Cure GM1 Held an INTERACT Meeting About ERT with The FDA

Cure GM1 Moves ERT One Step Closer to A Trial

We have some incredible news for the GM1 community. On July 16, 2026, Christine Waggoner and other representatives of the Cure GM1 Foundation met with representatives of the U.S. Food & Drug Administration (FDA) to discuss the future of Enzyme Replacement Therapy (ERT) as a treatment for those living with GM1 gangliosidosis. 

During this meeting, the Cure GM1 team discussed the years-long de-prioritization of GM1, despite a good number of clinical trials. The team shared the promise they see in ERT for all forms of GM1.

In order to hold this meeting, Cure GM1 had to submit extremely detailed information regarding the current project status and detailed plans to develop its own ERT treatment for GM1. This was a very significant milestone!

The first in a multi-step process in developing ERT for GM1

The road to a clinical trial is long, but this INTERACT meeting was a major milestone in moving forward in the process.

ERT Development

Why Cure GM1 believes in ERT for GM1

Enzyme replacement therapy works by supplying the body with the functional enzyme it is missing. Unlike small molecule approaches, ERT does not depend on a patient having residual enzyme activity.

Unlike AAV-based gene therapy, ERT is not limited by a patient’s antibody status. And ERT can be used in combination with other treatment approaches. For these reasons, ERT has the potential to benefit GM1 patients, across subtypes and across ages.

Moving Forward with ERT

The Cure GM1 team came away from the INTERACT meeting feeling hopeful and supported. In addition, with guidance from the FDA, the team now has a clear plan for next steps. 

This is incredibly exciting news. We need your help and support and continue to push forward. 

What you can do to keep us driving forward: 

Thank you for everything you have done to support those living with GM1.