Manchester – Confidence in Concept 2019
Lead Research Organisation:
University of Manchester
Department Name: UNLISTED
Abstract
Abstracts are not currently available in GtR for all funded research. This is normally because the abstract was not required at the time of proposal submission, but may be because it included sensitive information such as personal details.
Technical Summary
The Confidence in Concept (CIC) scheme is a key part of MRC’s translational research strategy and provides annual awards to institutions to flexibly support a portfolio of early stage translational research projects. CIC is designed to accelerate the transition from discovery research to viable translational projects by supporting preliminary studies to establish proof-of-concept so that the approach will then become competitive for more substantial translational funding.
People |
ORCID iD |
| Luke Georghiou (Principal Investigator) |
Publications
Ahmad G
(2023)
Prostaglandin F2a and Endometriosis: An Ongoing Pain
in Journal of Minimally Invasive Gynecology
Ali SM
(2023)
Feasibility and acceptability to use a smartphone-based manikin for daily longitudinal self-reporting of chronic pain.
in Digital health
Ali SM
(2023)
Exploring the Cross-cultural Acceptability of Digital Tools for Pain Self-reporting: Qualitative Study.
in JMIR human factors
Gonçalves C
(2024)
Modulation of circadian rhythms in articular cartilage by heat pulses
Horgan C
(2022)
Current and Future Treatment of Mucopolysaccharidosis (MPS) Type II: Is Brain-Targeted Stem Cell Gene Therapy the Solution for This Devastating Disorder?
in International journal of molecular sciences
McMorrow LA
(2022)
Advancing Our Understanding of the Chronically Denervated Schwann Cell: A Potential Therapeutic Target?
in Biomolecules
Smith TAD
(2022)
Comparison of multiple gene expression platforms for measuring a bladder cancer hypoxia signature.
in Molecular medicine reports
Van Der Veer SN
(2024)
Reliability, validity, and responsiveness of a smartphone-based manikin to support pain self-reporting.
in Pain reports
| Description | Immune-privileged, immortal, myogenic stem cells for gene therapy of Muscular Dystrophy. (UniMab) |
| Amount | € 2,345,625 (EUR) |
| Funding ID | 884952 |
| Organisation | European Research Council (ERC) |
| Sector | Public |
| Country | Belgium |
| Start | 01/2021 |
| End | 12/2025 |
| Description | Wellcome Trust A2E |
| Amount | £25,000 (GBP) |
| Organisation | Wellcome Trust |
| Sector | Charity/Non Profit |
| Country | United Kingdom |
| Start | |
| Description | AvroBio |
| Organisation | AVROBIO |
| Country | United States |
| Sector | Private |
| PI Contribution | MPSII (Hunter syndrome) is an X-linked recessive condition that affects children, predominantly males with female relatives being carriers. Traditionally, MPSII has been classified as either attenuated or severe, with both classifications including skeletal abnormalities, stiff joints, short stature, hepatosplenomegaly and cardiorespiratory problems. About 2/3 of patients with MPSII also display progressive neurocognitive dysfunction. The incidence of MPSII is roughly 1.3 per 100,000 live births in Europe and there are currently no curative therapies. In our lab we have developed a gene therapy strategy to treat MPSII. The work done includes a histopathology study undertaken by a third party company which has demonstrated the safety of the vector as well as a more widespread correction of organs such as the liver, kidney, heart and retina. The large-scale transduction work has been completed, showing efficacy and safety of the MPSII product when transducing human CD34+ haematopoietic stem cells. |
| Collaborator Contribution | We have since signed a £67M licence deal with AvroBio and a £9.1M clinical trial grant (Bigger PI) to progress this research to a phase I/II clinical trial clinical trial. In a separate development the large scale transduction work has been funded on the iMATCH extension project (Bigger CoI) to be translated into a GMP manufacturing solution in collaboration with NHSBT - that we hope will provide GMP manufacturing capability for advanced therapy medicinal products for phase I/II clinical trials in the north of England. |
| Impact | We have since signed a £67M licence deal with AvroBio and a £9.1M clinical trial grant (Bigger PI) to progress this research to a phase I/II clinical trial clinical trial. In a separate development the large scale transduction work has been funded on the iMATCH extension project (Bigger CoI) to be translated into a GMP manufacturing solution in collaboration with NHSBT - that we hope will provide GMP manufacturing capability for advanced therapy medicinal products for phase I/II clinical trials in the north of England. |
| Start Year | 2020 |
| Description | Royce/TATA Steel Collaboration |
| Organisation | Henry Royce Institute |
| Country | United Kingdom |
| Sector | Academic/University |
