Guidance

Regenerative medicine, cell and gene therapy

Updated 1 December 2021

Introduction to regenerative medicine, cell and gene therapy

Regenerative medicine is one of the most exciting and promising areas of science, and UK scientists have been involved since the outset.

Europe has the second largest regenerative medicine ecosystem in the world with 400 plus companies related to regenerative medicine. Nearly 1 in 3 small to medium sized businesses (SMEs) active in Europe’s advanced therapy medicinal product market are based in the UK.

Numerous UK organisations give support, advice or useful information for each development stage of digital health and care products.

Discovery and deep research development stage: UK support organisations

Biotechnology and Biological Sciences Research Council (BBSRC)

BBSRC is part of UK Research and Innovation, a new body which works in partnership with universities, research organisations, businesses, charities, and government to create the best possible environment for research and innovation to flourish.

Cardiff Institute for Tissue Engineering and Repair (CITER)

CITER is an internationally recognised centre of excellence in the field of tissue repair, regeneration and rehabilitation, focusing on:

• interdisciplinary research
• education
• clinical practice

Clinical Practice Research Datalink (CPRD)

CPRD is a governmental, not-for-profit research service. It’s jointly funded by the NHS National Institute for Health Research (NIHR) and the Medicines and Healthcare Products Regulatory Agency (MHRA), part of the Department of Health and Social Care (DHSC). CPRD has been providing anonymised primary care records for public health research for 30 years.

This data enables:

• observational studies
• clinical trial feasibility and protocol optimisation
• post-market surveillance

Engineering and Physical Sciences Research Council (EPSRC)

The EPSRC is the main funding body for engineering and physical sciences research in the UK, investing in research and postgraduate training. Its portfolio covers a vast range of fields from healthcare technologies to structural engineering, manufacturing to mathematics, advanced materials to chemistry.

EPSRC is part of UK Research and Innovation, a new body which works in partnership with universities, research organisations, businesses, charities, and government to create the best possible environment for research and innovation to flourish.

EPSRC Centres for Innovative Manufacturing has 16 centres for innovative manufacturing including:

• continuous manufacturing and crystallisation (CMAC)
• emergent macromolecular therapies
• medical devices
• regenerative medicine

Each centre can be used as a platform for organisations to invest in. They all:

• develop collaborations
• carry out feasibility studies
• supports research projects

The EPSRC Centre for innovative manufacturing in medical devices is developing advanced methods for functionally stratified design and near patient manufacturing, to enable cost effective matching of device function to the patient needs and surgical environment.

Medical Research Council (MRC)

The MRC improves human health through world-class medical research. It funds research across the biomedical spectrum and their research has resulted in life-changing discoveries for over 100 years. Their work ranges from laboratory research, for example on genes and molecules, right through to clinical trials and population studies.

MRC’s science is split into 6 broad areas of research which includes:

• infections and immunity
• molecular and cellular medicine
• neurosciences and mental health
• population and systems medicine
• global health
• translational research

Their research is carried out in universities, hospitals and a network of dedicated establishments across the UK and Africa.

National Biologics Manufacturing Centre (NBMC)

NBMC was established by the Centre for Process Innovation (CPI), the UK’s national technology and innovation centre for the process manufacturing industries.

CPI combines state of the art facilities and technical expertise to support the growth and development of the UK biologics industry and supply chain. They help companies de-risk process development through proof of concept testing to accelerate the commercialisation of new processes and technologies.

NHS Blood and Transplant, Tissue Services (NHSBT)

NHS Blood and Transplant, Tissue Services is the UK’s major provider of human tissue for transplant. Its role is to co-ordinate, recover, process, bank and supply human tissue grafts for use in surgery within the National Health Service (NHS) and independent hospitals in the UK.

Oxford Stem Cell Institute (OSCI)

It aims to create a seamless pathway from basic science, through translational medicine to clinical trials by harnessing world-leading expertise in the fields of:

• stem cell biology
• immunology
• tissue engineering
• materials science
• medicinal chemistry
• cancer biology

Scottish Centre for Regenerative Medicine

The Centre for Regenerative Medicine is a research institute based at the University of Edinburgh. Scientists and clinicians study stem cells, disease and tissue repair to advance human health.

