APPROACH

Project Management and Coordination

University College London (UCL) will lead the scientific, contractual, and financial management of the project. This role will ensure the effective coordination of activities, maintaining high standards of quality and timely delivery of project milestones and outcomes.

It will also include oversight of risk management, ethical considerations, and gender equality principles throughout the project’s duration.

Cell-microcarrier manufacturing process

Led by Instituto Superior Técnico, this work package will develop a robust, reproducible and scalable manufacturing process that complies with Good Manufacturing Practice (GMP) standards for attaching human skeletal muscle-derived cells to thermally induced phase separation (TIPS) microcarriers. The standardised process will first be verified by University College London before being transferred to NHS Blood and Transplant, where it will undergo further validation to support the manufacture of the combined cell–microcarrier therapy.

Manufacture of clinically ready TIPS microcarriers

Thermally induced phase separation (TIPS) microcarriers will be manufactured at pilot scale for clinical application. University College London will oversee verification to ensure the microcarriers meet all predefined quality and performance specifications.
Stability studies will be undertaken to evaluate the long-term integrity and performance of the implantable cell–microcarrier product. In parallel, the manufacturing process will be comprehensively assessed to determine its suitability for scale-up and future commercial production.

Clinical trial plan and regulatory approval

During the trial set-up phase, the necessary regulatory and ethical approvals will be obtained in each participating country before patient recruitment begins, with overall coordination provided by University College London.
The clinical trial will evaluate whether implantation of autologous skeletal muscle-derived cell–microcarrier therapy leads to a clinically meaningful reduction in the frequency of faecal incontinence episodes.
Patients, carers and members of the public will play an active role throughout the project, contributing to both the delivery of the study and the dissemination of its findings.

Non-clinical safety studies required for regulatory approval

A programme of non-clinical safety studies will be undertaken to support regulatory approval of the therapy. These studies will characterise the biological behaviour of the cellular component within the final product and evaluate the local tissue response, persistence and toxicological profile of the combined cell–microcarrier therapy, providing the evidence needed to demonstrate its safety for clinical use.

Product stability verification

A robust and reproducible manufacturing process has been developed and validated to ensure the final product can be consistently prepared for storage and transport while maintaining its quality, stability and integrity.

Collaboration, Dissemination and Stakeholder Engagement

Bowel Research UK and PNO Innovation have led stakeholder engagement activities throughout the project, working with industry partners, healthcare professionals, patients and the public, charities, third-sector organisations and the academic community.
Collaboration has been central to the AMELIE project. Consortium members will continue to work closely with external partners to maximise the dissemination, impact and future exploitation of the project’s research and outcome

Knowledge Management and Exploitation

This work package will evaluate the commercial potential of the implantable cell–microcarrier technology for the treatment of faecal incontinence while identifying opportunities to apply the platform in other therapeutic areas where it could deliver clinical benefit.

Kathryn Pretzel-Shiels

CEO at Bowel Research UK

“Living with faecal incontinence can be deeply distressing and have a profound impact on quality of life. Because it is rarely discussed openly, many people also face stigma and isolation, making it even harder to cope. Finding an effective treatment would represent not only a major medical breakthrough but also a life-changing development for millions of people.”

Richard Day

Professor of Regenerative Medicine Technology at University College London

“We are tremendously excited by the prospect of the AMELIE project and the potential benefits that may arise from the new therapeutic approach being investigated.
We look forward to working with the exceptional consortium of academics, clinicians, industry and charity partners from across Europe to develop our pioneering regenerative medicine approach for treating this debilitating condition. This will be a radical and innovative approach never before attempted at such a scale.”