バイオテクノロジー英語：必須語彙とフレーズ

"gene expression"

The process by which the information encoded in a gene is used to produce a functional product such as a protein

"The team measured gene expression levels using quantitative PCR to confirm that the target protein was being produced."

"recombinant DNA"

DNA that has been artificially created by combining genetic material from two or more sources

"The scientists inserted a human insulin gene into a bacterial plasmid to create recombinant DNA for large-scale protein production."

"plasmid"

A small, circular DNA molecule in a bacterium that can replicate independently of the chromosomal DNA

"The vector plasmid was transformed into E. coli cells, which then expressed the target therapeutic protein."

"transfection"

The introduction of foreign nucleic acids into eukaryotic cells to alter their genetic composition

"Lipid nanoparticles were used for transfection of the siRNA construct into HeLa cells to silence the oncogene."

"PCR (polymerase chain reaction)"

A technique used to amplify a specific segment of DNA through repeated cycles of heating and cooling

"PCR was performed to amplify the target gene fragment before ligation into the expression vector."

"Western blot"

A laboratory technique used to detect specific proteins in a sample after gel electrophoresis and membrane transfer

"A Western blot confirmed that the knockout cell line no longer expressed the BRCA1 protein."

"CRISPR-Cas9"

A genome-editing tool that allows precise modification of DNA sequences using a guide RNA and an endonuclease enzyme

"CRISPR-Cas9 was employed to introduce a point mutation in the target gene and study its effect on enzyme activity."

"sequencing"

The process of determining the precise order of nucleotides within a DNA or RNA molecule

"Next-generation sequencing revealed several off-target mutations introduced during the gene-editing procedure."

"lead compound"

A chemical compound that shows promise as a therapeutic agent and serves as a starting point for drug development

"After high-throughput screening, the team identified a lead compound with nanomolar potency against the target kinase."

"hit-to-lead"

The drug discovery phase in which promising screening hits are evaluated and optimised to generate lead candidates

"The hit-to-lead programme took eighteen months and reduced the initial 200 hits down to three viable lead compounds."

"ADMET"

Absorption, Distribution, Metabolism, Excretion, and Toxicity — the pharmacokinetic and safety properties of a drug candidate

"The compound failed early ADMET profiling due to poor metabolic stability in human liver microsomes."

"target engagement"

Evidence that a drug candidate interacts with and modulates its intended molecular target in a biological system

"CETSA assays were used to demonstrate target engagement of the inhibitor in live cancer cells."

"assay development"

The process of designing and validating a test system to measure the biological activity of compounds

"Robust assay development was critical before launching the high-throughput screen against the enzyme target."

"structure-activity relationship (SAR)"

The relationship between the chemical structure of a compound and its biological activity

"SAR studies guided the medicinal chemists in adding a fluorine substituent to improve potency tenfold."

"biologic"

A large, complex molecule derived from living organisms used as a therapeutic agent, such as a monoclonal antibody

"The biologic demonstrated superior efficacy compared to small-molecule inhibitors in the preclinical models."

"mode of action"

The specific biochemical interaction through which a drug produces its pharmacological effect

"Understanding the mode of action allowed the team to predict potential resistance mechanisms early in development."

"informed consent"

The process by which a participant voluntarily agrees to take part in a study after receiving full information about risks and benefits

"Investigators must obtain written informed consent from every participant before any study procedures begin."

"randomised controlled trial (RCT)"

A study design in which participants are randomly assigned to receive either the treatment or a control to reduce bias

"The Phase III randomised controlled trial enrolled 1,200 patients across 45 sites in twelve countries."

"placebo-controlled"

A trial design in which the control group receives an inactive substance to blind both participants and investigators to treatment allocation

"The double-blind, placebo-controlled study showed a statistically significant reduction in tumour volume at week 24."

"primary endpoint"

The main outcome measure pre-specified in the trial protocol that determines whether the study achieves its objective

"The primary endpoint of overall survival was met with a hazard ratio of 0.68 favouring the investigational arm."

"adverse event (AE)"

Any undesirable medical occurrence in a clinical trial participant, whether or not related to the study drug

"All grade 3 and above adverse events were reported to the sponsor within 24 hours of the investigator becoming aware."

"dose-escalation"

A clinical trial design in which successive cohorts of participants receive progressively higher doses to assess safety and determine the maximum tolerated dose

"The Phase I dose-escalation study identified 200 mg twice daily as the recommended Phase II dose."

"GCP (Good Clinical Practice)"

An international ethical and scientific quality standard for the design, conduct, recording, and reporting of clinical trials

"All site staff completed GCP training before the first participant was enrolled in the study."

"interim analysis"

A pre-planned statistical analysis conducted before the end of a trial to assess efficacy or safety at a defined time point

"The independent data monitoring committee recommended continuing the trial after a positive interim analysis at 50% of events."

"IND (Investigational New Drug)"

A regulatory application submitted to the FDA to request permission to test a new drug in humans before approval

"The company filed its IND application six months before the planned start of the Phase I study."

