Key Takeaways from the 21st Annual WORLDSymposium on Lysosomal Disease Research (11 February, 2025)

The field of lysosomal disorders is making great strides, with breakthroughs in basic science paving the way for potential new treatments. Yet, significant challenges remain—particularly in safely and effectively delivering therapies across the blood-brain barrier, a critical hurdle for both gene therapy and enzyme replacement approaches.

One of the most exciting developments has been the approval of gene therapy for Metachromatic Leukodystrophy (MLD), offering a curative option for pre-symptomatic patients. However, its reach remains limited, especially as universal neonatal screening for MLD is still far from widespread.

At Release Therapeutics, we are committed to expanding treatment options for MLD patients beyond the scope of gene therapy. Our implantable enzyme replacement therapy has the potential to usher in a new era of hope for patients and families who currently have no viable therapeutic options.

Participating in WORLDSymposium was a true honour, and we are deeply encouraged by the positive feedback from the MLD and lysosomal disease community on our technology and recent studies in non-human primates.

Dean Suhr, President and Co-Founder of the MLD Foundation, and Teryn Suhr, alongside our strategic collaborator Dr. Françoise Piguet, Head of GENOV at the Paris Brain Institute, and Julien Grogg, our CEO.

A huge congratulations to the MLD Foundation on receiving the 2025 Patient Advocacy Leadership Award—an incredible recognition of their unwavering dedication to the MLD community!

Stay tuned for more updates,

The Release Tx Team