NIHRDataInsights/HRCSData huggingface dataset:
Title: siRNA therapy in dominant skin and eye disorders
Funder: Medical Research Council

Abstract: Despite the progress in identification of genes causing human disease, development of therapies tailored to these genetic defects has been disappointingly slow. RNAi represents a promising new means of silencing genes or mutant alleles, particularly in dominant conditions, however, a major hurdle is delivery of these agents in vivo. The palm/sole epidermis and the ocular surface epithelium are two very accessible target tissues of limited size, with obvious advantages for development of RNAi therapy. Here, we will perform identification and lead optimisation of gene-specific and mutation-specific siRNA inhibitors for keratin disorders of palmoplantar epidermis and of cornea. We will systematically compare the efficacy, specificity and stability of alternative methods of gene-specific and allele-specific silencing including morpholino oligonucleotides, locked nucleic acids (LNA) and peptide nucleic acids (PNA), relative to optimised lead siRNA inhibitors. We will develop a novel multi-target reporter gene mouse where a bioluminescence signal is targeted to footpad epidermis as a preclinical model for epidermal keratin disorders. This will be complemented by a phenotypic model of footpad hyperkeratosis. We will develop an analogous multi-target bioluminescent reporter gene mouse and a phenotypic corneal dystrophy mouse as preclinical models of a corneal keratin disorder. We will develop and optimise non-invasive methods for siRNA delivery into the skin and into the corneal epithelium, using the appropriate animal models generated. Using two different, complementary target tissues increases the chances of successful translation of this technology into human application. Solving the siRNA delivery problem opens up this technology for treatment of a wide range of incurable genetic diseases that collectively amount to a massive healthcare burden.


Research activity codes: 5.2 CELLULAR AND GENE THERAPIES
Health categories: EYE;SKIN
Title: E-Health informatics Research Centres (E-HIRC)
Funder: Arthritis Research UK

Abstract: The E-HIRC Call is to establish centres of excellence in research using e-health records as part of a shared vision, by major funders of informatics research, to promote linkage of electronic data for research


Research activity codes: 2.6 RESOURCES AND INFRASTRUCTURE (AETIOLOGY);6.9 RESOURCES AND INFRASTRUCTURE (TREATMENT EVALUATION);8.5 RESOURCES AND INFRASTRUCTURE (HEALTH SERVICES);7.4 RESOURCES AND INFRASTRUCTURE (DISEASE MANAGEMENT)
Health categories: GENERIC HEALTH RELEVANCE
Title: British Regen Industry Tool Set (BRITS)
Funder: Innovate UK

Abstract: The British Regen Industry Tool Set (BRITS) is an industry driven project aimed at establishing reliable market data and creating both detailed bioprocess economics models and higher level business models for integration into a highly valuable and timely set of decisional tools. The individual components will themselves be highly novel, and the final integrated tool set will be a major step change for the cell therapy industry. BRITS will encompass the entire supply chain. This will including direct links through the main innovation routes available within the NHS. Via its wider business benefit activities BRITS will interact with all the UK stakeholders to facilitate the uptake of the outputs of the project and build and maintain the vital linkages between the diverse stakeholders in order to promote the joined-up approach that will be required if the UK is to be at the international forefront of cell therapy, not just scientifically but also commercially.


Research activity codes: 8.5 RESOURCES AND INFRASTRUCTURE (HEALTH SERVICES);5.9 RESOURCES AND INFRASTRUCTURE (TREATMENT DEVELOPMENT)
Health categories: GENERIC HEALTH RELEVANCE
Title: CPrep - Closed point-of-care-Preparation Device
Funder: Innovate UK

Abstract: This project will establish a world-class consortium to innovatively address a critical step in administering cellular products for the cell therapy industry (CTI) – a robust validated platform technology to enable post-manufacturing process steps to be carried out at the point of care (PoC). This Closed-point-of-care-Preparation device will increase patient safety and enable a new generation of cell therapies that would otherwise have to be shipped fresh with its inherent logistics problems This will address every step after a cell therapy leaves the central GMP facility through to the completion of the loading of the clinical implantation device at the PoC. Presently no such integrated closed PoC technology exists. Requirement for such a device is well recognised by CTI.


