Real hope for two children (My Daughter) Fund

❤️ A PERSONAL APPEAL TO MY ASML COLLEAGUES Dear colleagues, At ASML, we work every day on complex challenges and push the boundaries of what technology can achieve. But the greatest challenge in my life has nothing to do with lithography. My daughter Isis and another Dutch child, Seppe, have a rare and highly progressive heart disease called restrictive cardiomyopathy, caused by variants in the TNNI3 gene. Their heart muscles cannot relax properly, making it difficult for their hearts to fill with blood. Both children already have significantly elevated pressures in their hearts. There is currently no treatment that addresses the underlying disease. Within three years after diagnosis, around half of affected children have died or required a heart transplant. We are now 2.7 years beyond Isis’s diagnosis. For us, time matters. 🔬 A REAL SCIENTIFIC OPPORTUNITY A promising peptide called C27, discovered by Professor Jian-Ping Jin at the University of Illinois Chicago, is designed to restore the heart’s ability to relax. Published laboratory research supports its mechanism, although C27 has not yet been tested in patients. With money raised by Stichting Tikkertje and matching support from the Dutch Heart Foundation, researchers at UMC Utrecht created patient-specific heart models from Isis and Seppe. These models offer a unique opportunity to test C27 in heart tissue carrying the children’s own genetic variants. ✅ WHAT WE HAVE ACHIEVED • Published scientific evidence supporting the C27 mechanism • Patient-specific stem-cell models from Isis and Seppe • Support from experienced academic and clinical partners • A concrete research and development plan • A Foresight research proposal ready for funding 🚀 FROM SCIENCE TOWARDS PATIENTS I founded Lusitro B.V. to bring together the science, research partners and development expertise needed to move C27 towards patients. I also wrote and developed the Foresight research proposal together with leading academic partners. The proposed programme aims to: • Test whether C27 improves relaxation in the heart models of Isis and Seppe • Develop a PET tracer to determine whether C27 reaches the human heart • Prepare the manufacturing, safety and regulatory evidence needed for clinical development • Prepare an exploratory Phase 0 first-in-human microdosing study in parallel The Phase 0 study would use a very small, non-therapeutic dose. It would not test whether C27 works as a treatment, but whether it reaches the heart and how it behaves in the human body. Preparing this route in parallel could save valuable time. 💶 WHY YOUR SUPPORT IS NEEDED NOW The Foresight proposal is ready for funding, but Lusitro must provide its own substantial cash contribution to unlock the wider research programme. I am personally investing in this work, but I cannot fund the required contribution alone. Your donation through Bright Funds will support the programme via Stichting Tikkertje and help us take the following critical steps: • Patient-specific testing of C27 • Peptide and PET-tracer development • Biodistribution and safety research • Preparation for first-in-human microdosing • Regulatory and clinical-development work ❄️ A POSSIBLE SNOWBALL EFFECT If successful, this programme could generate the evidence and momentum needed to attract further research partners and funding. It could accelerate C27 towards patients and potentially open new possibilities for treating other cardiomyopathies in which impaired heart relaxation plays a role. The immediate priority, however, remains Isis and Seppe. 🙏 I AM ASKING FOR YOUR HELP We cannot promise that C27 will become a treatment. But we now have: • A scientifically grounded therapeutic candidate • Heart models made from the children’s own cells • Experienced research partners • A concrete development programme • A realistic opportunity to find out whether C27 can help I want to be able to tell my daughter that we did everything possible to give her a chance, and that we did not allow a lack of funding to stop us from finding out. ❤️ FOR ISIS. FOR SEPPE. FOR THEIR FUTURE. Every contribution, large or small, helps us take the next critical step. Thank you for reading our story and for any support you can give. Mark Hage Your colleague at ASML Founder of Lusitro | Father of Isis 👉 Please donate through this Bright Funds campaign. 🔗 More about the research: https://www.lusitro.com 🔗 About the foundation: https://www.stichtingtikkertje.nl