Research

We support scientific initiatives to aid in the development of genetic medicines for neglected rare diseases

Ultrarare diseases are often neglected by the pharmaceutical industry owing to the small number of patients who live with these conditions. The Research arm of the RTW Foundation contributes to addressing this problem in two ways: by funding drug-discovery projects on ultrarare diseases, and by providing advisory services to disease foundations interested in developing therapies for their patients.

Funding for drug-discovery projects on ultrarare diseases

Our current funding priority focuses on drug-discovery projects on diseases that affect fewer than 1000 people worldwide and for which no commercial drug-development program exists. We are therapeutic-area agnostic and have a strong preference for projects that involve clinically validated therapeutic modalities. We also have a strong preference for projects that have completed the proof-of-concept stage of development in cellular and/or animal models. Get in touch for more information.

Advisory services to disease foundations

We put our operational capabilities and our drug-discovery expertise to the service of the ultrarare disease community. Whether you have just created a new disease foundation or are already developing a therapeutic asset, we would be happy to provide our unbiased, expert opinion on the best path forward for your project. Get in touch for more information.