| PI Contribution | The global 3D cell culture market has been estimated to exceed the billion USD target and is predicted to grow annually in double digits over the next 5-10 years. Currently, animal-derived materials have the highest share of this market but synthetic or biotechnological materials are predicted the fastest growth due to increasing demand for animal-free products. Combination of our recombinant collagen technology with self-assembling hydrogels can provide tuneable cell-culture substrates with adjustable composition and stiffness to mimic a wide range of tissues and to better control stem cell phenotype, maintenance and differentiation. These collagen-modified hydrogels have the potential for easy and cost-effective production of biomaterials for tissue engineering and regenerative medicine. Our recombinant collagen technology can overcome some of the problems of animal-derived collagens associated with zoonotic risks, sample heterogeneity and degradation or limited engineering capability, or the difficulties in collagen-like peptide synthesis. We have already encountered instances where the synthesis of some designed collagen-like peptides has proven too challenging for peptide manufacturers, whereas recombinant versions of the same designs will be easy to produce in house with our technology. Additionally, we can manufacture recombinant collagens with sequences that do not occur in nature, much longer that what can be achieved currently via chemical peptide synthesis. These engineered collagens can lead to novel applications in tissue regeneration and potentially. As an indirect benefit, our protocols of hydrogel modification with collagens will be applicable to the design of hydrogels incorporating antimicrobial peptides, which could be particularly useful for wound healing applications. |
| Collaborator Contribution | As a follow-up of this research we have been successful in producing recombinant mini-collagens with enhanced thermal stability, with novel characteristics, and with potential antimicrobial activity. These mini-collagens have designed sequences (not existing in nature) and are effective cell adhesion substrates. They represent novel IP which could be potentially protected or licenced by the University. We have pending a meeting with University of Manchester Innovation Factory to discuss the new IP and to explore if there are potential avenues for commercial development. |
| Impact | Royce / TATA Steel collaboration. This is a research collaboration between the Royce Institute at the University of Manchester, University of Sheffield, Imperial College London, and University of Cambridge. I am one of the named collaborators. Details of the agreement still being finalised. |
| Start Year | 2023 |
| Description | iMATCH |
| Organisation | Innovate UK |
| Country | United Kingdom |
| Sector | Public |
| PI Contribution | This project has built upon our pre-clinical data in the MPSII mouse model and takes the treatment a step closer to the clinic. We now have funding through Avrobio to run a clinical trial in 21/22 for 5 patients with MPSII. This treatment could revolutionise the treatment of MPSII. |
| Collaborator Contribution | as aboe |
| Impact | publications |
| Start Year | 2021 |
| Title | GUIDANCE SYSTEM |
| Description | A guidance system for guiding a clinician in delivering a needle to a target location within a hollow organ in a patient's body. The guidance system includes: a computing device; a target sensor configured to provide target sensor data describing the position of the target sensor, wherein the target sensor is configured to be positioned at or proximate to the target location within the hollow organ; a needle sensor configured to be carried by the needle or a device on which the needle is mounted, and to provide needle sensor data describing the position and orientation of the needle; at least one information delivery device configured to deliver information to the clinician. The computing device is configured to, using the target sensor data and needle sensor data, compute targeting information describing at least the orientation of the needle relative to the target location, and to use the at least one information delivery device to deliver guidance information to the clinician for assisting the clinician in delivering the needle to the target location, based on the targeting information |
| IP Reference | WO2023285826 |
| Protection | Patent / Patent application |
| Year Protection Granted | 2023 |
| Licensed | No |
| Impact | This research demonstrates a clinically significant benefit that can be realised through further research in terms of patient outcomes, length of stay within the ICU and reduced healthcare costs. |
| Company Name | Polynerve |
| Description | Polynerve develops a synthetic nerve conduit for use in nerve graft surgeries. |
| Year Established | 2022 |
| Impact | Currently applying for funding |
| Website | http://polynerve.com |