies Bootcamp: Foundations, Frontiers, and Clinical Engagement OnDemand-03-10 16-15 318-323 Session 4 Panel Discussion Panel with Speakers and Moderators

Video Transcription

Video Summary

The session focused on the emerging role of clinical geneticists and genetic counselors in providing and guiding approved genetic therapies, including gene therapy and antisense oligonucleotides, especially for rare disorders. Audience polling revealed strong support for clinical geneticists taking leadership or supportive roles in delivering these therapies, emphasizing shared decision-making and risk evaluation for clinical trial enrollment. The importance of clinical trial training in genetic residencies was highlighted as a necessary component for future workforce development.<br /><br />Panel discussions addressed ethical considerations such as equitable access, safety, and benefit, acknowledging challenges in therapy implementation across different states and institutions. Collaboration was stressed across prenatal, newborn, and pediatric genetic communities to optimize timing and delivery of therapies, recognizing opportunities for in utero treatments.<br /><br />Key barriers include the need for nationwide guidelines, funding, institutional support, and standardized training pathways. Transparency in regulatory processes and data sharing were emphasized to improve approval and reimbursement, thereby enhancing equitable access. Programs like ARPA-H and the Bespoke Gene Therapy Consortium aim to address manufacturing bottlenecks and regulatory challenges.<br /><br />Finally, ethical concerns were raised about the potential for premature or inappropriate therapy delivery, underscoring the necessity for clinical geneticists to guide patient-centered decisions based on natural history and therapeutic benefit. The session concluded with a call for ongoing collaboration, education, and system-level support to realize the potential of genomic medicine.

Keywords

clinical geneticists

genetic counselors

gene therapy

antisense oligonucleotides

rare disorders

clinical trial training

ethical considerations

equitable access