UK Baby Receives First-Ever Personalized Gene Therapy

A baby in the UK has become the first person in the world to receive a personalized gene therapy created specifically for her rare condition. Eighteen-month-old Teddi Shaw, from Northumberland, was diagnosed with a rare genetic disorder called metachromatic leukodystrophy (MLD), which typically causes loss of motor and cognitive functions and is often fatal.

Scientists at Great Ormond Street Hospital (GOSH) designed a unique treatment called “otoferlin gene therapy” tailored specifically to Teddi’s DNA mutation. The therapy, developed in under a year, involved modifying a virus to deliver a healthy copy of the gene into her cells.

Doctors say the results are promising, with Teddi now showing signs of improved health and development. Her family expressed hope that this breakthrough could lead to similar treatments for other children with rare diseases.

Experts hailed the therapy as a major milestone in personalized medicine, though they acknowledged the challenges of cost, regulation, and access for future patients.