Accelerating research & development for advanced therapies


Advanced therapy medicinal products (ATMPs) such as cell and gene therapies have the potential to revolutionise treatments for many serious genetic diseases as well as some cancers. However, developing ATMPs is extremely challenging; in particular it is currently very difficult to know which patients will respond well to an advanced therapy and which will experience serious side effects.

The aim of ARDAT is to deliver the knowledge, tools and standards needed to speed up the development of ATMPs.

Among other things, the project will deliver tools and knowledge that will allow researchers to study how treatments are metabolised (i.e. broken down by the body); predict which treatments are likely to trigger a harmful immune response; and identify how best to deliver the therapy (e.g. via a drip / injection).

They also aim to deliver tools to assess the safety and efficacy of ATMPs, and to determine how many doses a patient will need to obtain a lasting improvement in their health.

Finally, ARDAT plans to work closely with regulators on regulatory harmonisation to accelerate ATMP product development.

ARDAT will focus on rare diseases caused by a single gene mutation. However, many of the project’s findings will be applicable to other diseases.

Achievements & News

Viruses can be hijacked to help cure rare diseases, but the immune system keeps fighting back

Advanced therapy medicinal products (ATMPs) such as cell and gene therapies could overhaul the way we treat genetic diseases, but it’s still extremely difficult to predict who will respond well to treatment and who will suffer serious side effects. IMI’s ARDAT project aims to answer some of the biggest questions surrounding ATMPs in a bid to get us closer to new treatments for rare diseases. ###

In an interview with the IMI Programme Office, the project leaders explain the many scientific and regulatory challenges facing the project, and how they plan to address them. If successful, the project could help to change the lives of people living with rare diseases.

‘The goal of gene and cell therapy is to provide, with a single treatment, sustained levels of therapeutic gene expression or cell activity, with potentially lifelong duration,’ they said. ‘For rare genetic diseases, this can provide a chance for patients to have a cure for their severe, debilitating disease state. It could free them from weekly intravenous infusions of clotting factor, free them from being confined to a wheelchair, or it could allow others to live who would otherwise would succumb to their disease.’

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EFPIA companies
  • Astellas Pharma Europe BV, Leiden, Netherlands
  • Bayer Aktiengesellschaft, Leverkusen, Germany
  • Janssen Pharmaceutica Nv, Beerse, Belgium
  • Lonza AG, Basel, Switzerland
  • Novartis Pharma AG, Basel, Switzerland
  • Novo Nordisk A/S, Bagsvaerd, Denmark
  • Pfizer Limited, Sandwich, Kent , United Kingdom
  • Sanofi-Aventis Recherche & Developpement, Chilly Mazarin, France
  • Spark Therapeutics, Inc, Philadelphia, United States
  • Takeda Pharmaceuticals International AG, Glattpark-Opfikon (Zurich), Switzerland
  • Viscofan SA, Tajonar, Spain
Universities, research organisations, public bodies, non-profit groups
  • Association Genethon, Evry, France
  • Centro De Neurociencias E Biologiacelular Associacao, Coimbra, Portugal
  • Commissariat A L Energie Atomique Et Aux Energies Alternatives, Paris, France
  • Institut Du Cerveau Et De La Moelle Epiniere, Paris, France
  • Institut National De La Sante Et De La Recherche Medicale, Paris, France
  • Itä-Suomen yliopisto, Kuopio, Finland
  • Lunds Universitet, Lund, Sweden
  • Medizinische Hochschule Hannover, Hannover, Germany
  • The Rosalind Franklin Institute, Didcot, United Kingdom
  • The University Of Liverpool, Liverpool, United Kingdom
  • The University Of Sheffield, Sheffield, United Kingdom
  • Universitatsklinikum Heidelberg, Heidelberg, Germany
  • University College London, London, United Kingdom
  • University of Cambridge, Cambridge, United Kingdom
  • University of Oxford, Oxford, United Kingdom
Small and medium-sized enterprises (SMEs) and mid-sized companies (<€500 m turnover)
  • Asphalion SL, Barcelona, Spain
  • Genosafe SAS, Evry, France
  • Instituto De Biologia Experimental E Tecnologica, Oeiras, Portugal
  • Lysogene, Neuilly sur Seine, France
  • Mimetas BV, Leiden, Netherlands
  • Pharmacoidea Fejleszto Es Szolgaltato Kft, Szeged, Hungary
  • Synvaccine LTD, Tel Aviv, Israel
  • Takis SRL, Roma, Italy

NameEU funding in €
Asphalion SL352 500
Association Genethon311 250
Centro De Neurociencias E Biologiacelular Associacao630 000
Commissariat A L Energie Atomique Et Aux Energies Alternatives245 026
Genosafe SAS299 658
Institut Du Cerveau Et De La Moelle Epiniere250 000
Institut National De La Sante Et De La Recherche Medicale298 518
Instituto De Biologia Experimental E Tecnologica400 000
Itä-Suomen yliopisto571 250
Lunds Universitet175 625
Lysogene450 000
Medizinische Hochschule Hannover177 719
Mimetas BV318 420
Pharmacoidea Fejleszto Es Szolgaltato Kft502 090
Synvaccine LTD229 000
Takis SRL225 063
The Rosalind Franklin Institute412 930
The University Of Liverpool1 000 000
The University Of Sheffield2 699 812
Universitatsklinikum Heidelberg300 000
University College London972 521
University of Cambridge324 740
University of Oxford626 879
Total Cost11 773 001