Cure GM1 is dedicated to investing in research, seeding new projects, and to the development of multiple treatment modalities for GM1 gangliosidosis.  In collaboration with our advisors and consultants, who have decades of experience in biotech and research, Cure GM1 has selected a contract research organization (CRO) to further the development of enzyme replacement therapy (ERT). 

Since the summer of 2024, we have been building a team and resources to enable the development of enzyme replacement therapy, a therapeutic approach that possibly can be used for all GM1 patients. ERT is different from other treatment approaches, such as small molecules which are limited to those patients with residual enzyme activity.  In addition, unlike AAV gene therapy, ERT can be provided to all GM1 patients, regardless of their AAV antibody burden.  Finally, and importantly, enzyme replacement can be used in conjunction with other treatment modalities, including both small molecules or gene therapy.  For these reasons, we are excited to announce this important investment in advancing development of enzyme replacement therapy today.

Rare disease patient advocacy groups are catalysts of change and we have worked for many years to build a team and to raise the funds to make this significant investment today.  It is only through the support of our friends, colleagues and community that this new initiative is possible.  We must continue to raise funds and to attract investment in GM1 to ensure that progress continues towards multiple potential approaches to treat this complicated and devastating disease.  Initiation of human clinical trials is the ultimate goal, which will require partnership, additional resources, and continued dedication.  The more funds we raise, the further and faster we can move forward.

A special thank you to the team members who have contributed so meaningfully to this exciting project thus far.  And thank you to every person who has and continues to support our critically important mission.

To contribute to our work, please donate.