New consortium to accelerate development of Advanced Therapy Medicinal Products

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The University of Liverpool is part of 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 the University of Sheffield and 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.

This is a significant development expected to change the landscape of research, innovation and regulatory activities for cell and gene therapies.

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.

Investigators at Liverpool provide the programme with an in depth knowledge in drug safety and immunogenicity. They will develop better, standardised models for predicting product immunogenicity in humans, and aim to understand the clinical factors around pre-existing immunity limiting patient access to ATMP therapy and adaptive immune responses affecting product safety, efficacy and persistence.

Professor Sir Munir Pirmohamed, Director of the MRC Centre for Drug Safety Science, said: “We are excited to be involved in this IMI programme which is aiming to advance cutting-edge advanced therapy medicinal products for rare diseases from bench to bedside. Our role in the MRC Centre for Drug Safety Science will be to understand the immune response to these advanced therapies which can limit their effectiveness and sometimes lead to adverse reactions. Collaborations with partners from all across Europe will allow work at scale that will provide novel insights to optimise advanced therapies for the benefit of 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.”