You are here: vision-research.eu » Vision Research » Vision in the European Focus » Developing a new treatment for hereditary retinal degeneration

Developing a new treatment for hereditary retinal degeneration

A novel combination of cGMP analogue and drug delivery system provides functional protection in hereditary retinal degeneration. Hereditary retinal degeneration is a neurodegenerative condition that leads to loss of photoreceptors and blindness and that still lacks effective treatments.

To promote the development of new treatments for these diseases, researchers from the Universities of Tubingen, Lund, and Modena and Reggio Emilia, as well as the companies BIOLOG and to-BBB have founded the DRUGSFORD project, which was supported by the 7th Framework Programme of the European Union with nearly 5 Million Euro (HEALTH-F2-2012-304963).

Previous research had identified an over-activation of the cGMP signalling pathway in degenerating photoreceptors. In an article published this week in the journal PNAS, DRUGSFORD researchers now report that administration of a nanosized liposomal formulation containing an inhibitory analogue of the signalling molecule cGMP helped reduce photoreceptor loss. Moreover, this novel compound formulation preserved retinal function in three different rodent models of hereditary retinal degeneration.

To forward the clinical development of this novel cGMP analogue formulation, the DRUGSFORD partners have jointly founded the start-up company Mireca Medicines. The future clinical translation efforts of Mireca Medicines will be further helped by an Orphan Drug Designation for the cGMP analogue that was granted by the European Medicines Agency (EMA; EU/3/15/1462). This designation facilitates clinical testing and improves the perspectives for a future commercialisation.

The original article by Eleonora Vighi et al. was published in Proceedings of the National Academy of Sciences of the USA (PNAS) on 12th March, 2018 (Article #17-18792: “A novel combination of cGMP analogue and drug delivery system provides functional protection in hereditary retinal degeneration”).

Further information may be found on the DRUGSFORD project website (www.drugsford.eu), or the company website of Mireca Medicines (www.mireca.eu).

Original Publication:

"Combination of cGMP analogue and drug delivery system provides functional protection in hereditary retinal degeneration"

Eleonora Vighi, Dragana Trifunović, Patricia Veiga-Crespo, Andreas Rentsch, Dorit Hoffmann, Ayse Sahaboglu, Torsten Strasser, Manoj Kulkarni, Evelina Bertolotti, Angelique van den Heuvel, Tobias Peters, Arie Reijerkerk, Thomas Euler, Marius Ueffing, Frank Schwede, Hans-Gottfried Genieser, Pieter Gaillard, Valeria Marigo, Per Ekström and François Paquet-Durand

PNAS 2018; published ahead of print March 12, 2018, https://doi.org/10.1073/pnas.1718792115

Contact:

Prof. Dr. rer. nat. François Paquet-Durand
Cell Death Mechanism Group
Institute for Ophthalmic Research
University of Tuebingen
Elfriede-Aulhorn-Straße 7, 72076 Tuebingen, Germany

e-mail: francois.paquet-durand@uni-tuebingen.de
Skype: francois.paquet-durand
Phone: +49 7071 29 87430
Fax: +49 7071 29 5777