Glossary

Age-related macular degeneration ('AMD')

A retinal degenerative disease causing progressive loss of central vision.

Candidate product

A molecule or substance with the potential to become a therapeutic product.

Cell banking

A process for the controlled preparation of a cell therapy product, resulting in a large number of vials of frozen cells.

Cell line

A continuous growing cell culture that is stable. Usually clonal or derived from a single cell.

CNS

Central nervous system

Diabetes

A disease characterised by absolute or relative insulin insufficiency and high blood sugar

Differentiation

The maturation of a stem cell into a functional cell

Dopamine

A chemical which carries signals from one nerve cell to another in the brain in those regions where movement is controlled

Dopaminergic neurons

Nerve cells releasing dopamine. Loss of dopaminergic neurons is a feature of Parkinson's disease.

Drug screening

The process by which large numbers of compounds are systematically evaluated to choose the most promising for further development

Gene

A length of DNA coding for a single protein

GLP

Good Laboratory Practice, a system of guidelines to ensure quality in pre-clinical work

GMP

Good Manufacturing Practice, a system of guidelines to ensure manufacturing quality

Huntington's disease

An inherited adult-onset disease of the brain characterised by dementia and involuntary movements. The disease is progressive and there is currently no known cure

Immortalisation

The ability of a genetically engineered cell line to reproduce indefinitely

Indication

The use for which a drug or therapy is intended

in vitro

Within a glass, observable in a test tube, in an artificial environment.

in vivo

Within the living body.

Neural stem cells/brain stem cells

Cells within the brain which can both make more of themselves and also mature into neurons and other brain cell types

Neurons

A nervous system cell able to conduct electrical impulses

Orphan Drug status

Status granted by the US Food and Drug Administration (FDA) which provides certain development, registration and marketing incentives, for development of treatments of small (under 200,000 per annum in the United States) incidence conditions

Parkinson's disease

A progressive neurological disease of older people characterised by tremor, difficulty in movement and speech

Phase I clinical trial

The assessment of the safety of a biologically active substance in volunteers

Phase II clinical trial

The assessment in patients of a drug to determine close range and preliminary efficacy

Phase III clinical trial

Definitive studies in patients to determine efficacy and safety of a drug prior to marketing approval

Pre-clinical studies

Tests carried out on a candidate drug or therapy, manufactured to meet regulatory guidelines, to ensure product safety and quality prior to commencing studies in humans

Proof-of-concept

Pre-clinical or clinical results demonstrating that a candidate drug or therapy is effective

Retinitis pigmentosa ('RP')

A group of inherited eye diseases causing the degeneration of photoreceptor cells in the retina

Stem cell

A cell that is both able to reproduce itself and, depending on its stage of development, to generate all other cell types within the body or within the organ from which it is derived

Stroke

Damage to a group of nerve cells in the brain due to interrupted blood flow, caused by a blood clot or blood vessel bursting. Depending on the area of the brain that is damaged, a stroke can cause coma, paralysis, speech problems and dementia

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