Molecular Biology of Retinal Degenerations
Preclinical development of drugs and drug delivery technology for the treatment of inherited photoreceptor degeneration
Facts & Figures
- Duration: 3 years,
- Funding Agency: EU (HEALTH-F2-2012-304963)
Dysregulation of cGMP is a pathological hallmark of inherited retinal degenerations (RD) affecting photoreceptors, the sensory cells of the retina. These RDs, including Retinitis Pigmentosa, Lebers Congenital Amaurosis, and Achromatopsia, are major causes of blindness, affecting approximately one in every 2000 individuals worldwide. They still remain without effective treatment. In photoreceptors, cGMP is produced by retinal guanylyl cyclase (GC). The two main cGMP targets are cyclic nucleotide gated ion channels (CNGC) and cGMP-dependent protein kinase (PKG). Recent data suggests that blocking either GC, PKG, or CNGC may constitute a viable therapeutic approach.
DRUGSFORD will develop targeted compounds and delivery systems preventing photoreceptor damage in preclinical disease models. Towards this goal, two SMEs have teamed up with three academic research groups focused on retinal degeneration: the German company BIOLOG specializes on development of cyclic nucleotide based drugs targeting PKG, CNGC, and GC; the Dutch company to-BBB develops systems to deliver drugs across the blood brain/retinal barrier (BBB, BRB, resp.); the groups of V. Marigo (Modena, Italy), P. Ekström (Lund, Sweden), and F. Paquet-Durand (Tübingen, Germany) have a strong joint collaborative track record of studying photoreceptor degenerative mechanisms and evaluating drug treatment effects. The results of the project will allow the SMEs to further co-develop these drugs towards translation into clinical studies, addressing the high needs of RD patients and the high economic benefit of such therapies.
- Pieter Gaillard, to-BBB
- Hans Gottfried Genieser, BIOLOG
- Per Ekström, Lund University
- Valeria Marigo, Modena University
Scientists in Ueffing Lab
- François Paquet-Durand (Project Coordinator)