Scottish National Blood Transfusion Service (SNBTS)

SNBTS is the specialist provider of transfusion medicine in Scotland, supplying high quality blood, tissues, cells and services. They work with communities, hospitals and professionals to ensure that the donor’s gift is used effectively for the benefit of patients.

UK Stem Cell Bank

The UK Stem Cell Bank is funded by the MRC and Biotechnology and BBSRC. It was established to provide an ethically approved, quality controlled and assured repository of human embryonic, fetal and adult stem cell lines to underpin high quality research and healthcare developments.

UK Stem Cell Foundation

The UK Stem Cell Foundation was set up to speed up the progress of promising stem cell research and technology into treatments and therapies for patients. It is the only UK charity to focus specifically on stem cell research as a potential treatment for a wide range of conditions and diseases.

Wellcome / EPSRC Centre for Medical Engineering

The centre, jointly funded with the EPRSC and King’s College London, is developing markers to allow early detection of disease so that its onset or progression can be prevented. It will focus on cardiovascular, oncological and neurological applications of medical imaging (primarily MRI and PET).

Development and translation development stage: UK support organisations

Catapults

The catapult centres are a network of world-leading centres designed to transform the UK’s capability for innovation in specific areas and help drive future economic growth. The catapults include:

• cell and gene therapy
• digital
• high value manufacturing
• medicines discovery
• precision medicine

The Cell and Gene Therapy Catapult:

• accelerates delivery of therapies to the patient
• works with industry to take products into clinical trial, de-risking them for further investment
• provide technical and regulatory expertise to ensure products can be made to good manufacturing practice and get to the clinic safely in the shortest time
• provide opportunities for collaboration and access to NHS clinical partners and expertise networks

Edinburgh BioQuarter

Edinburgh BioQuarter brings together academic scientists, clinicians, patients and other health professionals with companies. It utilises world class academic and clinical research facilities, to enable collaboration and accelerate the development of new drugs, diagnostic tools and medical devices, to diagnose and treat diseases.

Health and Care Research Wales

Health and Care Research Wales:

• provides an infrastructure to support and increase capacity in R&D
• runs a range of responsive funding schemes
• manages resources to promote, support and deliver research in health and social care
• facilitates the successful delivery of commercial research in the NHS
• promotes Wales as an effective place to do research

NHS Research Scotland

NHS Research Scotland makes it easier for commercial organisations to develop mutually beneficial partnerships with clinical and research expertise across medical universities and their associated NHS Health Boards in Scotland.

Innovate UK

Innovate UK is the UK’s innovation agency. It works with people, companies and partner organisations to find and drive science and technology innovations. Innovate UK fund the strongest opportunities and connect innovators with the right partners they need to succeed.

Innovate UK Knowledge Transfer Partnership is Europe’s leading programme helping businesses to improve their competitiveness and productivity through the better use of knowledge, technology and skills that reside within the UK knowledge base.

National Institute for Health Research (NIHR)

The NIHR is funded through DHSC. It builds capacity and leadership in the research workforce, and attracts investment into the UK by supporting partnerships with life science companies, including small and medium enterprises, and charities.

NIHR Biomedical Research Centres (NIHR BRC) were formed through partnerships between England’s leading NHS organisations and universities. Twenty NIHR BRCs conduct translational research to transform scientific breakthroughs into life-saving treatments for patients. Staffed by expert investigators and clinicians, the centres are leaders in translating lab-based discoveries into new cutting edge treatments, technologies, diagnostics and other interventions in clinical settings.

NIHR Biomedical Research Units (BRU) are based within England’s leading NHS organisations and universities. They undertake translational research in priority areas of high disease burden and clinical need and focus on specific therapeutic areas of disease including:

• musculoskeletal
• gastrointestinal
• respiratory
• cardiovascular
• dementia
• nutrition
• diet and lifestyle
• deafness and hearing

NIHR Clinical Research Facilities for Experimental Medicine (NIHR CRFs) are dedicated and purpose-built facilities, where specialist clinical research and support staff from universities and NHS Trusts work together on patient-orientated commercial and non-commercial experimental medicine studies.

Life science companies can access assistance for their studies throughout the research process from study design to data collection and management.

