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Collaboration on the optimisation of macromolecular pharmaceutical access to cellular targets


Many new medicines are based on biological molecules such as proteins, peptides or nucleic acids. The goal of the COMPACT project is to shed new light on the obstacles these drugs (which are known as biopharmaceuticals) need to overcome to get to where they are needed in the body. The team will then use this information to develop and validate biopharmaceutical formulations to deliver these novel drugs to their targets.


New drugs with a lot of potential

Biopharmaceuticals, medicines based on biological molecules such as proteins and nucleic acids, have already delivered effective treatments for a number of serious, often hard to treat diseases, such as Crohn’s disease and multiple sclerosis, dramatically improving patients’ quality of life.

The pharmaceutical industry is keen to expand its work on these novel drugs, but biopharmaceuticals still have a number of drawbacks which are hampering their broader application. For example, because these molecules tend to be complex and delicate, most biopharmaceuticals have to be injected; if they were administered orally (a more patient-friendly route), they would be destroyed by the harsh environment of the stomach. Furthermore, even once biopharmaceuticals are in the body, their large size means it is hard for them to get to their targets.

Overcoming obstacles

The overall aim of the COMPACT project is to overcome the delivery and targeting bottlenecks for biopharmaceuticals. It will do this by tackling a number of key issues. Firstly, it will identify and characterise the main transport routes across biological barriers and through cell membranes that could be exploited for drug delivery purposes. These include the intestinal barrier, skin barrier, and blood-brain barrier. 

Secondly, the researchers will devise and characterise formulations to allow the delivery of peptide and protein-based drugs via non-invasive routes (e.g. orally, via the airways, and via the skin). The team will also work to find ways to get these drugs across the blood-brain barrier. 

Another goal involves transporting drugs based on nucleic acids (e.g. RNA) into and through the cell. Throughout the project, the team will use advanced imaging techniques to track the movement of biopharmaceuticals at the (sub) cellular, tissue, and whole body level.

Benefits for patients

Biopharmaceuticals have the potential to improve the lives of many patients with diseases and conditions that are currently hard or even impossible to treat. By finding more effective ways of administering these drugs, and improving their ability to travel through the body to where they are needed, COMPACT will allow more patients to benefit from biopharmaceuticals. Furthermore, designing less invasive administration routes and reducing the dose (and therefore the side effects) and frequency of administration will help to improve patient compliance with treatments.


Achievements & News

IMI scientist wins entrepreneurship award
The 2013 award for Hungarian Young Entrepreneur of the Year was won by Tamas Letoha, Chief Executive Officer of Pharmacoidea, which is a partner in two IMI projects: COMPACT and AETIONOMY. Tamas was selected as the winner from over ###400 public nominations from more than 50 small and medium-sized enterprises (SMEs) from a number of different business sectors. Dr Letoha, a medical researcher by training, received his award from the Hungarian Prime Minister, Viktor Orbán at the Role Model of the Year Award gala in January. The annual award is sponsored by the Role Model Foundation which was set up in 2013 to recognise successful Hungarian entrepreneurs under the age of 40. Tamas heads up Pharmacoidea Ltd. which specialises in R&D in drug discovery, functional food development and experimental cellular therapeutics against carcinomas. Tamas said that his achievements ‘were pretty much due to international R&D projects like AETIONOMY ’ and that he highly valued his connections with IMI.
(February 2014)



  • Sanofi-Aventis Deutschland GmbH, Frankfurt am Main, Germany
  • Abbott GmbH & CoKG, Wiesbaden-Delkenheim, Germany
  • Boehringer Ingelheim International GmbH, Ingelheim, Germany
  • GlaxoSmithKline Research and Development Ltd, Brentford, UK
  • Merck KGaA, Darmstadt, Germany
  • Novo Nordisk A/S, Bagsværd, Denmark
  • Pfizer Limited, Sandwich, UK

Universities, research organisations, public bodies, non-profit groups

  • Universiteit Utrecht, Utrecht, Netherlands
  • Bioneer A/S, Hørsholm, Denmark
  • Cardiff University, Cardiff, UK
  • Helmholtz-Zentrum für Infektionsforschung GmbH, Braunschweig, Germany
  • Helsingin Yliopisto, Helsinki, Finland
  • Ludwig-Maximilians-Universität München, Munich, Germany
  • Norges Teknisk-Naturvitenskapelige Universitet NTNU, Trondheim, Norway
  • Stockholms Universitet, Stockholm, Sweden
  • Universität Wien, Vienna, Austria
  • Universität Zurich, Zurich, Switzerland
  • Universiteit Gent, Ghent, Belgium
  • Universiteit Leiden, Leiden, Netherlands
  • University of Copenhagen, Copenhagen, Denmark
  • University of Oxford, Oxford, UK


  • Pharmacoidea Development and Service Ltd, Szeged, Hungary

Facts & Figures

Start date 01/11/2012
End Date 31/10/2017
Contributions  €
IMI funding 10 184 909
EFPIA in kind 18 217 735
Other   3 237 056
Total Cost 31 639 700


Project Coordinator
Ekkehard Leberer

Sanofi-Aventis Deutschland GmbH
Tel: +49 69 305 18998
E-mail: Ekkehard.Leberer [AT]

Managing Entity
Enrico Mastrobattista

Universiteit Utrecht
Tel: +31 6 22736567
E-mail: e.mastrobattista [AT]