"BLA (Biologics License Application)"

A submission to the FDA requesting approval to market a biological product in the United States

"Following successful Phase III results, the regulatory team began compiling the BLA package for FDA submission."

"FDA approval"

Official authorisation from the U.S. Food and Drug Administration to market a drug or biologic for a specific indication

"The monoclonal antibody received FDA approval for second-line treatment of non-small cell lung cancer."

"EMA (European Medicines Agency)"

The European Union agency responsible for the scientific evaluation and supervision of medicines developed by pharmaceutical and biotech companies

"The parallel EMA and FDA review strategy was adopted to accelerate global market access for the new therapy."

"orphan drug designation"

A special status granted by the FDA or EMA to a drug intended to treat a rare disease, providing development incentives

"Orphan drug designation was granted for the gene therapy targeting a rare metabolic disorder affecting fewer than 10,000 patients."

"CMC (Chemistry, Manufacturing, and Controls)"

The section of a regulatory submission describing the composition, manufacture, and quality controls of a drug product

"The CMC team spent two years developing robust analytical methods to support the biologics licence application."

"breakthrough therapy designation"

An FDA programme designed to expedite development and review of drugs for serious conditions where preliminary evidence shows substantial improvement

"Breakthrough therapy designation was awarded after Phase Ib data showed an 80% overall response rate in heavily pre-treated patients."

"post-marketing surveillance"

The ongoing monitoring of the safety and effectiveness of a drug after it has been approved and is being used in the general population

"Post-marketing surveillance identified a rare immune-mediated adverse reaction not detected during clinical trials."

"whole-genome sequencing (WGS)"

The process of determining the complete DNA sequence of an organism's entire genome

"Whole-genome sequencing of the patient cohort revealed a novel somatic mutation associated with therapy resistance."

"single-cell RNA sequencing (scRNA-seq)"

A technique that measures gene expression across thousands of individual cells simultaneously

"Single-cell RNA sequencing resolved the heterogeneity of the tumour microenvironment into seven distinct cell populations."

"proteomics"

The large-scale study of all proteins expressed by a cell, tissue, or organism at a given time

"Proteomics analysis identified twenty differentially expressed proteins in treated versus untreated liver cancer cells."

"biomarker"

A measurable indicator of a biological state or condition that can be used for diagnosis, prognosis, or to predict treatment response

"PD-L1 expression level was validated as a predictive biomarker for response to the checkpoint inhibitor therapy."

"copy number variation (CNV)"

Differences in the number of copies of a particular segment of DNA among individuals

"Array comparative genomic hybridisation detected high-level amplification of the ERBB2 locus, indicating copy number variation in the tumour."

"epigenomics"

The study of chemical modifications to DNA and histone proteins that regulate gene expression without altering the DNA sequence

"Epigenomics profiling revealed widespread hypermethylation of tumour suppressor gene promoters in the patient samples."

"metagenomics"

The study of genetic material recovered directly from environmental or biological samples without prior cultivation of individual organisms

"Metagenomics of gut microbiome samples identified microbial signatures associated with improved immunotherapy outcomes."

"pathway analysis"

A bioinformatics approach used to identify which biological pathways are significantly enriched or affected in a dataset

"Pathway analysis of the differentially expressed genes highlighted dysregulation of the PI3K-AKT-mTOR signalling network."

"upstream processing"

The stages of biopharmaceutical manufacturing that involve growing cells and producing the biological molecule of interest

"The upstream processing team optimised the fed-batch culture conditions to increase monoclonal antibody titre to 8 g/L."

"downstream processing"

The stages of biopharmaceutical manufacturing that involve isolating, purifying, and formulating the final product

"Downstream processing using Protein A chromatography achieved a purity of greater than 99.5% for the therapeutic antibody."

"bioreactor"

A vessel or system in which biological reactions are carried out under controlled conditions for the production of biological products

"Cell density in the 2,000-litre bioreactor was monitored online using capacitance probes throughout the production run."

"GMP (Good Manufacturing Practice)"

A system of regulations ensuring that products are consistently produced and controlled according to quality standards

"The manufacturing facility received GMP certification from the EMA before the first commercial batch was released."

"cell bank"

A repository of cells stored under controlled conditions that serves as the source material for biopharmaceutical production

"A master cell bank and working cell bank were established and fully characterised prior to initiating process development."

"yield"

The amount of product obtained from a given manufacturing process, often expressed as a percentage of the theoretical maximum

"Process optimisation increased the overall yield from 45% to 72%, significantly reducing the cost of goods."

"technology transfer"

The process of moving a manufacturing process from one site or organisation to another while maintaining product quality

"Technology transfer from the development laboratory to the contract manufacturing organisation took nine months."

"contamination control"

Procedures and practices designed to prevent unwanted microorganisms or substances from compromising a biological process or product

"Stringent contamination control protocols, including regular environmental monitoring, ensured no microbial deviations over twelve production campaigns."