A new gene editing therapy for transthyretin amyloidosis (ATTR) has shown safety and effectiveness in a Phase I clinical trial conducted by UCL and the Royal Free Hospital.

This novel therapy employs CRISPR-Cas9 technology to inactivate a gene in the liver that produces transthyretin, significantly reducing its production.

Professor Julian Gillmore highlighted that the trial successfully demonstrated the selective and permanent inactivation of a gene in humans, paving the way for potential treatments for various diseases.

Most participants in the trial reported stabilization or improvement of their condition after 12 months, despite starting with advanced heart failure symptoms.

Current treatment options for ATTR primarily focus on managing symptoms and slowing disease progression, with common symptoms including breathlessness, fatigue, and dizziness.

Professor Marianna Fontana emphasized the therapy's potential to preserve quality of life for early-stage patients who do not require ongoing treatment.

To further validate the treatment's effectiveness, Professors Fontana and Gillmore are conducting a larger Phase III clinical trial involving over 700 patients.

The promising results from the trial were published in the New England Journal of Medicine, offering hope to those affected by ATTR, a condition that leads to heart failure due to amyloid protein accumulation.