DRUGSFORD-bis

Towards a clinical trial for hereditary retinal degeneration

01.09.2016 bis 31.12.2017

Inherited retinal degeneration (RD) is a devastating and currently untreatable neurodegenerative disease, which still lacks suitable molecular targets for therapeutic interventions. A major challenge for therapy development lies in the high genetic heterogeneity of RD, with disease-causing mutations in over 250 different genes. A second major challenge is the blood-retinal-barrier (BRB), which prevents pharmacological compounds from reaching the neuroretina. In the EU-funded DRUGSFORD project (www.drugsford.eu), these challenges were addressed by targeting cGMP-signaling as a disease driver common to many different types of RD and by combining pharmacological therapy with an innovative liposomal drug delivery system (DDS) designed to facilitate transcytosis across the BRB.
The DRUGSFORD-bis project proposed here will engage in all the preparatory work required to bring DRUGSFORD results forward to a first clinical trial in RD patients. These preparations concern the publication of available pre-clinical data, full analysis of toxicological studies, the potential broadening of the scientific basis and disease indications, the preparation of clinical trials, as well as the training of personnel needed for conducting clinical trials.