Collaboration on the optimisation of macromolecular pharmaceutical access to cellular targets
Ongoing | IMI1 |
Collaboration on the optimisation of macromolecular pharmaceutical access to cel


Start Date
End Date
IMI1 - Call 4
Grant agreement number

Type of Action: 
RIA (Research and Innovation Action)

IMI Funding
10 184 909
EFPIA in kind
16 561 578
3 237 243
Total Cost
29 983 730


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

COMPACT paves way for development of better biopharmaceuticals
November 2016

Many new medicines are based on biological molecules such as proteins or oligonucleotides. Although promising, most of these new drugs (known as biopharmaceuticals) cannot cross biological membranes and are therefore limited in reaching targets within the cell. This has so far limited the number of effective biopharmaceuticals available.### A new toolbox of methods and formulations, developed by IMI’s COMPACT project, will soon give scientists a step-by-step strategy to quickly and reliably assess both the quantity and the quality of the drug delivered to the cell’s interior, opening the way for the development of more effective drugs in the future. ‘The uptake of drugs into cells is one of the most critical steps in the delivery of biopharmaceuticals over biological barriers and robust methods that can monitor individual steps of this process were sorely needed’, said COMPACT scientific coordinator, Enrico Mastrobattista of Utrecht University. ‘COMPACT has invested time and resources to make robust new methods available for all consortium members and in due time, when all the work has been published, to the entire scientific community.’ The development of the toolbox was a joint effort between the industrial and academic project partners and parts of it were already published in the Journal of Controlled Release. ‘This achievement illustrates how public-private partnerships can synergise efforts’, said Mastrobattista. ‘IMI has helped in bringing together and aligning experts from industry and academia and boosted the collaboration by giving a financial impulse. The combination of academic curiosity and industrial project management skills has led to a fruitful and productive collaboration on a scale that would not be achievable outside of an IMI Project.’ This achievement contributes to the overall goal of the COMPACT project, which is to shed new light on the obstacles biopharmaceuticals need to overcome to get to where they are needed in the body.

IMI scientist wins entrepreneurship award
February 2014

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.### 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.  Pharmacoidea is a partner in two IMI projects, COMPACT and AETIONOMY. 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.

Participants Show participants on map

EFPIA companies
  • Abbvie Deutschland GMBH & Co Kg, Wiesbaden, Germany
  • Boehringer Ingelheim Internationalgmbh, Ingelheim, Germany
  • Glaxosmithkline Research And Development LTD., Brentford, Middlesex, United Kingdom
  • Merck Kommanditgesellschaft Auf Aktien, Darmstadt, Germany
  • Novo Nordisk A/S, Bagsvaerd, Denmark
  • Pfizer Limited, Sandwich, Kent , United Kingdom
  • Sanofi-Aventis Deutschland GMBH, Frankfurt / Main, Germany
Universities, research organisations, public bodies, non-profit groups
  • Bioneer A/S, Horsholm, Denmark
  • Cardiff University, Cardiff, United Kingdom
  • Helmholtz-Zentrum Fuer Infektionsforschung GMBH, Braunschweig, Germany
  • Kobenhavns Universitet, Copenhagen, Denmark
  • Ludwig-Maximilians-Universitaet Muenchen, Munich, Germany
  • Norges teknisk-naturvitenskapelige universitet - NTNU, Trondheim, Norway
  • Stockholms Universitet, Stockholm, Sweden
  • Universitat Wien, Vienna, Austria
  • Universitat Zurich, Zürich, Switzerland
  • Universiteit Gent, Gent, Belgium
  • Universiteit Leiden, Leiden, Netherlands
  • Universiteit Utrecht, Utrecht, Netherlands
  • University of Helsinki, University of Helsinki, Helsinki, Finland
  • University of Oxford, Oxford, United Kingdom
Small and medium-sized enterprises (SMEs)
  • Pharmacoidea Fejleszto Es Szolgaltato Kft, Szeged, Hungary


Project coordinator
Ekkehard Leberer
Sanofi-Aventis Deutschland GmbH
+49 69 305 18998
Managing entity
Enrico Mastrobattista
Universiteit Utrecht