Cure GM1 is dedicated to investing in research and to the development of multiple treatment modalities for GM1 gangliosidosis. In early 2026, we announced an additional allocation of up to $1.4 million towards enzyme replacement therapy for GM1.  This next phase supports critical development work required to advance enzyme replacement therapy further toward human trials.

This builds on our commitment of approximately $400,000 in early 2025. The work is progressing, and this new large investment represents a major step forward.

Why Enzyme Replacement Therapy?

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 the broadest possible population of GM1 patients, across subtypes and across ages.

Committing to Advancements in GM1 Research

Rare disease patient advocacy groups are catalysts of change. It is only through the support of our friends, colleagues, and community that this investment is possible. Initiation of human clinical trials is the ultimate goal, and the more funds we raise, the further and faster we can move forward.

We are grateful, and we are not done.