As families living with GRIN, GRIA, GRIK and GRID Disorders, we are all fighting the same clock. We want effective treatments, and we want them as fast as possible.
Today, I am incredibly proud to share a major milestone in our journey toward treatments and cures. CureGRIN is putting the finishing touches on a groundbreaking new resource for biotech and pharmaceutical companies: The ‘How to Treat and Cure GRI Disorders” Guide. Ahead of its official publication this July, we want to give our family community early access to the exact roadmap we are taking directly to biopharma leadership.
We recently shared a sneak peek of the data behind this guide online at GRICON26 and in person at the GRIUK conference in Edinburgh. Today, we want to share a more detailed version of the presentation deck with you.
Click Here to View the GRICON26 / GRIUK Slide Deck
What is the “How-To” Guide, and Why Does it Matter?
In the past, precision medicine for rare diseases has been slowed down because drug development was trapped in a ‘mutation-by-mutation’ bottleneck—treating every single child’s genetic spelling error as an entirely isolated puzzle. Our new guide completely changes the game for the biopharma industry by shifting to a mechanism-based, systemic approach. By grouping hundreds of unique variants into clear functional pathways, we provide companies with a single roadmap to develop therapies that can treat entire groups of patients at once.
We have effectively mapped out the entire GRI landscape to show industry exactly how to build drugs for our kids. The guide delivers:
- The 7 Therapeutic Strategies: A comprehensive framework that groups patients into clear, actionable treatment pathways based on how their variant behaves (including Boost, Reduce, Augment, Knockdown, Knockdown & Augment, Edit, and Read-through).
- 80+ Potential Interventions: A meticulously curated list of genetic tools, RNA therapies, and small-molecule targets that are ready for biotechnology companies to start developing into actual treatments right now.
- Essential Commercial & R&D Tools: A complete toolkit to help companies start working immediately, including curated lists of animal and cell models, detailed genotype-to-phenotype modeling, patient population estimates and updates on unpublished research.
Real Data from Real Families: The GRI Census Insights
The slide deck also features the initial results from the landmark Global GRI Census, which we launched to collect real-world clinical data directly from parents and caregivers.
Thanks to 546 amazing families who completed the census so far, we have been able to map out critical symptom data across 365 unique variants. For the first time, our deck shows industry exactly how symptoms like developmental delays, epilepsy, muscular issues, and behavior challenges vary by specific gene group and functional subtype.
This data is vital because it proves to biopharma that we are a cohesive, organized, and trial-ready community.
What’s Next: Taking the Map to the Market
Writing the guide was only step one. Now, we are aggressively pitching it to the companies with the technology to build these cures.
- The June Roadshow: This June, our Science Director, Dr. Quinn Pauli, and I will be traveling to major biotechnology clusters across the United States to brief executive and R&D teams at major biopharma companies.
- The Summer Virtual Meetings: We will follow these in-person roadshows with a packed schedule of virtual executive briefings throughout July and August to capture even more pipeline interest.
- The Family Version is Coming: While the primary guide is a deeply technical manual for drug developers, we are developing a family-friendly version of the “How-To” Guide, which will be released to all of you this summer.
Building a Custom Roadmap for All 11 Disorders
Looking ahead, CureGRIN will be utilizing this exact strategic framework to build distinct, custom therapeutic roadmaps for the specific disorders we represent, including GRIN1, GRIN2A, GRIN2B, GRIN2D, GRIA1, GRIA2, GRIA3, GRIA4, and GRIK2.
While many small-molecule treatments will target shared biological pathways across multiple GRI genes, highly specific DNA and RNA therapies must be custom-tailored to each individual gene. By applying our master ‘How-To’ framework to each gene community separately, we can pinpoint the exact genetic tools needed for your child’s specific disorder.
This targeted approach will also allow us to explore new ways to align donor funding directly with the precise research projects that matter most to your family. Whether your child has a GRIN2B haploinsufficiency variant or a GRIA1 gain-of-function variant, our goal is to show you exactly where your fundraising dollars are going and how they are driving a cure for your child.
As Dr. Stanley Crooke, the founder of the N-Lorem Foundation, beautifully notes on the opening slide of our deck:
“We have learned unequivocally and clearly that all the things we were taught… about developmental delays being permanent is simply not true anymore.”
We are proving that every day.
Thank you for your continued trust, your participation in our census, and your unwavering support as we build the road to a cure.
With hope and determination,
Keith McArthur Executive Director, CureGRIN Foundation
Father to Bryson (GRIN1)
