Antibody: A protein produced by the immune system that binds to a specific antigen.
Antibody-Drug Conjugate: An antibody that has a toxic drug, typically a chemotherapeutic agent, attached to it for target delivery to a specific tumor cell.
Antigen: A foreign substance which, when introduced into the body, stimulates an immune response.
Antisense: A short piece of nucleic acid whose sequence is complementary to another nucleic acid strand, referred to as the “sense” strand.
Biologics: Products of living organisms or cells used in the treatment or management of a disease.
Biologics Licensing Application (BLA): An application for marketing approval for a biologic drug; submitted to the FDA upon successful completion of Phase III clinical trials.
Biomanufacturing: The use of living cells to produce a biological product. An example is a therapeutic protein.
Biomarker: A physiological event or molecule that can be measured. Examples include the presence or absence of a protein or a mutated gene. Biomarkers are often used to indicate the presence or progression of a disease.
Biopharmaceutical: Drugs, either chemical compounds or biologics, made using the method of rational drug design.
Bispecific Antibody: An engineered protein composed of antigen-binding fragments from two different monoclonal antibodies. Bispecific antibodies can bind two targets simultaneously.
Biosimilar: A biologic drug that is produced using a different cell line, master cell bank, and/or different process than the one that originally produced the product.
Cell: The basic subunit of any living organism, typically containing at a minimum genetic material, an energy-producing system, and protein-making machinery, all surrounded by a membrane.
Cell Bank: A uniform population of cells, stored under defined conditions, typically frozen at -80 degrees Celsius or colder. The assumption is that each vial of cells is comparable, and may be used in a consistent manner after being thawed. See Master Cell Bank and Working Cell Bank.
Chimeric Antigen Receptor Therapy (CAR-T): A form of targeted cancer treatment that involves removing white blood cells from the patient’s body and engineering them to target a specific tumor antigen.
Companion Diagnostic: A diagnostic used by a physician to inform his prescribing decision.
Contract (or Clinical) Research Organization (CRO): A company that conducts preclinical or clinical trials for another company on a contract basis.
CRISPR/Cas9: A system that evolved in bacteria to help them destroy invading viruses and which has been adapted by the biotech industry for use in genome editing technology.
Deoxyribonucleic Acid (DNA): The molecule that encodes genetic information. DNA is a double-stranded helix held together by bonds between pairs of nucleotides.
Device: An instrument, apparatus, implement, machine, contrivance, or implant intended for use in the cure, mitigation, treatment, or prevention of disease.
Diagnostic (Dx): A test used to identify a disease or disorder, or to monitor the progression of treatment. Routine diagnostics are broad screening tools, whereas a specialty diagnostic screens for a specific disease.
Downstream Processing: The phase of a biomanufacturing campaign that consists of harvesting, purifying, and formulating the product.
Drug Development: The process of testing therapeutic molecules for safety and efficacy in animals and humans, and developing appropriate formulation, delivery, and manufacturing methods.
Drug Discovery: The process of identifying molecules with a therapeutic effect against a target disease.
Drug Target: Organ, tissue, or molecule involved in a disease that is modified or affected by a potential therapeutic.
Epigenetic Modification: Chemical modification made directly to a nucleotide base or to the histone protein around which the bases are wrapped. Epigenetic modification can change how much a gene is expressed. It is a normal part of development but irregularities are associated with various diseases.
Gene: A length of DNA that codes for a particular protein.
Gene Expression: The process by which the information in a gene is used to create proteins.
Gene Therapy: The insertion, alteration, or removal of genes within an individual’s cells to treat disease.
Generic Drug: A small molecule drug that is produced and distributed without patent protection.
Genetic Engineering: Altering the genetic material of cells or organisms in order to make them capable of producing new substances or performing new functions.
Genetic Predisposition: Susceptibility to a disease that is associated with a genetic mutation, which may or may not result in actual development of the disease.
Genetically Modified Organism (GMO): An organism whose DNA has been altered using genetic engineering techniques.
Genome: All of the genetic material in the chromosomes of a particular organism.
Genome Editing: A type of genetic engineering in which DNA is inserted, replaced, or removed from a genome using artificially engineered nucleases, or “molecular scissors.”
