Selvine Richter

Gene Editing: The Future of Genomic Medicine & Biotech Investing

Salveen Richter, Allison Nathan, Selvine Richter

Gene editing distinguishes itself from traditional gene therapy by utilizing molecular scissors like CRISPR-Cas9 to permanently correct genetic defects in liver, eye, and neurological applications, with recent ex vivo successes for sickle cell disease and the first in vivo proof-of-concept for TTR. As two CRISPR-based therapies approach regulatory filings in 2022 and pivotal studies advance for rare diseases, the industry faces rigorous FDA safety mandates requiring fifteen years of follow-up data alongside complex reimbursement challenges stemming from the high one-time cost of curative treatments. While significant venture capital and pharmaceutical acquisitions drive the integration of next-generation tools like base and prime editing, the field maintains a strict consensus against germline modifications to mitigate ethical concerns and long-term safety risks.