Biotechnology Glossary


Abbreviated New Drug Application (ANDA)

a simplified submission permitted for a generic version of an approved drug.

Absorption, Distribution, Metabolism, Excretion and Toxicology (ADMET)

an element of pre-clinical and clinical trials used to measure the effects of a drug on animal and human physiology.

Accelerated development and review

a process designed to speed the development of drugs that promise significant benefits over existing therapies or treat serious illnesses for which no therapy exists.

Action letter

An official FDA communication that informs the sponsor of an NDA or BLA of a decision by the agency. An approval letter allows commercial marketing of the product.


One of the four bases found in DNA and in RNA. It pairs with thymine in DNA, and with uracil in RNA.


Are medium-sized (90-100 nm), non-enveloped icosahedral viruses composed of a nucleocapsid and a double-stranded linear DNA genome.


A component of the body's immune response. A Y-shaped protein, it is secreted in response to an antigenic stimulus. It neutralizes the antigen it by binding to it.


Is a genus of gram-negative bacteria that uses horizontal gene transfer to cause tumors in plants.


Is one member of a pair or series of different forms of a gene.


Is a non-parasitic antigen capable of stimulating a hypersensitity reaction in atopic individuals.


agreement between two or more companies to cooperate in some way.

Amino acid

building block of proteins. Proteins consist of amino acids linked end-to-end. There are 20 different amino acid molecules that make up proteins. The messenger RNA tells the cell what amino acids are needed and what order they must be arranged in to build a particular protein.

Angel investor

wealthy individual who personally provides startup capital to very young companies to help them grow.


The process by which the body forms and develops new blood vessels.


Molecule that bond to the receptor site of a protein. Unlike agonists, antagonists suppress or inhibit the function of the protein to which they bind.


a chemical substance that can kill or inhibit the growth of a microorganism.


immune system protein produced by humans and higher animals to recognize and neutralize bacteria, viruses, cancerous cells, and other foreign compounds.


Is a unit made up of three nucleotides that can base-pair to one or more codons for an amino acid.


Is a substance that prompts the generation of antibodies and can cause an immune response.


a natural or synthetic DNA or RNA molecule that specifically binds with messenger RNA to selectively inhibit expression of a single gene.


The cell's self-destruct mechanism, also known as "programmed cell death." In this process, the cell dies via "self-digestion," without rupturing and releasing its contents into the surrounding tissue.


A technique using photography to detect radioactively-labeled molecules by placing molecular material on a piece of film, suspending movement, and exposing the film.

Applied research

aimed at gaining knowledge or understanding to determine the means by which a specific recognized need may be met. Applied research builds upon the discoveries of basic research to enable commercialization.


Bacillus thuringiensis

a naturally occurring bacteria that produces Bt toxin, a protein that is toxic to certain kinds of insects. The Bt toxin gene has been genetically engineered into corn and cotton plants to reduce the need for chemical pesticides.


Are a large group of unicellular microorganisms. Typically a few micrometers in length, bacteria have a wide range of shapes, ranging from spheres to rods and spirals.

Barrier to entry

a condition that makes it difficult for competitors to enter the market; e.g. patent, trademark, high initial investment requirement.


A key component of DNA and RNA molecules. Four different bases are found in DNA: adenine (A), cytosine (C), guanine (G) and thymine (T). In RNA, uracil (U) substitutes for thymine.

Basic research

aimed at gaining more comprehensive knowledge or understanding of the subject under study, without specific applications in mind. Basic research advances scientific knowledge but does not have specific immediate commercial objectives, although it may be in fields of present or potential commercial interest.

Bayh-Dole Act

provides the statutory basis and framework for federal technology transfer activities, including patenting and licensing federally funded inventions to commercial ventures.


The totality of genes, species, and ecosystems of a region.


demonstration that a generic drug has the same chemical and biological properties as its pioneer counterpart.


the application of information technology to manage and analyze the vast amounts of data generated from biological research.


medicine made by biological processes rather than by chemical synthesis or extraction. Biologics typify biotechnology-derived drugs. Contrast with small-molecule drugs.

Biologics License Application (BLA)

application filed with the FDA Center for Biologics Evaluation and Research (CDER) for approval to market a biologic drug.


the use of biological systems, usually microorganisms, to decompose or sequester toxic and unwanted substances in the environment.


the application of molecular biology for useful purposes.


drug with &63;1 billion or more in sales.


starting a business with little or no external funding.

Brand-name drug

the patented version of a drug. Contrast with generic drugs.

Bridge loan

a short-term, high-interest loan provided by venture capitalists to companies in dire need of cash.

Burn rate

The rate at which an unprofitable company is going through its available money.



Is the process in which the rate of a chemical reaction is either increased or decreased by means of a chemical substance known as a catalyst. Unlike other reagents that participate in the chemical reaction, a catalyst is not consumed by the reaction itself. The catalyst may participate in multiple chemical transformations. Catalysts that speed the reaction are called positive catalysts. Catalysts that slow down the reaction are called negative catalysts or inhibitors.


The basic organizational unit of all living organisms.

Cell line

A biological group of individual cells that are genetically identical as a result of dividing mitotically from a single progenitor cell.

Cell therapy

Is the process of introducing new cells into a tissue in order to treat a disease.