Research activity codes: 5.2 CELLULAR AND GENE THERAPIES;5.3 MEDICAL DEVICES
Health categories: GENERIC HEALTH RELEVANCE
Title: Enhanced Cell Stability during manufacture and administration
Funder: Innovate UK

Abstract: Effective and safe manufacture of human cells for therapy requires application of multiple-input, Quality by Design principles throughout the whole process, from cell sourcing through expansion to patient. Particularly important are hold stages which are essential for flexibility, the poor specification of which will be the cause of process failures and loss of cell specification on application to the patient.The project objective is the creation and testing of a new technology toolset which will integrate the novel commercial formulation skills of Stabilitech with proven microscale process discovery skills of UCL to help regenerative medicine companies discover at an early stage, using small quantities of precious cells at lower R&D costs, innovative ways in which cells may be held so that both manufacture and administration to the patient meet the current regulatory and economic challenges.


Research activity codes: 5.2 CELLULAR AND GENE THERAPIES
Health categories: GENERIC HEALTH RELEVANCE
Title: High Throughput Platform for the Discovery of GMP Compatible Stem Cell Manufacturing Protocols
Funder: Innovate UK

Abstract: The manufacture of cells in a controlled and reproducible manner is a critical challenge in regenerative medicine. We will develop an integrated platform technology for the rapid identification of novel, efficient stem cell expansion and differentiation protocols that are optimised for translation to clinical grade manufacturing. Several innovative technologies will be combined to perform high throughput GMP compatible screens to discover novel cell production protocols. These protocols will be transferred to a scaled-up bioprocess where a novel cell imaging technology will be utilised to monitor cell performance. Successful protocols will be translated to a GMP manufacturing process for final validation. In this way, safe, robust, cost-effective GMP validated protocols will be rapidly discovered, greatly reducing the cost and time for development of regenerative cell therapeutics.


Research activity codes: 5.2 CELLULAR AND GENE THERAPIES
Health categories: GENERIC HEALTH RELEVANCE
Title: Cell labelling for in vivo use and in process quality control
Funder: Innovate UK

Abstract: The consortium of Altrika, the University of Edinburgh’s School of Chemistry, Roslin Cellab and Barts and The London bring R&D, manufacturing, clinical and commercialisation experience to the challenge of tracking clinically relevant cell populations in vitro and in vivo. The absence of an in vivo tracking methodology leads to R&D, manufacturing and clinical constraints, valuable researcher time and cell yield losses, and associated costs. This project utilises an innovative, non-toxic cell label that is stable, cost-effective and easy to detect. It will demonstrate applicability to a range of regenerative medicine therapies for unmet clinical needs, as well as increasing the efficiency of R&D activities and manufacturing processes, and improving the efficacy of clinical delivery. Given the growth of regenerative medicine, this platform could reduce industry costs by as much as $275m in 2020.


Research activity codes: 5.2 CELLULAR AND GENE THERAPIES
Health categories: GENERIC HEALTH RELEVANCE
Title: Development of a GMP cryogenic cold chain for clinical delivery of regenerative medicine therapeutics
Funder: Innovate UK

Abstract: Asymptote Ltd is proposing to develop the first GMP compliant cryogenic cold chain for the clinical delivery of regenerative medicine therapeutics. This project focuses on the key elements of the cold chain: controlled rate freezing of large volumes, short and long term frozen storage, and controlled thawing of large volumes. Cryopreservation of cells with high functionality on thawing is essential for cost effective cell production and geographically widespread clinical treatments, and this depends on accurate control of the freezing and thawing. This project will use Stirling cryocooler technology which has unique control ability and is able to meet the GMP requirement of regenerative medicine. It is intended to produce demonstrators for the key elements of the cold chain in collaboration with four centres developing different cell therapies.