NIHR Clinical Research Network (NIHR CRN) is the research delivery arm of the NHS in England. One of its priorities is to proactively support the life-sciences industry to deliver high quality commercial contract clinical research across all therapeutic areas. Its free-of-charge study support service helps life-science organisations effectively find investigators and sites, set-up and deliver their studies to time and target.

NIHR Clinical Research: Network Study Support Service is the research delivery arm of the NHS in England. One of its priorities is to proactively support the life-sciences industry to deliver high quality commercial contract clinical research across all therapeutic areas. Its free-of-charge study support Service helps life-science organisations effectively find investigators and sites, set-up and deliver their studies to time and target.

NIHR Medical Device and Diagnostics Industry Partnerships support the development and evaluation of new medical technologies, devices and diagnostics across England. It brings together experts in the design and conduct of clinical studies, working in dedicated facilities embedded within NHS-university partnerships. The NIHR infrastructure provides an important opportunity to work in partnership with the medical device and diagnostics sectors, successfully helping to translate ideas into innovative technologies.

NIHR Office for Clinical Research Infrastructure (NOCRI) provides potential partners, including the life sciences industry and charities, with a direct and simplified route to a wide range of experimental medicine facilities and expert NIHR investigators.

NOCRI works with organisations to help them navigate this infrastructure and, where required, form partnerships and collaborations to bring new treatments to patients faster.

NHS Research Scotland (NRS)

NHS Research Scotland promotes and supports excellence in clinical and translational research in Scotland for patients to benefit from new and better treatments. NRS connects NHS, industry and academia to accelerate the development of new treatments, devices and diagnostics to tackle complex healthcare needs.

NHS Research Scotland Permissions Coordinating Centre

The NHS Research Scotland Permissions Coordinating Centre offers a coordinated and streamlined process for obtaining Scotland-wide NHS R&D permission for multicentre clinical studies.

NICE Office for Market Access (NICE OMA)

NICE OMA provides expert advice to the life sciences industry on pharmaceuticals, health technologies, diagnostics, and devices.

They advise on:

• NICE processes
• how to work with NICE to achieve your aims
• how NICE links with different parts of the system
• the Early Access to Medicines Scheme (EAMS) and how NICE supports it

NICE Scientific Advice (NICE SA)

NICE SA offers a fee-based consultancy service to developers of:

• medicines (including orphan drugs)
• devices
• diagnostics
• a range of services for different product types

Roslin Cells

Roslin Cells, founded in 2006, is the parent company of Roslin Cell Therapies and Roslin Cell Sciences. It has developed 2 distinct investment and business opportunities in the development and manufacture of cell based therapies and the generation of stem cells to support commercial academic and drug research.

UK Regenerative Medicine Platform (UKRMP)

The platform is a £25 million initiative that addresses the important translational challenges of regenerative medicine. Central to its operation are 5 interdisciplinary and complementary research hubs that collectively provide a national resource through the generation of new tools, protocols and resources. These can be utilized by other UK research groups in both academia and industry.

Regulatory and health technology assessment development stage: UK support organisations

Medicines and Healthcare products Regulatory Agency (MHRA)

MHRA is the regulatory agency responsible for regulating all medicines and medical devices in the UK by ensuring they work and are acceptably safe. Robust and fact-based judgements underpin all the work carried out to ensure that the benefits justify any risks.

MHRA Innovation Office is a single point of access to expert regulatory information, advice and guidance that helps organisations of all backgrounds and sizes develop innovative medicines, medical devices or novel manufacturing processes. They provide expert knowledge, guidance and experience to help develop products and save time and money.

National Institute for Biological Standards and Control (NIBSC)

The institute is a global leader in the characterisation, standardisation and control of biological medicines. NIBSC plays a major role in assuring the quality of biological medicines worldwide through the provision of biological reference materials, by testing products and carrying out research.

National Institute for Health and Care Excellence (NICE)

NICE publishes guidance in 4 areas including the use of health technologies within the NHS such as the use of the new and existing medicines, treatments and procedures.

NICE SA offers a fee-based consultancy service

Scottish Medicines Consortium (SMC)

The SMC accepts for use those newly licensed medicines that clearly represent good value for money to NHS Scotland. It analyses information supplied by the medicine manufacturer on the health benefits of the medicine and justification of its price.

The consortium is made up of lead clinicians, pharmacists and health economists together with representatives of health boards, the pharmaceutical industry and the public.

