Clinical Progress: Moderna Advances mRNA Therapy for Fabry Disease

Moderna Therapeutics has launched a Phase 2 clinical trial for its investigational mRNA therapy targeting Fabry disease, a rare lysosomal storage disorder characterized by deficient alpha-galactosidase A enzyme activity. The trial will assess safety, dosing, and preliminary efficacy among adult patients with classic Fabry disease symptoms.

Innovation Spotlight: mRNA Beyond Vaccines

This program marks a significant expansion of mRNA technology into rare genetic disease treatment, leveraging Moderna’s platform to deliver therapeutic proteins systemically. Fabry disease offers a compelling target due to its well-characterized genetic basis and unmet clinical need for more effective, less burdensome therapies than current enzyme replacement options.

Industry and Patient Impact

Experts believe positive outcomes could forge a new paradigm in managing lysosomal storage disorders using mRNA therapeutics. Patient advocacy groups have welcomed the trial, emphasizing the importance of expanding clinical trial access to underrepresented populations.

Future Outlook

Moderna aims to generate pivotal Phase 2 data by late 2025, supporting future regulatory submissions worldwide. The company plans additional research into related disorders, reinforcing its leadership in innovative RNA medicines.