ARDAT General Assembly, Coimbra, Portugal 15/16 June 2023

The third face to face meeting for members of the ARDAT consortium was kindly hosted by the team from the Center for Neuroscience and Cell Biology (CNC) based in the beautiful city of Coimbra in central Portugal. Over 50 individuals from across the consortium met over two days to receive progress updates and discuss progress and future for the four key work packages. The meeting was once again set up as a real time hybrid format so that members who were unable to travel to Coimbra could follow the proceedings and indeed, present their own work to the Assembly.

After a full day of presentations and discussion the meeting moved to the spectacular hall of the Colegio da Trindade to listen to invited speaker Jude Samulski give an entertaining and thought provoking talk on the history of gene therapies, highlighting the challenges and learning that has taken place over the past 50 years.

After the day’s formal proceedings the Assembly decamped to a local restaurant for the Meeting Dinner where discussions continued in a more relaxed environment. It was great to see the professional and personal bonds between individuals and groups developing.

As before, the second day was given over to breakout meetings where the different work package teams could sit down and engage in a thorough review of progress, roadblocks, issues and mitigation, very important ahead of the Project’s Mid-point Review on the 29th June.

In summary, this was another successful meeting where it was obvious that real strides are being taken to achieving the objectives of the ARDAT project.

 

The plenary session underway with remote presenters contributing via video link

Programme leader Mimoun Azzouz opening the meeting

The participants in the Third ARDAT General Assembly

ARDAT team members socialising at the Meeting Dinner

Jude Samulski Presenting his talk in the Colegio da Trindade

ARDAT General Assembly, Edinburgh UK 16/17 October 2022

Although the ARDAT Project has been running for almost 2 years, because of the restrictions placed upon travel and meeting throughout the COVID pandemic, all interactions between  the project partners had been through various forms of  videoconferencing. The General Assembly 2022 was therefore the first opportunity that most participants had to meet face to face. The Meeting was scheduled to happen immediately  after the European Society of Gene and Cell Therapy Meeting in Edinburgh so that the many ARDAT members who were attending that meeting could stay on to attend the General Assembly.
After an informal get together the night before where everyone was required to “guess the oher ARDAT member”  the Meeting proper started on the Morning of 16th October with 50 ARDAT members convening together for the first time. To allow full participation, the meeting was run in  a hybrid real-time/virtual format, allowing members who could not physically attend to see and interact as though they were in the room. Thanks to the team at Video Production Edinburgh for providing a seamless hybrid meeting service over both days of the meeting.
The first days proceedings were kicked off by a welcome from Greg LaRosa (Project Lead) and Mimoun Azzouz (Project Coordinator) who then gave way for a full agenda of presentations and discussions on the work carried out in the various work-packages over the preceding 2 years. For many, it was the first opportunity to engage with members outside of their work-package groups and by the end of the day many new alliances started to form. The first day was concluded by a Meeting Dinner  where people could continue their conversations in a more relaxed environment.
The second day was given over to round table break-out groups split by work package to allow an uninterrupted discussion on the progress and future direction of the Tasks and Sub-Tasks, the face to face format allowing real progress to be made on many of the ongoing issues.
All in all, the meeting was a great success and helped to bring together some very good and productive work streams as the project enters its third year.

 


One of the many useful discussion sessions


Manuela Braun (Bayer) presenting on assay development strategies


Chris Mann (Asphalion) explaining the developing relationships with Regulatory Agencies pursued by WP5


Mark Trautwein (Bayer) presenting an overview of activities in WP2

ARDAT Publishes its first Journal Article

Biodistribution, shedding and persistence of gene/cell therapies was identified by ARDAT WP5 partners as a key area for the focus of regulatory and harmonisation activities in the ARDAT consortium. Despite the growing body of published research characterising biodistribution, persistence and shedding of ATMPs, a lack of common methodological standards and reagents to assess the presence and persistence of gene and cell-based therapies was identified as a key issue that could benefit from further regulatory harmonisation.

A multidisciplinary team from ARDAT have therefore collaborated on a regulatory landscape assessment in order to a) identify regional differences in regulation and b) identify opportunities for harmonisation and where new recommendations / standards may be proposed with regard to biodistribution, shedding and persistence.

A number of information sources were consulted as part of the landscape assessment and used to identify a preliminary list of issues that may benefit from regulatory harmonisation. The key sources were:

  • Existing international guidelines (in particular EMA, FDA and ICH, with other relevant guidance from international sources consulted where appropriate).
  • Feedback obtained from consortium partners from regulatory consultations (e.g., scientific advice) with EMA and other competent authorities.
  • Public assessments of approved, withdrawn and rejected ATMPs.

After conducting the landscape assessment, a WP5 sub-team was set up to draft and publish a  Whitepaper utilising the information collated from the landscape assessment and encompassing the following aspects:

  •  A comparative evaluation of global regulatory expectations for biodistribution, shedding (including environmental risk assessment) and bioanalytical methods.
  • A description of methodological aspects for the construction of the database designed to collect published data on biodistribution and shedding.
  • Use of the database to identify and discuss reporting inconsistencies across the published literature with regard to biodistribution and associated bioanalytical methods.

As the database  has to date only collected data from AAV-based therapies, it was decided to focus on AAV-based therapies in the first Whitepaper. A preliminary analysis of the most relevant database outputs from the perspective of data-driven regulatory support in the context of AAV-based therapies was included in the Whitepaper. This analysis will inform similar approaches to other gene and cell-based therapies in the future and potential future articles / whitepapers from the consortium.

The finalised Whitepaper, entitled “Current global regulatory landscape for biodistribution & shedding assessment of rAAV gene therapies & recommendations of the IMI ARDAT consortium on future directions” was submitted for review at the journal “Cell & Gene Therapy Insights” on 18th March 2022 and was published on 26th April 2022.

Please follow this link to access the publication.

New Consortium aims to Standardize and Accelerate Development of Advanced Therapy Medicinal Products in €25.5 million project

Accelerating Research and Innovation for Advanced Therapies (ARDAT), a new, five-year European consortium supported by the Innovative Medicines Initiative (IMI), today announces its launch. The precompetitive consortium brings together the leading expertise of 34 academic, nonprofit and private organizations, led by Pfizer (ARDAT Project Lead: Dr Gregory LaRosa) and the University Of Sheffield (ARDAT Coordinator: Professor Mimoun Azzouz), and experts from across Europe and the US 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.

The field of ATMP 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 ARDAT consortium will aim to bring together researchers from public and private organizations 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.

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

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.

“We are very excited to bring together world leading experts to accelerate delivery of advanced therapies to patients suffering from rare diseases”, said Professor Mimoun Azzouz, Chair of Translational Neuroscience, Director of Research and Innovation at the University of Sheffield’s Institute of Translational Neuroscience (SITraN) and ARDAT Coordinator. “This is an significant development expected to change the landscape of research, innovation and regulatory activities for cell and gene therapies”

# # #

About ARDAT

The ARDAT project is a precompetitive €25.5M, 5 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  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.

Contacts:

Pfizer Media Relations                                University of Sheffield Media Team

Francesca Russo                                                      Amy Huxtable
Pfizer Uk                                                                   Media Relations Officer
+44 (0) 7920 548118                                                 +44(0)114 222 9859
pressofficeuk@pfizer.com                                         a.l.huxtable@sheffield.ac.uk

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