Glossary
A
Acute Kidney Injury
Rapid reduction in or acute loss of kidney function.
Acute Lung Injury
A distinct form of acute respiratory failure.
AMD Age related Macular Degeneration
Degenerative eye disease that causes damage to the macula (central retina) of the eye.
Angiogenesis
Formation of new blood vessels (i.e. in wound healing, menstrual cycle); Tumor angiogenesis is the formation of new blood vessels that grow into the tumor, giving it nutrients and oxygen to assist its growth.
Antisense molecules
A sequence of nucleic acids, typically created in the lab, whose sequence is exactly complementary/opposite to an mRNA molecule made by the body. mRNA molecules made by the body serve as templates for the synthesis of protein (see Translation). Since the "antisense" mRNA molecule binds tightly to its mirror image, it can prevent a particular protein from being made.
ASCO
American Society of Clinical Oncology.
AtuPLEX®
The AtuPLEX® system broadly delivers siRNA to the endothelial cells of the vascular system.
AtuRNAi
A proprietary form of RNAi (siRNA) developed by Silence Therapeutics.
C
Cancer
A group of diseases in which cells grow unrestrained in an organ or tissue in the body. Cancer can spread to tissues around it and destroy them or be transported through blood or lymph pathways to other parts of the body.
CD31
Cluster molecule (number 31) also referred to PECAM-1 or Platelet Endothelial Cell Adhesion Molecule1.
D
DACC
A proprietary siRNA delivery system focused to the lung developed by Silence Therapeutics
DBTC
A proprietary siRNA delivery system focused to the liver developed by Silence Therapeutics
Delayed Graft Function
A form of acute kidney failure in the immediate post-transplant period
Development
The process by which a compound discovered in research is progressed through human clinical trials prior to approval to market
Diabetes
A range of conditions characterised by high blood sugar and glucose intolerance
Diabetic Macular Oedema
A disorder of the eye caused by leaking blood vessels in the retina leading to impairment of vision
DNA
The molecule that encodes genetic information and serves as a template for the production of RNA and indirectly also proteins
Drug optimisation
A method to improve the efficacy of a drug
Drug target
A gene or gene product (protein) against small molecule drugs will be screened and developed
E
Endothelium
A thin layer of cells that lines the interior surface of blood vessels from the heart to the smallest capillary
Expression
The process by which the information in a gene is used to create proteins
F
Formulation
A combination of an active drug and pharmacologically inactive ingredients used to achieve adequate bioavailability
G
Gene
Structurally, a basic unit of hereditary material; an ordered sequence of nucleotide basis that encodes a product (this product could be just RNA like rRNA or finally coding for a protein)
Gene product
The RNA or protein that results from the expression of a gene
Gene silencing
Targeting or interfering with a specific gene and preventing its expression (in other words, preventing it from leading to a protein)
Gene target
A gene or its product (protein) which plays a critical role in pathology
Genome
DNA sequence of an organism; its size is generally given as its total number of base pairs
I
In vitro
A biological or biochemical process carried out in a test tube or similar vessel
In vivo
A biological or biochemical process carried out in a living organism.
Indication
A relevant patient condition
Ischemia Reperfusion Injury
Ischemia is a restriction in blood supply causing absence of oxygen and nutrients. Reperfusion injury is the tissue damage caused when blood supply returns to the tissue after a period of ischemia.
M
Metastases
Tumours produced by the spread of cancer cells to other parts of the body.
Micro RNA
Mall regulatory antisense RNA that may regulate the expression of other genes.
Monoclonal antibodies
One possible treatment for cancer; they bind only to cancer cell-specific antigens and induce an immunological response against the target cancer cell.
mRNA
Messenger RNA.
P
Phase I
Studies conducted usually in healthy subjects to determine initial saftey, tolerability and pharmokinetics of a drug and from which the participants do not derive any therapeutics benefit.
Phase IIa
Early phase II studies, typically pilot or feasability studies conducted in a small number of patients.
Pre-clinical
Experiments performed before starting clinical trials to assess a compound and its potential to cause adverse side effects.
Protein
Organic compounds made up of amino acids. Proteins are responsible for most of the function and much of the structure of living organisms, including humans.
Pulmonary Hypertension
High blood pressure in the arteries of the lungs.
R
R&D
Research and development.
RNA
Ribonucleic acid.
RNAi
RNA interference: a mechanism used to prevent translation of specific genes by targeting and degrading mRNA embodying the genetic sequence of the relevant gene with the intention of inhibiting production of disease causing proteins.
RNAi Therapeutics
A potential new class of therapeutics which mimic the natural RNAi mechanism in order to suppress (silence) unwanted gene expression and consequently function as it occurs in diseases.
S
Sequence
The order of nucleotides in a DNA or RNA molecule, or the order of amino acids in a protein molecule.
siRNA
Short interference RNA: short double stranded (19 to 21 nucleotides in length) RNAi molecule.
Small molecule
Chemical entity used for screening against drug targets.
T
Target
The molecule that a substance or a drug binds to (often targets are proteins).
Therapeutic
A pharmaceutical product targeted to treat a specific disease.
Transfection
The alteration of the genetic code within a cell by the addition of exogenous genetic sequences.
Translation
The process of using a messenger RNA sequence to build a protein. The messenger RNA serves as a template on which transfer RNA molecules, carrying amino acids, are lined up. The amino acids are then linked together to form a protein chain.
V
Validate, Validation
Proof of relevance and/or correctness, for example the relevance of a gene for a disease.