A Deep Dive Into The Genetic Therapies Bootcamp At The ACMG 2026 Annual Meeting

The Genetic Therapies Bootcamp at the 2026 ACMG Annual Clinical Genetics meeting provided an overview of current advances in gene therapy, gene editing, and antisense oligonucleotide (ASO) therapeutics. Presentations reviewed the clinical rationale for gene replacement in loss-of-function disorders, key considerations for AAV-based delivery, and emerging approaches in base and prime editing. Liver-directed editing using lipid nanoparticles was discussed as a flexible platform for targeted genomic intervention, alongside a single-patient expanded access case illustrating translational and regulatory considerations.

The session also highlighted the growing application of ASOs and their distinct therapeutic advantages and limitations. Broader discussion focused on persistent challenges in rare disease clinical trials, including phenotypic heterogeneity, endpoint selection, and the ethical constraints of first-in-human study design. Across topics, speakers emphasized the increasingly important role of geneticists and genetic counselors in therapeutic development, implementation, and long-term patient management within the evolving field of therapeutic genomics.