A frozen cell therapy could solve the supply-chain bottlenecks that currently force clinics to coordinate surgery schedules with customs clearance and quality-control release. The team has already developed implantable microcarriers—tiny scaffolds that hold muscle cells in their natural anchored state—and tested them in humans for treating faecal incontinence caused by childbirth injury. But the fresh product lasts only hours, making global shipping nearly impossible. This project will verify that freezing the cells on their microcarriers, then thawing them at the clinic, preserves their potency and safety. If successful, the approach turns a just-in-time manufacturing nightmare into a standard cold-chain product that can be stored, shipped, and used on demand. That removes a major barrier to scaling advanced therapies beyond single-centre trials. The same frozen-microcarrier platform could later be adapted for other cell-based treatments, making regenerative medicine more like ordering a vaccine than scheduling emergency surgery.
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Advanced therapies, notably those based on cell therapy or tissue-engineered medicinal products, have the potential to cure disease by addressing the root cause of the condition rather than treating the disease symptomatically. Such therapies often rely on centralized manufacturing and global distribution, requiring a durable supply chain that does not risk product functionality. We have developed a novel implantable microcarrier technology that enables transplantation of anchorage dependent cells in a more optimal anchored state. The microcarriers alone have undergone human clinical safety testing and are now being investigated for delivery of fresh autologous skeletal muscle derived cells for treatment of faecal incontinence (FI) as part of a multinational Phase I/IIb clinical study in patients with obstetric trauma-induced FI. The fresh tissue engineered combination product has a short shelf-life and supply chain logistics have highlighted significant risks caused by bottlenecks associated with Customs clearance, QP release, and matching day-of-delivery with end-user clinic schedules. To address the challenges associated with delivery of a fresh product, the project aims to verify use of cold-chain product supply for our planned Phase II study. Specifically, the objectives of the project are: Verify drug product integrity at different stages of the cold chain (cryopreservation, storage, transportation, and thawing); Demonstrate the proposed cold chain is not detrimental to the phenotypic traits and potency of SMDC and allows for adequate in-use shelf life when thawed. Manufacturing cells attached to implantable microcarriers and subsequent cryopreservation will increase the shelf-life of product and offer a ‘one-stop’ solution to manufacturing and shipping bottlenecks that exist, providing confidence for the clinical end-user. Our approach will disrupt current cell therapy manufacturing and remove just-in-time supply constraints associated with fresh products. A successful outcome will provide a ready to integrate cGMP process and associated technical development data for filing with the IMPD supporting its’ use in the next Phase of clinical testing for FI and in due course other clinical conditions, enabling the product to be shipped, received, and handled at the clinical sites in a robust and cost-effective manner, while being easy for end-users to adopt, ensuring safety, product quality, and stability.
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