Glossary

A

Acute kidney injury (AKI) Acute kidney injury (AKI) is a rapid deterioration of kidney function, resulting in inability to maintain electrolyte and acid-base balance. AKI is often caused by the blockage of renal circulation due to decreased renal perfusion or renal artery occlusion.
Adverse Event Adverse event is an occurrence of unexpected or undesired harmful effects in the course of exposure to the testing drugs, such as in clinical trials. Meanwhile, side effect, which is rather common, is often used when the effect cannot be denied for the relevance with the drugs. Thus the adverse event not only means the occurrence of effect caused directly by drug itself, but also the one caused by the administration procedures or other environmental factors.
Antibody drug Antibodies are the proteins produced by the B cells, a type of immune cells, to identify and neutralize pathogens or the endogenous transformed malfunctioning cells. Antibody drug is the drug which eliminates target or inhibits function of target protein taking advantage of specific binding property.
Autoimmune disease The immune system usually does not attack own tissues (cells, organs); malfunctioning immune system may mistakenly attack own tissues to cause tissue damages or inflammation, of which the condition is called autoimmune diseases.

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B

B cells A type of lymphocytes, which produce antibodies to eliminate pathogens upon activation by infection.
B cells to produce autoantibodies B cells which cause autoimmune diseases by the production of autoantibodies attacking own cells and tissues.
B-cell Chronic Lymphocytic Leukemia (B-CLL) B-cell chronic lymphocytic leukemia (B-CLL) is a type of slowly progressing cancer that caused by the transformation of B cell lymphocytes into malignant cells with primary symptoms such as increase of white blood cells, swollen lymph nodes, fatigue, loss of weight, etc.

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C

Cell cycle Cell cycle is a series of events that repeatedly occur in a cell, leading to its division and replication. A cell cycle is divided into four steps that produce two daughter cells from one: G1 (Gap) → S (DNA replication) → G2 (Gap) → M (mitosis).
Cell cycle checkpoint mechanism Cell cycle checkpoints are control mechanisms in eukaryotic cells which ensure proper division of the cell by deceleration of the cell cycle progression when abnormality occurs. The checkpoints allow the restart of cell cycle when damages are repaired properly; they otherwise induce apoptosis program when the abnormalities are too serious to repair.
Clinical development Clinical development is the activity to proceed the examinations of the drug seeds at clinical stage for the evaluation of in human efficacy and safety after the preclinical experiments.
Clinical research Clinical research, which is conducted under distinctive system from clinical trial, is performed at clinical sites to administer study drugs on small group of patients for understanding cause and pathology of diseases, improving prophylaxis, diagnosis of diseases and therapeutic strategy to enhance QOL (quality of life) of patients’.
Clinical trial Clinical trial is conducted for the evaluation of in human safety and efficacy of the drugs.
Cytokine Cytokines are a broad range of proteins which activate various immune responses.

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D

Delayed Graft Function(DGF) Delayed Graft Function (DGF) is a renal failure caused by ischaemia and reconstitution of blood flow after kidney transplantation, which is defined by the need of dialysis in the first week after surgery.
Dendritic cell (DC) Dendritic cells (DCs) are named based on the stellate extensions like stems of trees; the cells function in the immune system by making orders to other immune cells to identify the “enemies” when the body is challenged by infection or autologous malfunctioning cells.
Diabetic Macular Edema (DME) Diabetic macular edema (DME) is categorized as the diabetic retinopathy among three main complications of diabetes. DME reveals thickening and swell of macula and formation of capillary hemangioma in the small retinal vessels, which is the result of the vascular damages caused by high blood sugar, followed by fluid accumulation in macula or central portion of the eyes.
DNA nucleotide drug DNA nucleotide drug is a category of drug generated with DNA.
Drug combination Drug combination refers to combinatorial use of two or more drugs at the same time to expect synergistic efficacy with lower dosage and fewer side effects.
Drug delivery Drug delivery refers to effective transport of the pharmaceutical compounds to the location of the body where the therapeutic effects are needed.

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F

Fast track designation Fast track designation is a designation of certain drug development program by FDA to accelerate of the review process in the aim of facilitation of such programs; the drugs are often which treat the life-threatening diseases or intractable diseases.

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G

Gene expression Gene expression is the process by which gene information recorded in DNA exerts its function. Gene information is transcribed into messenger RNA and delivered to ribosome where functional proteins, such as enzymes, are synthesized; the function of the gene is executed by the action of such proteins.