Delivery, supply and NHS / patient development stage: UK support organisations

Academic Health Science Networks (AHSNs)

The AHSNs facilitates access into the NHS and aligns education, clinical research, informatics, innovation, training and healthcare delivery providing a network aimed at supporting the adoption and dissemination of innovative technologies.

There are 15 AHSNs across England, and each AHSN works across a distinct geography serving a different population in each region.

Clinical Commissioning Group (CCG)

CCGs are clinically-led statutory NHS bodies responsible for the planning and commissioning of health care services for their local area. CCGs commission most of the hospital and community NHS services in the local areas for which they are responsible.

National Health Service (NHS)

The NHS, launched in 1948, is the largest unified healthcare system in the world. It treats almost 1 million patients on a daily basis. It was born out of a long-held ideal that good healthcare should be available to all, regardless of wealth and remains free at the point of use for anyone who is a UK resident. That is currently more than 64.6 million people in the UK.

NHS England sets the priorities and direction of the NHS in England. It shares out more than £100 billion in funds in the commissioning of health care services in England and holds organisations to account for spending this money effectively.

NHS Scotland is the publicly funded healthcare system in Scotland. Health and social care policy and funding are the responsibility of the Health and Social Care Directorates of the Scottish Government. NHS Scotland currently employs approximately 140,000 staff who work across 14 territorial NHS Boards, 7 Special NHS Boards and 1 public health body.

NHS Northern Ireland provides the gateway to Health and Social Care Services in Northern Ireland, and links to the Hospital and Community services provided by the 6 Health Trusts, General Practices, Health and Social Care Board and other HSC Agencies. The Northern Ireland Executive, through its funding of the Health Department is responsible for the funding of the service.

NHS Wales is the official corporate name of the Welsh National Health Service, the publicly funded healthcare system which is the responsibility of the devolved Welsh Government. There are 7 Local Health Boards (LHBs) in Wales and 3 national NHS Trusts. Each LHB is responsible for delivering all NHS healthcare services within a geographical area. Three NHS Trusts, called ‘all-Wales trusts’, operate nationwide agencies and services.

NHS procurement

There are several routes to market for companies interested in supplying goods and services to the NHS.

Read more on Partnering with the NHS to sell goods and services.

NICE Office for Market Access (NICE OMA)

NICE OMA provides expert advice to the life sciences industry.

NIHR Clinical Research Network Study Support Service

NIHR Clinical Research: Network Study Support Service is the research delivery arm of the NHS in England. One of their priorities is to proactively support the life-sciences industry to deliver high quality commercial contract clinical research across all therapeutic areas. Their free-of-charge study support service helps life-science organisations effectively find investigators and sites, set-up and deliver their studies to time and target.

NHS procurement

There are several routes to market for companies interested in supplying goods and services to the NHS.

Read more on Partnering with the NHS to sell goods and services.

Scottish Medicines Consortium (SMC)

The SMC accepts for use those newly licensed medicines that clearly represent good value for money to NHS Scotland. SMC analyses information supplied by the medicine manufacturer on the health benefits of the medicine and justification of its price. The consortium is made up of lead clinicians, pharmacists and health economists together with representatives of health boards, the pharmaceutical industry and the public.

Launch global access development stage: UK support organisations

Department for International Trade (DIT)

DIT is the government department that helps UK-based companies succeed globally and assists overseas companies to bring investment to the UK.

DIT’s combined network of government and private-sector specialists in the UK and in British Embassies and Consulates throughout the world offer practical advice and free and confidential support.

Life Sciences Organisation (LSO)

LSO helps UK life science companies do business overseas and encourages foreign life science companies to invest in the UK. It provides support from the earliest stages of research and development through to clinical trials, commercial operations and business partnerships.

Healthcare UK

Healthcare UK is a joint initiative of DHSC, DIT and NHS England.

Healthcare UK helps UK healthcare providers do more business overseas by:

• promoting the UK healthcare sector to overseas markets
• supporting healthcare partnerships between the UK and overseas healthcare providers

NICE OMA

NICE OMA provides expert advice to the life sciences industry.

UK life science membership associations and networking organisations

Find UK life science membership associations and networking organisations who can aid business growth, competitiveness and innovation in the life sciences sector.