One Infant, One Breakthrough: CRISPR Enters a New Phase

Acuitas, CHOP, and Penn pioneer safer, patient-tailored base editing through a landmark CRISPR collaboration

28 Oct 2025

In February 2025 an infant with a rare metabolic disorder became the first patient to receive a custom-made base-editing treatment. Behind the experiment stands an unusual alliance between Acuitas Therapeutics, the Children’s Hospital of Philadelphia (CHOP), and the University of Pennsylvania. Their collaboration, revealed in May, marks a cautious step toward turning the promise of genetic editing into practical medicine.

The science is dazzling but fragile. CRISPR can rewrite DNA with great accuracy, but only if its molecular scissors reach the right cells. Delivery is the sticking point. Acuitas, known for the lipid nanoparticles that carried mRNA vaccines, is providing its proprietary LNP technology as an in-kind contribution. These microscopic carriers now shuttle base-editing machinery directly to diseased tissue.

The treatment, designed for a single patient or "N = 1", embodies both ambition and restraint. It shows how far personalized medicine has advanced while reminding scientists that each success still amounts to a case study. "This collaboration shows how precision delivery systems can transform treatment for patients with genetic diseases," said Thomas Madden, Acuitas’s chief executive. Observers see the partnership as proof of Acuitas’s move from supplier to scientific partner, and of CHOP and Penn’s continuing strength in translating laboratory work into clinical reality.

Challenges abound. Regulators will scrutinise safety claims, and scaling production for future trials will test both budgets and technology. Yet optimism endures. Rivals such as Intellia and Beam are pursuing similar delivery methods, suggesting that collaboration, rather than competition, is propelling the field forward.

Should this first treatment succeed, it could inaugurate a new era in base editing, one in which genetic cures are not just conceived in laboratories but tailored, patient by patient, at the bedside.