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I

Immune modulation drug Immune modulation drugs affect immunity to upregulate or suppress the functions of immune system, which protects oneself from non-self substances, such as pathogens. Followings are the examples of the drugs of this category: the drugs to upregulate immune response to remove abnormal cells such as cancer cells; immunosuppressive drugs for autoimmune diseases which inhibit overreactivated immune responses and protect body from attack against own normal tissues.
Immunity (Immune system) Immunity is the biological defense system to fight against pathogens like viruses or bacteria or abnormal cells such as fast-growing cancer cells.
Immunocompetent cell (Immune cell) Immunocompetent cells are the cells which play important roles in the immune system, which functions to clear non-self substances from the body. Immunocompetent cells elicit immune reaction by the production of antibodies (by B cells) or cytokines (by T cells) or attack against abnormal cells (by NK cells).
Immunosuppressant Immunosuppressants are the drugs which suppress immune system. They are, for example, used to prevent the rejection of transplanted organs or alleviate symptoms of autoimmune diseases.
Investigational New Drug (IND) The data package showing safety and efficacy of the drug for the application of commencement of clinical trials.
Interferon (IFN) Interferon (IFN) is produced and released by pDCs and other immune cells in response to pathogen infection; it activates and transfers signals to other immune cells to suppress viral replication and cell proliferation.
Intractable Disease Intractable Diseases refer to the diseases that result from unidentifiable causes and are without established treatment; they are problematic due to the high risk of permanent damages. They often chronically develop and require a significant amount of labor for the patient’s care, causing a heavy burden on other family members of the patient both financially and mentally.
Intractable Disease Designation Regarding the diseases designated as Intractable Diseases, the government runs funding program to support the researches on the designated diseases and offers governmental program to support the patients, such as subsidization of a part of medical expenses.
Ischaemia Ischaemia is a condition with the shortage of oxygen caused by the restriction of blood supply to the local tissues; it occurs by the conditions such as blood vessel injury.

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L

Laser photocoagulation Laser photocoagulation is a conventional treatment for DME to inhibit neovascularization which causes DME and reduce the swelling of retinal tissue.
Lead compound A lead compound in the drug discovery is a compound that has expected biological activity found in the early discovery stage. Drug seeds are developed later from the lead compound by the chemical modification to improve the potency and down-regulate the side effects, which is called lead optimization step.
Lipophilicity Lipophilicity refers to the ability of a chemical compound to dissolve in lipids such as fats or oils. Compounds with higher lipophilicity usually are difficult to dissolve in water, called high hydrophobicity.

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M

MCM protein complex MCM proteins play important roles during the eukaryotic DNA replication initiation before the cell division. The regulation of MCM complex activation status allows only one round of chromosome replication per each cell cycle.
myeloid DC (mDC) Myeloid DC (mDC), one subset of dendritic cells, which recognizes non-self such as pathogens and then transmits information of such invaders to T cells to initiate immune responses; it is also called cDC (conventional DC).
Messenger RNA (mRNA) mRNA is a polymerized RNA which interprets the information encoded in nucleic DNA and transfer it to the sites of protein synthesis in the ribosomes.
Mode of action (Mechanism of action) Mode of action describes functional changes of effective drugs in vivo at the molecular levels.
Modification Modification refers to the alteration in the structure of a compound through chemical reaction to create new character of compound.
Molecular targeted drug Molecular targeted drugs refer to drugs that target specific molecules (proteins, and etc.) to exhibit efficacy. For example, cancer growth is suppressed by the inhibition of the molecules needed for the cancer cell proliferation. Molecular targeted drugs are expected to have lower side effects since they specifically bind to target molecules but not to the molecules which may cause unwanted effects.
Myeloma Myeloma is a type of blood cancer that originates from cells in the bone marrow called plasma cells. Due to its occurrence in the bones of entire body, it is also called multiple myeloma. Its symptoms include anemia, general tiredness, loss of weight, backache and susceptibility to infections, etc.

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N

NK cell NK cells are a type of lymphocytes, which rapidly responds to antigens upon invasion to induce immune responses; it is activated by IFN.
Non-Arteritic Anterior Ischemic Optic Neuropathy (NAION) Non-Arteritic Anterior Ischemic Optic Neuropathy (NAION) is the disease of irreversible loss of vision caused by diabetes, hypertension or arteriosclerosis induced optic neuropathy.
Nucleic acid Nucleic acids are the substances such as DNA or RNA majorly exist in nucleus of the cells; DNA records genetic information, and RNA interprets the genetic information and function as an instruction of the protein synthesis.
Nucleotide drug Nucleotide drug, which is mainly made from nucleic acids, is one of the next generation drugs. We expect this class of novel drugs to be widely applied to incurable diseases and unmet medical needs in future, based on its different mechanisms of actions from conventional small molecule drugs or antibody drugs.

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O

Oligonucleotide Nucleotides are monomers of nucleic acids. Oligonucleotide is the polymer of several to 20 nucleotides.

