Odylia Therapeutics

Freya is a happy, caring ,fun, bright 9 year old girl, who loves baking, crafts, reading, horse riding and raising money for different charities and causes as she loves to help others. Watching Freya you probably wouldn’t realise that she is registered visually impaired, having been born with Nystagmus, which causes blurred and incentive vision, and LCA (lebers congenital amourosis) RGRIP1 gene which is a degenerative condition. As Freya’s parents (Gav & Paula Birch) we hope and pray that treatment can become available to prevent this and retain her vision The support we have had over the years is amazing, and our hopes are for it to continue and grow bigger and stronger. Odylia Therapeutics is a charity developing a gene therapy to treat vision loss caused by RPGRIP1 mutations, for which there is currently no treatment. Odylia partners with patients, families, and researchers all over the world to accelerate therapeutic development and is currently conducting late-stage preclinical experiments and is preparing for IND submission for use of the gene therapy in clinical trials. Odylia’s focus starts and ends with patients. We are committed to changing the future for families affected by rare disease around the world. YOU can help bring promising treatments to patients. You can donate from this page - donations are made in USD ($) but then will be converted converted on the backend to GBP which you will see on your statement after the transaction. At Odylia, we accelerate therapeutic development for people with rare diseases, changing the way treatments are brought from the lab to the clinic. We strive to bring life altering treatments to people with genetic disease regardless of prevalence or commercial interest. Odylia is shifting the paradigm to focus on proven science and removing traditional roadblocks. We work to lower costs, reduce development time, focus research, and continuously drive successful programs towards clinical trials and regulatory approval. Strategic collaborations with patient groups, research labs, and commercial partners facilitate our work to ensure treatments are safe, effective, and ultimately reach patients who need them.

This is Rem Nabuurs Kim, born on the last day of 2023. He is a very lively boy who radiates a zest for life and shares it with everyone around him. Unfortunately, he has a visual impairment. His sight is poor due to a rare genetic disorder, and without available treatment his vision will likely continue to deteriorate. There is currently no cure for this condition — Leber Congenital Amaurosis (LCA) caused by a mutation in the RGRIP1 gene — yet Odylia Therapeutics, a non‑profit organization in the United States, is working hard on a solution. The progress of their research is promising, but it still depends on sufficient funding. Only with an adequate budget can the full development process be completed and the gene therapy become available — not just for Rem, but for everyone affected by this mutation. That’s why we kindly ask you to contribute to this research. Let’s hope this will help Rem preserve, or even improve, his vision! Thank you in advance for your support, on behalf of Rem and his family. If you would like to learn more about LCA/RGRIP1 and the status and progress of the research, please continue reading on this page .