Research activity codes: 1.3 CHEMICAL AND PHYSICAL SCIENCES;5.2 CELLULAR AND GENE THERAPIES
Health categories: GENERIC HEALTH RELEVANCE
Title: Expandable Clinical Grade Human Feeder Cells for hESc Derivation
Funder: Innovate UK

Abstract: We aim to generate an inexhaustible supply of immortalised clinical grade human dermal fibroblasts (hDFs) for derivation of cGMP hESc lines. Human Embryonic stem cells (hESc) have great potential for use in clinical therapies but current hESc lines have not been generated under conditions suitable for clinical use. We will exploit our successful TSB-funded feasibility study to generate suitable immortalised human dermal fibroblasts (hDFs) for derivation of cGMP hESc lines. We will immortalise 2 primary (clinical grade) hDF lines, already shown to support culture and derivation of cGMP hESc lines in a unique media system, using a method shown by us to generate hESc-supportive feeder cells at the research level. The immortalised cells will be tested for derivation of new cGMP hESc lines and will provide a supply of marketable cGMP feeder cells for use in generating hESc for therapy.


Research activity codes: 5.2 CELLULAR AND GENE THERAPIES
Health categories: GENERIC HEALTH RELEVANCE
Title: Novel excipients to allow superior liquid antibody formulation at high concentration
Funder: Innovate UK

Abstract: Drug formulation has the power to move a product from a hospital setting directly to the home, empowering the patient to control their own treatment. High concentration formulations of antibodies have the potential to shift the burden of outpatient intravenous infusions in the clinic to the use of a ready-to-use autoinjector. Novel chemicals designed at Arecor have the potential to allow such a shift. Arecor has generated proof of concept data showing their ability to dramatically increase the stability of concentrated antibodies. The stability achieved is measured in not months, but years at elevated temperatures. The objective of this project is to examine a wide range of non-clinical toxicology assays of the novel excipients, the effect on further antibody models and to address the problem of viscosity. The aim is to produce a data package for regulatory approval in injectables and facilitate partnerships to allow this technology to reach the market.


Research activity codes: 5.1 PHARMACEUTICALS
Health categories: GENERIC HEALTH RELEVANCE
Title: Flat panel X-ray source for Highly Portable 3D Planar X-ray Solution
Funder: Innovate UK

Abstract: The business opportunity presented by MAX is to introduce a radical technology to create a shift from vacuum tubes to crystal based sources that can be manufactured using existing semiconductor foundry processes. The MAX source enables a flat panel X-ray source that does not require an external high voltage supply, high voltage electronics or a vacuum tube. As a result the most costly, bulky, expensive and fragile components of an X-ray source are eliminated. The result will be as transformational in clinical, security and industrial applications as the introduction of flat panel LCD and Plasma screens have been in Visual Display Units. This project will deliver a full scale (40cm x 40cm) solid state source manufactured in a semiconductor development environment and tested on a phantom. This device will then be ready to transfer to a manufacturing environment, CE tested and released for commercial production.


Research activity codes: 1.3 CHEMICAL AND PHYSICAL SCIENCES
Health categories: GENERIC HEALTH RELEVANCE
Title: A novel platform for targeted sequence enrichment prior to clinical NGS
Funder: Innovate UK

Abstract: Oxford Gene Technology, in collaboration with Professor Mike Griffiths at the University of Birmingham, will develop novel strategies to help implement next generation sequencing (NGS) into routine clinical practice. NGS offers significant benefits in delivery of improved and earlier diagnosis, informed therapeutic selection, and monitoring of disease progression and remission. Current technology platforms are not ideal for clinical practice, in terms of cost and/or performance. OGT has extensive experience in genomic technology and service provision. As a UK based life sciences company, OGT will focus its expertise to address some of the limitations of current NGS targeted sequencing technology, and in the process, will drive further investment in R&D and life sciences in the UK.


