How Peptides Could Transform Treatment of Genetic Disorders

This is a narrative review published in Drug Discovery Today that surveys the landscape of peptide-based approaches to genetic disorders. The authors examine the underlying biology of various genetic diseases, current peptide drugs and clinical candidates, and emerging applications of peptides as gene delivery vehicles and diagnostic tools.

Genetic disorders affect millions of people worldwide with limited treatment options — most therapies only manage symptoms. The peptide field has largely focused on metabolic, cardiovascular, and oncology indications, but this review highlights an underexplored frontier: using peptide versatility (as drugs, carriers, and diagnostics) to address genetic diseases. As gene therapy advances, peptide-based delivery systems could solve the critical 'last mile' delivery problem that has limited many gene therapies.

The intersection of peptide science and genetic medicine is an emerging frontier. As gene therapy becomes more practical, the delivery challenge — getting the corrective gene to the right cells — has become the primary bottleneck. Peptides offer a potential solution: cell-penetrating peptides, targeting peptides, and peptide-nucleic acid conjugates could overcome the delivery barriers that have limited gene therapy's reach. This review positions peptides as key enablers of the next generation of genetic treatments.

As a narrative review, it doesn't use systematic methodology or quantitatively assess evidence. The peptide-based genetic disorder space is relatively early-stage, so much of the review covers preclinical and conceptual work rather than proven therapies. The breadth of genetic disorders covered means depth on any single condition is limited.