News

March 13, 2018

Developing a new treatment for 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 Tübingen, Lund, and Modena and Reggio Emilia, as well as the companies BIOLOG and to-BBB 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.

Publication

The original article by Eleonora Vighi et al. is published in Proceedings of the National Academy of Sciences of the USA (PNAS) on 12th March 2018 (https://doi.org/10.1073/pnas.1718792115: “Combination of cGMP analogue and drug delivery system provides functional protection in hereditary retinal degeneration”).

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

March 15, 2016

DRUGSFORD files PCT patent application

After a provisional patent filing in March 2015, the DRUGSFORD consortium has now filed a PCT patent application for a patent entitled: "Targeted liposomal delivery of cGMP analogues". This invention relates to some of the most important findings of the DRUGSFORD research project, namely the conjugation of an active pharmaceutical ingredient comprised in a nanocontainer, to mediate enhanced drug delivery  across the blood–ocular barrier. This methodology is to be used preferably for the treatment or the prevention of diseases of the retina, but may also be applicable to other diseases of the central nervous system. In addition, the DRUGSFORD consortium is preparing for further patent applications to protect several of its key research findings.

November 18, 2015

EU Commission grants 12 months extension to the DRUGSFORD consortium

The European Commission has granted a one year extension to the DRUGSFORD consortium, bringing the total project duration to four years. The amendment of the project became necessary after the bankruptcy of one of the original DRUGSFORD partners, the company to-BBB. This bankruptcy had entailed a number of delays, notably in the manufacturing and regulatory testing of the consortium´s first lead compound LP-DF003. To remedy these issues and to allow the continuation of the successful pre-clinical drug development programme, the company SP Process Development (Södertälje, Sweden) has joined DRUGSFORD and will concern itself with the establishment of a GMP-like production process. In the following, the consortium will commence the regulatory testing of LP-DF003 necessary to engage into clinical phase 1 testing after project conclusion. 

April 23, 2015

DRUGSFORD receives Orphan Drug Designation for their LP-DF003 compound

Rare diseases of the retina, such as Retinitis pigmentosa (RP), cause the photoreceptors of the eye to die. The consequences are severe vision loss or even blindness, not the least amongst young people, and unfortunately there is still no treatment for these diseases. The so called Orphan Drug Designation (ODD) relates to special legislation in Europe for rare and severe diseases, which facilitates clinical testing and provides for 10 years of exclusive market access.

DRUGSFORD, a Collaborative Project funded by the European Commission under the 7th Framework Programme (HEALTH-F2-2012-304963), aims to develop a drug and drug delivery system combination for the treatment of RP, up to the point where the combination is ready to enter clinical testing. The DRUGSFORD consortium was formed in late 2012 by three academic institutions, the University of Tubingen, the University of Modena and Reggio Emilia, and the University of Lund, as well as two small-to-medium enterprise partners, with expertise in cyclic guanosine monophosphate (cGMP) analogue synthesis (Biolog Life Science Institute, Bremen) and drug delivery systems (to-BBB, Leiden), respectively.

The DRUGSFORD concept relies on the fact that RP often involves dysregulated cGMP signalling in dying photoreceptors. This can be counteracted by synthetic cGMP analogues, which are delivered to the photoreceptors across the blood-brain-barrier.

Through testing in a variety of RP models, DRUGSFORD has identified a candidate cGMP analogue and delivery system combination, named LP-DF003. Importantly, treatment with LP-DF003 resulted in structural and functional rescue of diseased retinas in RP animal models. These very promising effects have now resulted in the European Medicines Agency (EMA) granting an Orphan Drug Designation to LP-DF003 (EU/3/15/1462; http://ec.europa.eu/health/documents/community-register/html/o1462.htm).

In obtaining an ODD for its first lead compound formulation, the DRUGSFORD consortium has made a significant step towards the clinical development of a new treatment for RP and has strongly improved the prospects for future commercialisation of LP-DF003.

Press Release in German [PDF]

October 09, 2012

EU unterstützt den Kampf ums Augenlicht

Tübinger Forscher übernehmen Federführung: Neue Wege in der Behandlung erblicher Seherkrankungen

[German language press release from the University of Tübingen]

Mit nahezu fünf Millionen Euro unterstützt die Europäische Union in den nächsten drei Jahren einen Zusammenschluss aus Firmen und Wissenschaftlern unter der Führung des Forschungsinstituts für Augenheilkunde am Universitätsklinikum Tübingen. Ziel des Forschungsprojektes „Drugsford“ ist es, neue Wege für die Behandlung von erblichen Seherkrankungen zu finden.

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July 09, 2012

to-BBB awarded European FP7 funding

to-BBB awarded EUR 1.25 million European FP7 funding to develop treatments for inherited retinal diseases

to-BBB, the Dutch brain drug delivery company, has been awarded funding of EUR 1.25 million by the European Commission’s 7th Framework Programme as part of a EUR 5 million consortium project for the preclinical development of novel treatments for inherited retinal degenerative diseases. The consortium additionally consists of Coordinator University of Tübingen (Germany), SME Biolog (Germany), University of Modena and Reggio Emilia (Italy) and Lund University (Sweden).

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