TCAR started out to help develop a treatment for Rose and in that process, determine if we could build better systems and platforms for the thousands of diseases like Rose’s, that pharma will never help. We have stayed true to this mission. At every step of the way, your support has helped us develop new systems and new processes that can be scaled for the future of genetic medicines, and for children like Rose.

Since Our Inception: 

• We created Rosiphersen, a treatment for Rose’s disease. It's shown to be safe in animals and human neurons, and it's working in these models!
• We started RareLabs, a scalable multi modality lab for families. To date RareLabs is working on 20 diseases, and has discovered 8 new treatments.
• We spun out our commercial company, AlphaRose Therapeutics. AlphaRose is a public benefit corporation that is focused on scaling ultra rare genetic treatments sustainably. AlphaRose was co-founded by ex-Genzyme executives, and will be taking Rosiphersen and many other ultra rare treatments into clinical trials and commercializing them globally. 
• Our impact is spreading, across the globe with interviews in People Magazine, Newsweek, and many others!

When Rosiphersen is commercialized, TCAR will recieve royalty payments potentially in the millions, and a sustainable foundation will be created. We want to turn every dollar you donate into $100 dollars. We want to turn Rosiphersen into hundreds of treatments for thousands of children, globally.  

Our Immediate Focus and Why Your Donation is Crucial:

Our current mission is to complete the necessary preparations for a successful clinical trial of Rosiphersen in 2026. This requires two critical initiatives that must be open-source and accessible to all researchers and institutions, not proprietary.

Biomarker Discovery: We are developing biological readouts that are essential for gaining FDA approval and ensuring insurance coverage for Rosiphersen. These biomarkers will provide objective evidence that the drug is working.

Natural History Study Platform: Establishing a patient-specific baseline is vital for demonstrating the meaningful benefit of Rosiphersen to the FDA and justifying the cost to insurance providers. Given the variability in children with neurodevelopmental diseases, we are pioneering a new model. This platform uses cutting-edge wearables to allow every patient to serve as their own control—a potential breakthrough for accelerating clinical trials for ultra-rare conditions like Roses.

• With your support, we can treat Rose and other children with Rosiphersen.

• Rose has had seizures now, like many of these children, and we don't know how much time we have left. Urgency is critical.

• Children die every day from these neurological diseases. We can change that with scalable genetic medicines.

Hope has a timeline. Donate Today. Let's fuel this fight for 2026!!