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Klin Monbl Augenheilkd. 2019 Mar;236(3):253-260. doi: 10.1055/a-0842-6778. Epub 2019 Mar 21.

[Development of cGMP Analogues for the Pharmacological Treatment of Neurodegenerative Diseases of the Retina].

[Article in German; Abstract available in German from the publisher]

Author information

1
Forschungsinstitut für Augenheilkunde, Department für Augenheilkunde, Eberhard Karls Universität Tübingen.

Abstract

in English, German

Hereditary retinal degenerative diseases are mostly diseases of the photoreceptors and/or the retinal pigment epithelium, which lead to loss of vision or even complete blindness. To this day, these diseases are mostly untreatable and represent a considerable burden for patients and their relatives. This review article highlights some of the challenges that arise in the development of new therapies for inherited retinal degeneration, in particular the problem of the enormous genetic heterogeneity of these diseases and the question of how new forms of treatment can be made to cross the blood retinal barrier to the nerve cells of the retina. In this context, the central role of the messenger substance cyclic guanosine mono-phosphate (cGMP) in the photoreceptor is discussed and how this can be used to develop mutation-independent therapies. The DRUGSFORD project will be used as an example to explain how new drugs can be formulated to overcome the blood retinal barrier. In addition, other difficulties will be discussed that arise when positive results from applied research are to be transferred into clinical development. On the one hand, gaps and a lack of interdisciplinarity in the training of scientists and physicians are pointed out; on the other hand, lack of robust data on the natural progression of these disorders and suitable biomarkers also impede clinical development.

PMID:
30897644
DOI:
10.1055/a-0842-6778

Conflict of interest statement

François Paquet-Durand ist Erfinder auf 2 Patentschriften (PCTWO2016/146669A1, PCT/EP2017/066113) und hat für die Leitstruktur CN03 eine Orphan Drug Designation der European Medicines Agency (EMA) erhalten (EU/3/15/1462). François Paquet-Durand ist Anteilseigner an der Firma Mireca Medicines GmbH, die die klinische Entwicklung von CN03 vorantreibt.

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