Summary
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
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.’
Find out more
- Read the interview in full
Participants
Show participants on mapEFPIA 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
| Participants | |
|---|---|
| Name | EU funding in € |
| Asphalion SL | 352 500 |
| Association Genethon | 311 250 |
| Centro De Neurociencias E Biologiacelular Associacao | 630 000 |
| Commissariat A L Energie Atomique Et Aux Energies Alternatives | 245 026 |
| Genosafe SAS | 299 658 |
| Institut Du Cerveau Et De La Moelle Epiniere | 250 000 |
| Institut National De La Sante Et De La Recherche Medicale | 298 518 |
| Instituto De Biologia Experimental E Tecnologica | 400 000 |
| Itä-Suomen yliopisto | 571 250 |
| Lunds Universitet | 175 625 |
| Lysogene | 450 000 |
| Medizinische Hochschule Hannover | 177 719 |
| Mimetas BV | 318 420 |
| Pharmacoidea Fejleszto Es Szolgaltato Kft | 502 090 |
| Synvaccine LTD | 229 000 |
| Takis SRL | 225 063 |
| The Rosalind Franklin Institute | 412 930 |
| The University Of Liverpool | 1 000 000 |
| The University Of Sheffield | 2 699 812 |
| Universitatsklinikum Heidelberg | 300 000 |
| University College London | 972 521 |
| University of Cambridge | 324 740 |
| University of Oxford | 626 879 |
| Total Cost | 11 773 001 |
Mimoun Azzouz
The University Of Sheffield