World-First Personalized CRISPR Gene Therapy Treats Rare Disease

CRISPR gene-editing technology has reached a historic milestone with the first successful personalised gene-editing treatment of a baby named KJ, who was born with a rare genetic disorder, CPS1 deficiency. This pioneering therapy, developed by researchers at the University of Pennsylvania and the Children's Hospital of Philadelphia, customizes gene editing to correct individual mutations, potentially opening pathways for treating other rare diseases. While the clinical promise of CRISPR continues, the gene-editing field faces uncertainties stemming from evolving technological, regulatory, and leadership challenges, as well as ongoing mergers and acquisitions in the biotech sector. Legal battles have also intensified, with recent Federal Circuit rulings reopening priority disputes over CRISPR-Cas9 patents and emphasizing the need for rigorous patent documentation. Concurrently, next-generation gene-editing tools such as base and prime editors, evoCAST, and bridge recombinases are advancing clinical applications and expanding the competitive intellectual property landscape. Meanwhile, CRISPR Therapeutics, a key player in the field, shows mixed financial outlooks with recent downward revisions in earnings estimates, reflecting market and industry pressures.