Our global, grassroots effort brings new hope to families affected by Aspartylglucosaminuria
We are thrilled to share that the U.S. Food and Drug Administration (FDA) has given approval to the start of a clinical trial for the treatment of the ultra-rare disease Aspartylglucosaminuria (AGU) 🧬✨
For families affected by AGU, this moment represents something long hoped for: 🙏 the possibility of a treatment where none has existed before.
Announcement:
Study Overview
Brief Summary
The goal of this clinical trial is to learn if the treatment is a safe, tolerable, and efficacious treatment for adults and children with Aspartylglucosaminuria (AGU).
Detailed Description
This study is a first in human (FIH) open-label study designed to assess the safety and efficacy of administration of an adeno-associated viral vector serotype 9 (AAV9) carrying the gene encoding aspartylglucosaminidase (AGA) in participants with Aspartylglucosaminuria (AGU). Safety will be monitored continuously throughout the study for adverse / serious adverse events and dose limiting toxicities. All outcomes (primary, secondary, exploratory) will be assessed at 52 and 104 weeks.
Offical Title
An Open-Label, Single Center, Phase 1/2 Study to Evaluate the Safety and Efficacy of DANAGALEX (scAAV9/AGA) in Participants With Aspartylglucosaminuria (AGU)
It Takes a Global Village
This extraordinary milestone marks the culmination of more than 13 years of dedication led by our sister organization, Rare Trait Hope Fund. Our Rare Trait Hope Society was established in Canada five years ago to help support the fundraising efforts that made this moment possible 🫶
From beginning to end, this has been a truly global 🌍 grassroots initiative. With no pharmaceutical company backing and no major institutional funders, a determined group of parents—facing the devastating realities of this disease—came together 🤝 They supported one another, sought out experts willing to help, and contributed their own professional skills and time to move this work forward.
More than 10,000 individuals around the world have donated to this effort 🙌 Alongside significant fundraising in the United States, hundreds of thousands of dollars have been raised in Australia while here in 🇨🇦 Canada supporters have contributed over $1 million to date. AGU relatives in Switzerland, France, Spain, Finland and Mexico also sent contributions from local fundraisers.
Those Who Have Brought Us to This Point
We extend our deepest gratitude to Julia Taravella and her team of experts, who moved this program forward and successfully secured FDA acceptance of the Investigational New Drug (IND) application. Many of these contributors volunteered their time, working tirelessly behind the scenes to make this possible.
We also honour the AGU families whose courage and determination mobilized communities, inspired generosity, and made this achievement a reality 👏👏👏
And of course, a HUGE THANK YOU to YOU 🙏 who have loyally supported Rare Trait Hope Society through this long journey.
The Next Steps to Come
🏃🏻 This latest milestone brings real hope—but the work is not yet finished. We look forward to sharing more as we move forward, together.
Please donate TODAY to support the clinical trial.
Help bring this treatment closer to the kids and families who need it.