Glossary of terms/abbreviations used in report
General terms
Cell therapy (cellular therapeutics, cell-based therapies): administration of cells to the body to the benefit of the recipient
Gene Modified Immune Therapies/Ex vivo gene therapy: cells from the patient’s blood or bone marrow are removed and grown in a laboratory
Gene therapy: deliberate introduction of genetic material into cells
Limbal stem cell transplantation: involves the grafting of stem cells that have been taken from donor eyes and grown in tissue culture, with the aim of improving vision and other symptoms such as eye irritation and dryness.
Regenerative medicine: replaces or regenerate human cells, tissues or organs to restore or establish normal function
Tissue engineering: use of a combination of cells, engineering, materials and methods ex-vivo living tissues and organs that can be implanted to improve or replace biological functions.
Transplantation: process of implanting cells, tissues or organs.
Non-cellular components
Biomaterial: material intended to interface with biological systems to evaluate, treat, augment or replace any tissue, organ or function of the body.
Manufacturing and production
Good Manufacturing Practice (GMP) is a system for ensuring that products are consistently produced and controlled according to quality standards. It is designed to minimise the risks involved in any pharmaceutical production that cannot be eliminated through testing the final product.
Marketing authorisation: authorisation by a European regulatory authority for a medicinal product to be placed on the market.
Translation: active turning of a basic science discovery into a safe and effective therapy deployed in routine clinical practice.
Regulatory terms for products and therapies
Advanced therapy medicinal product (ATMP): medicinal product for human use that is a gene therapy product, medicinal product, a somatic cell therapy medicinal product or tissue engineered product.
Health Technology Assessment (HTA): refers to the systematic evaluation of properties, effects and/or impacts of health technology. It is a multidisciplinary process to evaluate the social, economic, organisational and ethical issues of a health intervention or health technology.
Organisations/reviews
Accelerated Access Review was announced in November 2014 by the then Minister for Life Sciences, George Freeman MP. Its aim is to speed up access to innovative drugs, devices, diagnostics and digital products for NHS patients.
Advanced Therapy Manufacturing Taskforce (ATMT) was launched by the Medicines Manufacturing Industry Partnership to secure the future of manufacturing advanced therapies in the UK.
Cell and Gene Therapy Catapult was established in 2012 as an independent centre of excellence to advance the growth of the UL cell and gene therapy industry, by bridging the gap between scientific research and full scale commercialisation.
Medicines Manufacturing Industry Partnership (MMIP) represents the voice of medicine manufacturers in the UK. It was established jointly by Government and the biopharmaceutical industry in 2014 to ensure that the UK is recognised by the global medicines industry as a world-class, advanced centre for medicines manufacturing.
Regenerative Medicine Expert Group (RMEG) is an expert group formed following publication of the Lords Science and Technology Committee report on regenerative medicine. Their remit is to develop an NHS regenerative medicine strategy so that the NHS is fully prepared to deliver innovative treatments as well as also assess the effect of regulation on the development of regenerative medicines in the UK.
UK Cell Therapy Manufacturing Centre: the Cell and Gene Therapy Catapult will be building a £55 million large scale GMP manufacturing centre in Stevenage to help bring cell and gene therapies to market in the UK and internationally. The centre is due to be opened in 2018.
UK Regenerative Medicine Platform (UKRMP): a £25 million initiative to address regenerative medicine translational challenges. The UKRMP was established in 2013 by the Biotechnology and Biological Sciences Research Council, the Engineering and Physical Sciences Research Council and the Medical Research Council. The UKRMP provides academic expertise, innovation and knowledge, connected to commercial and clinical end-users.
28 April 2017