Research activity codes: 4.1 DISCOVERY AND PRECLINICAL TESTING OF MARKERS AND TECHNOLOGIES
Health categories: GENERIC HEALTH RELEVANCE
Title: Personalised iPS Cell Culture System
Funder: Innovate UK

Abstract: We plan to develop a novel, automated cell culture system specifically designed for iPSC and autologous cell culture. Early stage iPS cell culture is very labour intensive since cells must be monitored and fed daily for many weeks. For clinical applications cells must be cultured in a GMP clean room environment, with high staff and infrastructure costs. Our system will automate routine feeding, monitoring and incubation of cells in a 'closed', low cost unit, with remote monitoring and operation. It will maintain multiple plates of cells from one patient, to ensure segregation, within a small footprint, and be GMP compliant. We aim to deliver more consistent, higher quality cells, to minimise the cost, resource and 'hands on' time needed for iPSC and autologous cell culture, and to address a major barrier to wider adoption of iPSC for pre-clinical and clinical studies. The partners, TAP Biosystems, The Wellcome Trust Sanger Institute and University of Cambridge together have outstanding track records in developing innovative automation for cell culture and world class stem cell research, with a broad focus on developing and translating iPSC-based research into novel therapies.


Research activity codes: 1.3 CHEMICAL AND PHYSICAL SCIENCES
Health categories: GENERIC HEALTH RELEVANCE
Title: Automating quality assessment of B-mode ultrasound scans (AQABUS)
Funder: Innovate UK

Abstract: The diagnostic quality of ultrasound scans of the same patient can vary significantly, depending on the level of skill and expertise of the person scanning the patient and the equipment used. This variation in quality can lead to patients not being diagnosed correctly, having to be recalled for a rescan or being unnecessarily referred for more invasive and costly tests. This can make diagnosis more expensive delay treatment and make hospitals inefficient. Users of ultrasound equipment are encouraged to follow guidelines about how to get the best quality scans and clinical studies have demonstrated the benefit of this approach. However, routine reviews of scan quality are not undertaken because of the the time and cost involved, so the variations in scan quality and their potentially serious consequences persist. In a collaboration between Intelligent Ultrasound Ltd and Oxford University, the AQABUS project will develop and test an automated system ("Intelligent Quality Assurance" or "IQA") for the assessment of ultrasound scan quality. IQA will provide hospitals with a means to assess scans (that is, to determine their fitness for purpose), identify staff training needs and monitor of the impact of any training undertaken. The project will require innovations that include new software to automate the analysis and scoring of ultrasound scans, and computer-based techniques of artificial intelligence known as machine learning.


Research activity codes: 7.3 MANAGEMENT AND DECISION MAKING
Health categories: GENERIC HEALTH RELEVANCE
Title: New software to optimise the design of Stratified Medicine and Benefit-Risk studies
Funder: Innovate UK

Abstract: Exploristics seek to develop an innovative, new simulation platform and additional software modules which will allow users to simulate and evaluate clinical studies with multiple objectives prior to their initiation, allowing determination of the optimal design and analysis approach to meet study objectives with the minimum number of patients, and therefore cost. Exploristics believe that the future success of all clinical development of new drugs is fundamentally dependent on the application of correct study design, analysis strategy and decision criteria, given constraints. The platform will be particularly useful in development of Stratified Medicine studies and in assessing the Benefit/Risk of new treatments. It will be of interest to drug developers, data providers, diagnostics companies and to the Regulators. The concept has broad application to many other areas of clinical research.


Research activity codes: 8.4 RESEARCH DESIGN AND METHODOLOGIES (HEALTH SERVICES)
Health categories: GENERIC HEALTH RELEVANCE
Title: Magnetic Antibodies in Point-of-Care Diagnostics
Funder: Innovate UK

Abstract: The proposal is to assess the feasibility of using novel magnetic proteins as reagents in new point-of-care diagnostic devices. These devices will allow a GP or nurse, or eventually as the technology develops, a patient themselves to almost instantly obtain a blood test result using only a tiny pin-prick amount of blood. These unique magnetic proteins have been invented, developed and patented in the UK and will be produced in the UK.


