November 2025 Cure GM1 Catalyst

Thank You Tayara!

We wish Tayara well in a new full-time endeavor closely aligned with her interest in public policy. We are truly grateful for her work at Cure GM1 and we are grateful for her dedication to our community throughout her time at Cure GM1. Please feel free to mail info@curegm1.org or message Cure GM1 at any time with questions

Check Our Annual Calendar

Don’t forget to check our annual calendar and plan ahead our major events and dates!

View the Calendar

Registration is Free of Charge for GM1 Families for the WORLD Conference in San Diego

“WORLDSymposium 2026 in San Diego will be the place to connect, collaborate, communicate and continue to make global progress in lysosomal disease research.” Registration for families is free of charge for both the virtual and online access. Be sure to follow the special instructions to access free admission.

Cure GM1 Supports Clarity in Rare Disease Evidence Principles Introduced by FDA

Cure GM1 signs onto the Haystack Project’s letter to the FDA regarding concerns with the Rare Disease Evidence Principles (RDEP) framework and encourages fit-for-purpose, scientifically rigorous trial designs for all rare disease communities. Read more here.

GEMMA Biotherapeutics Creates New Affiliate RareTx for Ultra-rare Diseases

Rare Therapeutics, Inc., an affiliate company of GEMMA Biotherapeutics focused on gene therapy for ultra-rare diseases, was launched on 10/7! Its clinical-stage portfolio includes GM1 gangliosidosis, Krabbe disease, and metachromatic leukodystrophy.  

PRESS RELEASE
VISIT RareTx

Cure GM1 will continue to share new press releases and information regarding recruitment for all upcoming clinical trials as soon as the information comes available.

Clinical Trial Results Published on Two Studies 

Several important publications are now available as pre-prints regarding past clinical trials and results. We wish to express our gratitude to the teams for these years-long efforts to bring these trials to fruition. 

Venglustat in GM2 Gangliosidoses and Related Disorders: Results of the AMETHIST Randomized Controlled and Basket Trials
The trial was closed in 2024 due to the absence of positive trends on clinical endpoints in the primary population of late-onset Tay-Sachs participants. The authors write “Despite the randomized placebo-controlled design, this study faced limitations. Primary population participants were adults with longstanding disease manifestations that are unlikely to be reversible.” 

GM1 trial participants were in the secondary basket trial arm. There were seven GM1 participants which included those with juvenile GM1 and adult-onset GM1. Of particular note in the publication was a decrease in GM1 ganglioside in CSF by a median 29.1% and stability in several outcome measures.

Read more. 


AAV9 Gene Therapy in GM1 Gangliosidosis Type II: A Phase 1/2 Trial
This publication reports results on a phase 1-2 open label dose escalation study at the National Human Genome Research Institute. The authors state “This study provides preliminary evidence that administration of a single dose of AAV9-GLB1 is safe and well tolerated and suggests that it may lead to biochemical and neuroimaging improvements in Type II GM1 gangliosidosis.”

Read more

The same team recently published an article on a brain imaging technique that was developed during the AAV9 trial here.