Genome-Wide Association Studies (GWAS): A study that compares DNA markers across the genome (the complete genetic material in a person) in people with a disease or trait to people without the disease or trait. These studies may uncover clues to help prevent, diagnose, and treat disease.
Genomics: The study of genes and their function, as well as the characterization of non-coding regions.
High Throughput Screening (HTS): The automated trial-and-error testing, typically using robotics, of very large sets of chemicals or materials.
Humanized Antibody: An antibody produced in a non-human species whose DNA sequence has been altered to make it more closely resemble a human antibody.
Immune System Checkpoint: Proteins on or in T-cells that inhibit their activity in order to prevent an overactive immune system.
Immune System Checkpoint Therapy: Therapeutics that lift the inhibitions on T-cells in order to make them more likely to attack cancer cells.
Induced Pluripotent Stem Cell (IPSC): A type of pluripotent stem cell artificially derived from a non-pluripotent cell, typically an adult somatic cell, by inducing a “forced” expression of specific genes.
Institutional Review Board (IRB): A committee that has been formally designated to approve, monitor, and review biomedical and behavioral research involving humans with the aim to protect the rights and welfare of the research subjects.
International Conference on Harmonisation of Technical Requirements for Registration of Pharmaceuticals for Human Use (ICH): A panel consisting of representatives from the pharmaceutical industry and the regulatory agencies of the United States, Europe, and Japan. The goal of the ICH is to make recommendations in order to reduce duplications in the drug testing process in each country.
Investigational New Drug (IND): A drug which has gained FDA approval to be shipped across state lines, typically for clinical trials, but has not yet gained approval for marketing.
Large Molecule Drug: Another name for protein therapeutics. Large molecule drugs are too large to enter cells.
Master Cell Bank: A culture of fully characterized cells distributed into separate vials, processed together in such a manner as to ensure uniformity. The master cell bank is usually stored at -80 degrees or colder (liquid nitrogen), and at two geographically distinct locations.
Microbiome: The totality of microbes, their genomes, and environmental interactions in a particular environment.
MicroRNA (µRNA): A small non-coding RNA that blocks the translation of target RNAs.
Monoclonal Antibody (mAb): An antibody produced by a single clone of cells, which therefore consistently binds to the same epitope of an antigen.
New Drug Application (NDA): An application for marketing approval for a small molecule drug; submitted to the FDA upon successful completion of Phase III clinical trials.
Non-Coding DNA: Chromosomal DNA that is found between genes and does not code for proteins.
Nucleic Acid: One of the family of molecules which includes the DNA and RNA molecules.
Nucleotide: The “building block” of nucleic acids, such as DNA and RNA molecules. A nucleotide consists of one of five bases – adenine, guanine, cytosine, thymine, or uracil – attached to a sugar-phosphate group.
Orphan Drug: A drug developed for a condition that affects fewer than 200,000 individuals in the U.S.
Patent: A set of rights issued by the government, which allows the patent holder to exclude others from making, using, selling or distributing a patented invention for a certain term.
Pharmaceutical: A substance intended for use in the cure, mitigation, treatment, or prevention of disease.
Pharmacodynamics (PD): The study of the effect of a drug on the body; in particular, the effect of the drug as it relates to increasing dose.
Pharmacogenomics: The science of understanding the correlation between an individual patient’s genetic make-up (genotype) and their response to drug treatment. Some drugs work well in some patient populations and not as well in others. Studying the genetic basis of patient response to therapeutics allows drug developers to more effectively design therapeutic treatments.
Pharmacokinetics (PK): The study of drug absorption, drug distribution within the body, drug metabolism, and drug excretion.
Phase I Clinical Trial: Initial studies to determine the metabolism and pharmacologic actions of drugs in humans, the side effects associated with increasing doses, and to gain early evidence of effectiveness; may include healthy participants and/or patients.
Phase II Clinical Trial: Controlled clinical studies conducted to evaluate the effectiveness of the drug for a particular indication or indications in patients with the disease or condition under study and to determine the common short-term side effects and risks.