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P

plasmacytoid DC (pDC) plasmacytoid DC (pDC) is a subset of dendritic cells, which produces large amount of IFNa in response to infection. It is well known for the roles in innate immune response which copes with broad range of pathogens in nonspecific way.
Pharmacological effect Pharmacological effects are defined as physiological or biochemical changes in the body produced by the drugs. For example, a painkiller can ease the pain and relieve the fever.
Pipeline Pipelines (clinical compound candidates, new drug candidates) are a group of drug candidates being developed from the discovery phase, preclinical/nonclinical stage and clinical trials up to marketing stages. Below are general schemes for the development of pipelines.
Preclinical development Preclinical development includes evaluative experiments in cells or animals for testing safety and efficacy of drugs before starting clinical trials in human.
Proof of Concept (POC) Proof of Concept (POC) is a demonstration to verify that a potential drug has a real potency by the administration to animals or human.
Psoriasis Psoriasis is one of skin diseases characterized by the skin lesions such as redness, scaly patches, papules and plaques. As it was reported to be accompanied by the overproduction of IFN, psoriasis is categorized as one of the autoimmune diseases.

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Q

Quality of life (QOL) Quality of life (QOL) is a perceived quality of an individual’s life and an evaluation of the well-being.

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R

Rare disease Rare disease refers to the disease with small prevalence. Some countries have the legal systems to support the researches that will expand the knowledge of such diseases and activities to develop novel therapies. The prevalence relating to the legal definitions varies among countries.
Research Project for the Specified Disease Treatment (offered by Japanese government) The Japanese Governmental Research Project for the Specified Disease Treatment which subsidizes medical support for patients of intractable diseases. The national or prefectural government partly covers the patients’ copayment of medical expenses by this program.
Rheumatoid Arthritis (RA) Rheumatoid Arthritis (RA) is a kind of autoimmune diseases showing swelling and pains in joints, consequently resulting in deformation and loss of function of joint. Conventional therapies include immune suppressants or disease-modifying antirheumatic drugs (DMARD) to ease pains, and to reduce swelling and inflammation.
RNA nucleotide drug RNA nucleotide drug is a category of drug generated with RNA.
RNAi technology RNAi is an abbreviation for RNA interference; RNAi technology refers to the inhibition of a specific gene expression based on the destruction of messenger RNA by the administration of double-strand RNA (siRNA).

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S

Side effect Side effects refer to undesirable secondary effects by administration of the medication. For example, some anti-cancer drugs are reported to cause loss of hair or anemia, etc.
Small interfering RNA (siRNA) Small interfering RNA (siRNA) is a class of double-stranded short RNA with specific sequence which can bind to the target mRNA. The destruction of target mRNA by the specific binding of siRNA will lead to the cancellation of genetic information transferred by the mRNA.
Small molecule drug Small molecule drugs are the drugs made of low molecular weight (300~500 Da) organic compounds. Many of therapeutic drugs are categorized as small molecules before antibody drugs were invented.
Steroidal Anti-Inflammatory Drug Steroids are a class of drugs with anti-inflammatory and immunosuppression used to treat a variety of medical conditions. The steroids are also notorious about the various side effects such as infection due to its strong immunosuppressive property.
Systemic Lupus Erythematosus (SLE) Systemic Lupus Erythematosus (SLE) is an autoimmune disease showing inflammation of unknown cause in various organs, such as joints, skins or other internal organs. Current treatments are mainly immunosuppressive drugs or drugs for symptomatic treatments; there is no exclusive cure available for SLE yet.

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T

T cell T cells are a type of lymphocytes that play a central role in immune response. Helper T cells to exert its function in immunity by the production of IFNs. Moreover, there is another type of T cells, cytotoxic T cells, which directly kill virus-infected cells or tumor cells.
Target molecule Target molecule refers to the molecule that drugs are designed to specifically bind to exhibit therapeutic effects. For example, a small chemical compound which specifically targets an enzyme involved in cancer cell proliferation may suppress cancer progression.
Toll-like Receptor (TLR) Toll-like receptors (TLRs) are a class of proteins expressed on the surface of animal cells that play key roles in innate immunity; they induce nonspecific innate immune responses upon recognition of molecules derived from viruses or bacteria.

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U

Unmet medical needs Unmet medical needs refer to the diseases without effective therapies.

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V

Vascular endothelial growth factor (VEGF) Vascular endothelial growth factor (VEGF) is a signal protein which activates vascular endothelial cells and induces cell proliferation during angiogenesis.

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W

Wet type Age-related Macular Degeneration (AMD) Age-related Macular Degeneration (AMD) is a medical condition of loss of vision in aged adults caused by the retinal damage, which may eventually leads to the loss of vision. Wet type refers to the symptom that blood vessels grow up from the choroid behind the retina and result in protein leakage below the macula.

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