Radboudumc and LUMC launch center for RNA therapy for ultrarare diseases

4 March 2020

Researchers and clinicians of Radboud University Medical Center (Radboudumc) and Leiden University Medical Center (LUMC) and are jointly launching the Dutch Center for RNA Therapeutics (DCRT). The aim of this new virtual centre is to develop customised RNA therapies for patients with rare genetic conditions.

The DCRT will focus on RNA therapy for patients for whom local treatment of the affected tissue is possible. The treatment targets progressive eye, muscle or brain diseases. The focus is on genetic diseases and mutations, which are so rare that pharmaceutical companies have no interest to invest in developing treatments. The DCRT will be the first center in Europe to develop RNA therapy for such rare diseases.

RNA therapy

Most hereditary diseases are caused by errors in the DNA, resulting in either lack of protein or production of harmful proteins. RNA therapy uses antisense oligonucleotides (AONs) to restore protein production or reduce the amount of harmful protein. This does not reverse existing damage, but it can slow the progress of the disease.

“At the LUMC we have more than 20 years’ experience with designing and testing AONs. We see it as a duty of academic centres to make this treatment available to this specific patient group,” says professor of Translational Genetics Annemieke Aartsma-Rus, one of the initiators of DCRT. "Research in AONs has also been conducted in Nijmegen for more than ten years, including in the field of hereditary eye disorders and Usher syndrome, with promising results” says Rob Collin, research leader at the Department of Human Genetics at Radboudumc.

The DCRT has been made possible by a financial contribution from the Department of Human Genetics from the LUMC. The centre is not for profit and will be led by Dr. Willeke van Roon-Mom and Prof. Annemieke Aartsma-Rus from the department of Human Genetics at the LUMC. Also involved are neurologists Dr. Erik Niks and Prof. Jan Verschuuren, ophthalmologists Prof. Camiel Boon and Prof. Gré Luyten, and hospital pharmacist/clinical pharmacologist Prof. Henk-Jan Guchelaar.

Besides Dr. Collin, the following researchers and medical specialists from Nijmegen are involved in DCRT: geneticists Prof. Frans Cremers, Dr. Susanne Roosing and Dr. Alex Garanto,  Dr. Erwin van Wijk as researcher of the ENT department, ophthalmologist Prof. Carel Hoyng, and ENT specialist Dr. Ronald Pennings.

European Reference Networks

The DCRT focuses specifically on the treatment of rare diseases, a key priority for patient care within the LUMC. The LUMC is the coordinating centre of the European Reference Network (ERN) for rare endocrine disorders (Endo-ERN). In addition, both the LUMC as well as RadboudUMC are participating centre of expertise in several ERNs for e.g. bone diseases, eye diseases, lung diseases, solid cancers, haematological diseases, neuromuscular diseases and connective tissue and musculoskeletal diseases.