New €25.5 million European project to accelerate development of ATMPs

• The field of Advanced Therapy Medicinal Products research, which includes gene and cell therapies, is expected to grow exponentially in the coming years, with potentially up to 10-20 new drug applications submitted per year to the FDA by 2025

• The consortium brings together the leading expertise of 34 academic, nonprofit and private organisations

• Consortium will help to fill the knowledge gaps in how advanced therapies could potentially work, and to develop appropriate standards to aid researchers, developers and regulators in accelerating effective and safe gene and cell therapies to benefit patients

The University of Sheffield and Pfizer have today (23 November 2020) launched a new consortium which aims to standardize and accelerate the development of Advanced Therapy Medicinal Products (ATMPs), allowing potentially transformative treatments to reach patients sooner.

The new, five-year consortium, Accelerating Research and Innovation for Advanced Therapies (ARDAT), is supported by the Innovative Medicines Initiative (IMI) and brings together the leading expertise of 34 academic, nonprofit and private organisations from across Europe and the US.

Led by ARDAT Coordinator, Professor Mimoun Azzouz from the University of Sheffield, and ARDAT Project Lead, Dr Gregory LaRosa from Pfizer, the new consortium will pioneer a €25.5 million project to accelerate the development of ATMPs which includes gene and cell therapies.

The field of ATMP research is expected to grow exponentially in the coming years, with potentially up to 10-20 new drug applications submitted per year to the FDA (US Food and Drug Administration) by 2025.

The ARDAT consortium will aim to bring together researchers from public and private organisations to help fill the knowledge gaps in how these therapies could potentially work, and to develop appropriate standards to aid researchers, developers and regulators in accelerating effective and safe gene and cell therapies to benefit patients.

Professor Mimoun Azzouz, Chair of Translational Neuroscience and Director of Research and Innovation at the University of Sheffield’s Institute for Translational Neuroscience (SITraN) and ARDAT Coordinator, said: “This is a significant development expected to change the landscape of research, innovation and regulatory activities for cell and gene therapies.

“We are very excited to bring together world-leading experts to accelerate delivery of advanced therapies to patients suffering from rare diseases.”

The consortium aims to develop standardized models for predicting ATMP immunogenicity in humans; build understanding of ATMP drug metabolism within a host; identify adaptive immune responses that could affect ATMP safety, efficacy and persistence; and engage regulators to help support filings that address standardized regulatory, safety and efficacy concerns.

“While still an emerging field, ATMP research has largely been fragmented and siloed within organisations with little opportunity to share best practices and information,” said Dr Greg LaRosa, Head of Scientific Research, Rare Disease Research Unit at Pfizer.

“As gene and cell therapies research grows and more potential ATMPs move into later-stage clinical trials, it is in the interest of the industry and of patients to further our collective understanding of their mechanisms by sharing data and regulatory expertise.”

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Notes to editors

About ARDAT

The ARDAT project is a precompetitive €25.5M, five-year consortium that brings together the leading expertise of 34 academic, nonprofit, and private organizations, with the shared goal of helping to standardize and accelerate development of Advanced Therapy Medicinal Products (ATMPs) and potentially helping to bring these transformative treatments to patients sooner.

For more information on ARDAT, visit

http://www.

ardat.

org

About the IMI

The IMI is Europe’s largest public-private initiative aiming to speed up the development of better and safer medicines for patients. IMI supports collaborative research projects and builds networks of patients, industrial and academic experts in order to boost pharmaceutical innovation in Europe. IMI is a joint undertaking between the European Union and the European Federation of Pharmaceutical Industries and Associations (EFPIA).

For further details please visit:

http://imi.

europa.

eu/

Acknowledgement

This project has received funding from the Innovative Medicines Initiative 2 Joint Undertaking under grant agreement No [945473]. This Joint Undertaking receives support from the European Union’s Horizon 2020 research and innovation programme and EFPIA.

Disclaimer

This communication reflects the views of the authors and neither the IMI nor the European Union, EFPIA or any other partners are liable for any use that may be made of the information contained herein.

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