Research activity codes: 4.1 DISCOVERY AND PRECLINICAL TESTING OF MARKERS AND TECHNOLOGIES
Health categories: GENERIC HEALTH RELEVANCE
Title: Evaluation of membrane-retentive pro-drugs to increase duration of effect to enable once-a-day dosing of inhaled muscarinic M3 receptor antagonists
Funder: Innovate UK

Abstract: Inhalation is an increasingly important delivery approach of therapeutic agents to the lung. There remains an unmet medical, commercial and practical need for 'once-a-day' treatment in various therapeutic areas and this is lacking for some current therapies. Once daily treatment is possible in some cases, but this approach can involve over-dosing to give the required duration of action. A negative issue is often side-effects due to initial higher than desired drug exposure. The proposed project involves chemically modifying current drugs via a pro-drug approach. The active drug will be released in a controlled manner at the target site, so the duration of pharmacological effect will be significantly longer than the traditional approach. The concept will be tested pre-clinically with the iterative design and synthesis of various test agents and drug metabolism tests in vitro and in vivo to prove that the required extension of duration is realised.


Research activity codes: 5.1 PHARMACEUTICALS
Health categories: GENERIC HEALTH RELEVANCE
Title: Improved method of freeze drying
Funder: Innovate UK

Abstract: Freeze drying is a method used for the stabilisation of a range of medical products, it has been estimated that over 50% of current medical products are freeze-dried due to their instability in the liquid state. However there exist a range of recalcitrant materials which cannot be satisfactorily freeze dried using conventional technology. In this project we will determine the benefits of a new method of freeze drying. The benefits of this technology could range from an improvement in the quality of materials which can currently be freeze dried (enzymes, protein therapeutics) to the creation of new products, for example freeze dried stem cells for application in regenerative medicine Freeze drying is a method used for the stabilisation of a range of medical products, it has been estimated that over 50% of current medical products are freeze-dried due to their instability in the liquid state. However there exist a range of recalcitrant materials which cannot be satisfactorily freeze dried using conventional technology. In this project we will determine the benefits of a new method of freeze drying. The benefits of this technology could range from an improvement in the quality of materials which can currently be freeze dried (enzymes, protein therapeutics) to the creation of new products, for example freeze dried stem cells for application in regenerative medicine Freeze drying is a method used for the stabilisation of a range of medical products, it has been estimated that over 50% of current medical products are freeze-dried due to their instability in the liquid state. However there exist a range of recalcitrant materials which cannot be satisfactorily freeze dried using conventional technology. In this project we will determine the benefits of a new method of freeze drying. The benefits of this technology could range from an improvement in the quality of materials which can currently be freeze dried (enzymes, protein therapeutics) to the creation of new products, for example freeze dried stem cells for application in regenerative medicine


Research activity codes: 1.3 CHEMICAL AND PHYSICAL SCIENCES
Health categories: GENERIC HEALTH RELEVANCE
Title: High Value Chemical Extraction from Sustainable Garlic Biomass
Funder: Innovate UK

Abstract: Neem Biotech is a specialist manufacturer of high value extracts from sustainable botanical sources and has a world-leading position in the isolation and extraction of high value ingredients from UK-sourced garlic (Allium sativum) which it sells in Europe, Asia Pacific and the USA. The company has a successful track record in the development and commercialisation of patented, plant-derived active ingredients that have broad application in industries such as pharmaceuticals, foods & beverages and agrochemicals. In this project, the company will explore the commercial and technical feasibility of developing new product lines that expand its portfolio whilst minimising the environmental footprint of its production processes. These new products have potential applications as high value food ingredients, animal feeds and/or agrochemicals.


Research activity codes: 1.3 CHEMICAL AND PHYSICAL SCIENCES
Health categories: GENERIC HEALTH RELEVANCE
Title: Model for Optimising Scaleable Telehealthcare
Funder: Innovate UK

Abstract: MOST aims to deliver a combination of guidelines and technology to aid adoption of remote monitoring solutions that enable better patient care. This will be achieved by the development of a ‘toolkit’ of guidelines, best practice, business models and technology to enable replication of large-scale Telehealthcare deployment across the care pyramid and through various commissioning channels. This will be supported by an independent quantitative & qualitative evaluation of the deployment of Telehealth to high-acuity patients in NHS North Yorkshire & York.


Research activity codes: 8.1 ORGANISATION AND DELIVERY OF SERVICES
Health categories: GENERIC HEALTH RELEVANCE