Phase III Clinical Trial: Expanded trials after preliminary evidence suggesting effectiveness of the drug has been obtained, and are intended to gather additional information to evaluate the overall benefit-risk relationship of the drug and provide an adequate basis for physician labeling.
Pluripotent: Capable of differentiating into all cell types except for placental cells. Embryonic stem cells are pluripotent.
Polymerase Chain Reaction (PCR): A method for creating millions of copies of a particular segment of DNA.
Preclinical Studies: The testing of experimental drugs in the test tube or in animals – the testing that occurs before trials in humans may be carried out.
Protein: A biological molecule that consists of many amino acids linked together by peptide bonds. As the chain of amino acids is being synthesized, it is also folded into higher order structures. Proteins are required for the structure, function, and regulation of cells, tissues, and organs in the body
Proteome: The complete set of proteins expressed by a cell or organism.
Proteomics: The study of the proteome.
Quantitative Reverse Transcriptase PCR (qRT-PCR): A technique used to measure the levels of gene expression of a particular gene.
Rational Drug Design: The development of new therapeutics based on an understanding of the underlying disease mechanism.
Receptor: A protein usually found on the surface of a cell that binds to a specific chemical messenger, such as a neurotransmitter or hormone.
Recombinant DNA (rDNA): DNA molecules that have been created by combining DNA from more than one source.
Recombinant Protein: A protein that has been produced using recombinant DNA.
Restriction Enzyme (RE): A protein that recognizes specific, short nucleotide sequences and cuts DNA at those sites.
Reverse Transcriptase (RT): An enzyme used to form a complementary DNA sequence (cDNA) from an RNA template.
Ribonucleic Acid (RNA): A nucleic acid similar to DNA but based on the sugar ribose and containing the nucleotides guanine, adenine, uracil, and cytosine instead of guanine, adenine, thymine, and cytosine, and typically single-stranded.
RNA Interference (RNAi): A technique used to block the expression of a particular protein.
Scale Up: The process of slowly increasing the volume of a cell culture from a few milliliters to several thousand liters.
Scientific Advisory Board (SAB): A group of independent scientists who advises a company’s management regarding scientific direction for the company.
Sequence: The order of nucleotides in a DNA or RNA molecule, or the order of amino acids in a protein.
Short Interfering RNA (siRNA): Short, synthetic pieces of RNA used to block the expression of a target mRNA in RNA interference.
Signaling Molecule: A molecule that binds to a cellular receptor. This binding usually results in the cell starting or stopping the production of a particular protein.
Single Nucleotide Polymorphism (SNP): A difference in one base pair between two DNA sequences.
Small Molecule Drug: A drug that is chemically synthesized in the lab. Small molecule drugs are small enough to enter cells.
Stem Cell: Undifferentiated cells that have the ability to self-renew and to differentiate into one or more specific cell type in response to extracellular cues.
Stratified Medicine: The practice of separating patient populations into different groups based on the genetic signature of their disease. This enables the development and use of targeted therapeutics.
Susceptibility Gene: A gene that predisposes someone to a particular disease.
T-Cell: An immune system cell that recognizes specific pathogens based on the shape of its cell-surface receptor.
Target Validation: Determining if targeting a particular molecule thought to be involved in a disease mechanism will be a safe and effective means of therapy.
Technology Transfer: The process of transferring discoveries made by basic research institutions to the commercial sector, to be developed into useful products and services.
Transcription: The process during which the information in a length of DNA is used to construct an mRNA molecule.
Transgenic Organism: An organism whose genome has been altered by the incorporation of foreign DNA.
Translation: The process during which the information in mRNA molecules is used to construct proteins.
Trispecific Antibody: An antibody capable of binding to three different targets at once.
Upstream Processing: The phase of biomanufacturing that consists of establishing cell banks and seeding and scaling up cell cultures.
Unipotent: Capable of developing into only one type of cell or tissue.
Uracil (U): One of the four chemical building blocks (nucleotides) that makes up RNA.
Vaccine: A substance used to stimulate the immune response against diseases, prepared by delivering the causative agent of the disease, or a synthetic substitute, treated to act as an antigen without inducing the disease.
Vector: A vehicle for the transfer of DNA from one organism to another.
Working Cell Bank: A cell bank that is established from one of the master cell bank vials.
