As we wrap up an incredible year, we at Rare Trait Hope Society want to thank you 🙏 our valued supporters and sponsors 🫶 for being the heart of our mission. Through your generosity, we’re making strides in advancing hope for those impacted by Aspartylglucosaminuria (AGU) and other rare conditions. This holiday newsletter shares exciting developments, upcoming projects, and reflections on our year together 🤗
Your support makes everything we do possible! 🙌
💁🏼♀️ About Rare Trait Hope ❤️
Rare Trait Hope is dedicated to pioneering gene therapy solutions and supporting families impacted by AGU, a rare genetic disorder. Our work focuses on advancing treatment, understanding, and support for AGU patients and families worldwide.
Together, we’re creating hope where it’s needed most.
👧🏽 Together, Let’s Fund Hope! 🧒🏻
ACHIEVED!
💊 Orphan Drug Designation for DANAGALEX ✍🏻
What a way to end 2024!
We’re thrilled to announce that in November, our sister organization, Rare Trait Hope Fund, received ‘orphan drug designation’ from the FDA for our investigational gene therapy, DANAGALEX, aimed at treating Aspartylglucosaminuria (AGU). AGU is a rare disorder caused by mutations in the AGA gene, leading to significant health challenges. DANAGALEX is designed to target the root cause of AGU with a one-time gene therapy dose and is currently awaiting FDA approval to begin a Phase 1/2 clinical trial 🧑⚕️👩⚕️
The FDA’s orphan drug designation supports the development of treatments for rare diseases affecting fewer than 200,000 individuals in the U.S. This designation not only recognizes the importance of DANAGALEX but also offers benefits like market exclusivity, tax credits, and a waiver of drug approval fees. We are hopeful that this progress will transform lives by addressing AGU at its source ⭐️
We recently launched an AGU Registry and Natural History Study on the National Organization for Rare Disorders (NORD) “IAMRARE”® Research platform, https://agu.iamrare.org/. This study will document the progression of AGU over time, providing critical insights to guide clinical trials and improve patient care. 🧑💻 On November 26th, the AGU Patient Registry began collecting data about individuals with AGU, helping to understand the disease’s impact, track long-term health outcomes, and enable collaboration among researchers. The registry will be available globally starting in early December. 📔
Meet Nazira, a brilliant and compassionate cat 😸 who narrates the inspiring journey of a family battling the ultra-rare genetic disease Aspartylglucosaminuria (AGU). Through humor, love, and resilience, this memoir highlights the parents’ relentless efforts to secure a diagnosis and develop a groundbreaking gene therapy. Written by Dr. Alla Comardelle and Julia Taravella, illustrated by Portland artist Kiel Parsons, this “meow-moir” 📖 offers a heartfelt glimpse into the world of rare diseases, celebrating the power of determination. All proceeds support our American sister charity raretrait.org in funding AGU treatment development, and turning hope into life-saving reality.
Print Version: CAD$25.90
Kindle Version: CAD$9.99
🔔 The TMX Market Close ceremony was an exciting opportunity to create awareness, while our panel discussion was engaging and thought-provoking, thanks to the expert panellists who shared their insights and experience in the field of gene therapy. We believe that the knowledge gained from this event will undoubtedly help us in our mission to revolutionize healthcare for many neurologically-based rare diseases. Special thanks to TMX for sponsoring our inaugural Toronto event—and especially to John McKenzie, CEO of TMX Group for his awesome role as moderator for the panel discussion 🎤
Click here to watch the market close!
📣 Help Us Spread the Word! 🙋
Our global parents group meets with health officials, the academic community and organizations from around the world. If your business, service group or organization is interested in learning more about what is happening in the world of rare diseases—and how you can help us fight for the health of all our kids—please contact our Executive Director Barbara Insley at barbara@raretraithope.ca
We would love to have you help us spread the word!
Our inaugural 50/50 draw raised nearly $10,000, with the winner taking home half 🥳 We’re excited to host our next draw in late Winter/ Spring 2025. Stay tuned for details and thank you to everyone who participated! Every dollar counts when it comes to AGU research 💸
🎰 On September 19th, we hosted the Red Diamond Casino Night at the beautiful Ernst & Young space in ‘The Stack’ on Melville Street in Vancouver. With breathtaking views and an array of fun casino games, fantastic food and an eye-popping silent auction, it was an unforgettable evening 🎲 The proceeds of the event got us one significant step closer to paying for our upcoming AGU clinical trial. A heartfelt thanks to our ‘Casino Royale Sponsor’ Capital Direct Financial and our ‘Presenting Sponsors’ Great Canadian Entertainment and Gateway Casinos & Entertainment for making this event possible.
As we approach Rare Disease Month in February 2025, we’ll be sharing more exciting fundraising ideas From hosting your own event to a wine tasting 🍷 to purchasing a 50/50 ticket 🤲 there will be more ways to get involved. Stay tuned! Contact us for guidance on how you can make an impact 💫
End-of-Year Giving
We are so grateful for your generous support this year, which has helped make so much possible. As we close out 2024, please consider making an end-of-year gift to Rare Trait Hope to further our work in 2025 🎁 Not only will your donation bring hope to those facing AGU, but it may also provide tax benefits for you this season. We gratefully accept donations large and small, monthly donations, and stock and legacy gifts. Please reach out if you have any questions—we’d be delighted to chat 💬
Again, thank you for being a part of our Rare Trait Hope family 🤍
We wish you a joyous holiday season and look forward to achieving even more together in the year ahead! ☃️