New mRNA Treatment Pathway
We have finally DONE it! Grann Pharmaceuticals on behalf of HOPE for Harvey filed the first application to the FDA for a Pre-Investigational New Drug on January 28, 2026 for the first ever targeted treatment of DLG4. This mRNA-based treatment will be delivered via regular infusions of Lipid Nano Particles (LNPs), which will send good copies of DLG4 into the body, passing the blood brain barrier, and hopefully expressing healthy copies of DLG4 protein. This LNP mRNA treatment is showing amazing and safe results in another active clinical trial in just 18 months. Right now we are trying ot raise another $100,000 by the end of February 2026, and we have a $25,000 matching pledge right now, which will help pay for our safety studies this spring 2026. Help make that $25,000 into $50,000 or more.
We hope to submit our final IND FDA application by early summer 2026 with hopes to start our first clinical trial for an Investigational New DLG4 Drug in Austin, TX by the end of summer 2026! We hope to quickly move into expanded access and start adding other patients by fall 2026.
You can help make this possible by donating today!
See the exciting press release below regarding the other clinical trial.
We have HOPE for Harvey and others like him! DLG4 is a protein associated with brain plasticity, which means, if we alter the impact of these mutations, learning can start to begin and seizures should decrease.
With that hope is the continued need to fundraise. 100% of your dollars go directly to researchers. Please consider donating today!
