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Preclinical development of drugs and drug delivery technology for the treatment of inherited photoreceptor degeneration

Objective

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, and 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). Since attenuation of PKG and CNGC activity can reduce photoreceptor cell death, both proteins constitute potential targets to prevent RD. Recent data suggest that blocking retinal GC may also constitute a viable therapeutic approach.
This consortium will study and develop targeted compounds and delivery systems aimed at 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 and joint collaborative track record of studying photoreceptor degenerative mechanisms as well as on testing and evaluating drug treatment effects. Manufacturing of the most promising drugs fitted to a suitable delivery system will be scaled up towards clinical-size batches and studied towards efficacy, toxicology and off-target effects in model animals. 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.

Field of science

  • /natural sciences/biological sciences/biochemistry/biomolecules/proteins
  • /medical and health sciences/clinical medicine/ophthalmology
  • /natural sciences/biological sciences/genetics and heredity/nucleotide

Call for proposal

FP7-HEALTH-2012-INNOVATION-2
See other projects for this call

Funding Scheme

CP-FP - Small or medium-scale focused research project

Coordinator

EBERHARD KARLS UNIVERSITAET TUEBINGEN
Address
Geschwister-scholl-platz
72074 Tuebingen
Germany
Activity type
Higher or Secondary Education Establishments
EU contribution
€ 992 813,74
Administrative Contact
Thomas Wheeler-Schilling (Dr.)

Participants (5)

TO-BBB TECHNOLOGIES BV

Participation ended

Netherlands
EU contribution
€ 719 117,27
Address
Jh Oortweg 19
2333 CH Leiden
Activity type
Private for-profit entities (excluding Higher or Secondary Education Establishments)
Administrative Contact
Pieter Gaillard (Dr.)
BIOLOG LSI FORSCHUNGS- UND BETEILIGUNGS-GMBH
Germany
EU contribution
€ 1 396 647,48
Address
Flughafendamm 9A
28199 Bremen
Activity type
Private for-profit entities (excluding Higher or Secondary Education Establishments)
Administrative Contact
Hans-Gottfried Genieser (Dr.)
UNIVERSITA DEGLI STUDI DI MODENA E REGGIO EMILIA
Italy
EU contribution
€ 760 292,52
Address
Via Universita 4
41121 Modena
Activity type
Higher or Secondary Education Establishments
Administrative Contact
Fabio Prati (Prof.)
MAX IV Laboratory, Lund University
Sweden
EU contribution
€ 766 650,64
Address
Paradisgatan 5C
22100 Lund
Activity type
Higher or Secondary Education Establishments
Administrative Contact
Per Ekström (Dr.)
SP PROCESS DEVELOPMENT AB
Sweden
EU contribution
€ 335 906,35
Address
Po Box 857
501 15 Boras
Activity type
Research Organisations
Administrative Contact
Christine Svärd (Ms.)