Many devastating human diseases are the result of an inability of the body’s cells to make specific proteins that have an essential function for the whole body. The replacement of proteins with normally functioning ones provides a potential approach to treat multiple devastating diseases. Ribonucleic acids (RNAs) that encode for such proteins have been the focus of much biopharmaceutical research and development, however, RNA’s inherent instability and immunogenicity has proven to be a significant hurdle in the past.
Based in Munich, Germany, ethris and its co-founders – Dr. Carsten Rudolph and Prof. Christian Plank, have developed a novel RNA modification technology that creates Stable and Non-Immunogenic Messenger RNA (SNIM®-RNA) molecules that are ideally suited for use in protein replacement therapies to treat monogenic genetic diseases.
“We are delighted to extend our existing collaboration with Shire that may bring novel therapeutics to patients suffering diseases with significant unmet medical need. We aim to provide significant numbers of patients with new therapeutic alternatives that may provide a lasting solution to their disease,” stated Dr. Carsten Rudolph, CEO and co-founder of ethris.
“As a leader in the treatment of rare diseases, we are pleased to be working with ethris to develop SNIM®-RNA based therapies, with the objective of developing new treatment options to patients suffering from these rare disorders” said Dr. Philip J. Vickers, Global Head of Research and Development, Shire Human Genetic Therapies.
“With Shire we have a partnership based on a shared appreciation of the importance of our SNIM® technology. Under this partnership, we are aiming to develop an RNA modification and delivery technology that efficiently results in protein replacement in relevant cellular populations, providing hope to patients suffering from genetic diseases which have devastating clinical outcomes” commented Prof. Christian Plank CSO and co-founder of ethris.