Committed to Gene Therapies for Orphan Diseases

An “orphan” disease is considered to be a disease that affects fewer than 200,000 people. Yet, the impact of orphan diseases is anything but small. In the US alone, 25 to 30 million people suffer from any of 6,800 orphan diseases. And for those people, treatment options can be few, and cures, even fewer.

At Rimedion, our mission is to change that. Recent breakthroughs in DNA mapping have identified the genes that are responsible for many rare disease paths. As many as 6,000 of them have been identified as being caused by a single genetic defect. As gene therapy takes a historical leap forward, the promise of being able to reliably cure genetic disorders using viral vector technologies is now a reality.

At Rimedion, we are working to realize that promise, one disease at a time. Our proprietary viral vector technology shows promise as a flexible platform for providing long lasting vector caused by a single gene. We are currently conducting Phase I and II trials for a genetic therapy to treat Fanconi Anemia, a genetic disease that destroys the blood marrow and is usually fatal by young adulthood.

We do our work at Dr. Wade Clapp’s laboratories at Riley Children’s Hospital in Indianapolis, Indiana. Our headquarters office is located near the Indiana University School Of Medicine, also in Indianapolis. We are currently looking for development partners and investors to help us develop cures, and genetic treatment protocols, for other single gene diseases, as well. We’re committed to bringing the treatments of tomorrow here today, for some of the patients who need it most. Contact us for more information at
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