AUGUST 2024LIFE SCIENCES REVIEW 19CXO INSIGHTSTHE RUB: MEDICAL AFFAIRS IS FAR MORE THAN A COMMODITY IN RARE AND GENETIC DISEASEe are at the cusp of a new era in therapeutics. Rare and genetic diseases are providing targets for therapeutic intervention and increasingly yield life-changing results for patients and their families. Our tools of intervention are comprised of a spectrum of therapeutics, including oral small molecules, injectables, anti-sense oligonucleotides, gene therapies including CRISPR-Cas-9 and Prime Editing, enzyme replacement, and a host of novel delivery approaches to break barriers to delivery of the investigational product to the target site, including the eye, crossing the blood-brain barrier and beyond.We are at the dawn of a wellspring of spectacular, promising interventions. Meaningful work is happening broadly in neurologic disease, hearing loss, blindness, cancer, heart disease, kidney disease and beyond. By Henry David Cremisi, MD FACP, Executive Medical Director, Medical Affairs, CaidyaW
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