A complex of DNA, RNA and proteins within the cell nucleus, out of which chromosomes condense during cell division.


the DNA-protein complexes that contain all the genes in a cell.

Clinical pharmacology study

clinical trial designed to determine the absorption, distribution, metabolism, elimination, and toxicity (ADMET) of a drug.

Clinical trial

human study designed to measure the safety and efficacy of a new drug.


A living organism produced asexually from a single ancestor, to which it is genetically identical.


The production of a cloned embryo by transplanting the nucleus of a somatic cell into an ovum


a sequence of three DNA or RNA bases that specifies an amino acid in the synthesis of a protein.

Combinatorial chemistry

a product discovery technique that uses robotics and parallel chemical reactions to generate and screen as many as several million molecules with similar structures in order to find chemical molecules with desired properties.

Competitive advantage

an advantage that a firm has relative to competing firms; may be in the form of intellectual property, expertise, partnerships, assets, etc.


Present since birth.

Controlling interest

ownership of more than 50 percent of a company's voting shares.


Securities (usually bonds or preferred shares) that can be converted into common stock.

Cooperative research and development agreement (CRADA)

an agreement enabling federally funded laboratories to perform for-profit contract work for commercial firms.


agreement in which two or more firms with competing and similar technologies strike a deal to reduce the need for legal actions to clarify who is to profit from applications of the technology.

Culture medium

A liquid or gel containing nutrients that is used to cultivate microorganisms.


Is the part of a cell that is enclosed within the plasma membrane. The cytoplasm has three major elements; the cytosol, organelles and inclusions.


A base, which pairs with guanine in DNA and RNA.


Data mining

using computers to analyze masses of information to discover trends and patterns.


It is a constituent of the nucleotides that comprise the biopolymer or DNA. A derivative of the pentose sugar ribose in which the 2' hydroxil is reduced to a hydrogen.


a product used for the diagnosis of a disease or medical condition.


the decrease in relative ownership among existing investors as additional shares are issued.

Discovery rights

selling only research findings while keeping rights to all the knowledge that is uncovered along the way.

DNA (deoxyribonucleic acid)

the primary source of genetic information in cells. DNA is made up of nucleotides and is composed of two strands wound around each other, called the double helix.

DNA complementary (cDNA)

Is DNA synthesized from a mature mRNA template in a reaction catalyzed by the enzyme reverse transcriptase.

DNA fingerprinting

a DNA analysis method that measures genetic variation among individuals. This technology is often used as a forensic tool to detect differences or similarities in blood and tissue samples at crime scenes.

DNA Polymerase

It is an enzyme that assist DNA replication

DNA sequencing

the process of determining the exact order of bases in a segment of DNA.


an experimental protocol whereby neither the experimental subjects nor the administrators know whether a drug or placebo is being administered. Double-blind protocols are used to eliminate bias.

Down round

a financing event in which a company is valued lower than it was previously.

Drug delivery

the process by which a formulated drug is administered to the patient.

Drug development

the process of taking a lead compound, demonstrating it to be safe and effective for use in humans, and preparing it for commercial-scale manufacture.

Due diligence

the process by research is conducted to determine the value of an investment, licensing agreement, merger, or other similar activity.

Dumb money

funding from investors who cannot provide additional benefits such as guidance or networking.



It is the branch of biology dealing with the relationships of organism with their environment and with each other.


The interconnectedness of organisms (plants, animals, microbes) with each other and their environment.

Elevator pitch

a short, typically less than two-minute, summary used to quickly describe a business to investors


A functional protein that catalyzes (speeds up) a chemical reaction. Enzymes control the rate of naturally occurring metabolic processes such as those necessary for growth and reproduction.

Equity investment

an investment buying partial ownership of a company.

Escherichia coli (E. coli)

a common gut bacterium that is a workhorse and model organism for molecular biology.

Essential amino acid

Is an amino acid that cannot be synthesized by the organism (usually referring to humans), and therefore must be supplied in the diet.


It is a term that applies to all organisms whose cells have a nucleus.

Eukaryotic Cell

Is a cell with nucleus.


an inactive ingredient (there are no absolutely inert excipients) added to a drug to give it a pill form or otherwise aid in delivery.


a temporary FDA-granted monopoly, distinct from patent or other intellectual property protection. Exclusivity may be granted for developing drugs for rare diseases, novel drugs, conducting pediatric clinical trials, or successfully challenging invalid patents.


Is the region of a transcribed gene present in the final functional RNA molecule.


a highly specific process in which a gene is switched on at a certain time and begins production of its protein, resulting in the manifestation of a characteristic that is specified by a gene. Genetic predispositions to disease can occur, for example, if a person carries the gene for a disease but it is not expressed.


False negative

an experimental outcome that incorrectly yields a negative result. False negatives can complicate disease diagnosis.

False positive

an experimental outcome that incorrectly yields a positive result. False positives can frustrate assessing the performance of lead compounds.

Fast Track

a process for interacting with the FDA during drug development, intended for drugs to treat serious or life threatening conditions that demonstrate the potential to address an unmet medical need.


An anaerobic biochemical reaction in which an enzyme catalyzes the conversion of one substance into another.

First-in-man study

Phase I trial primarily concerned with establishing the safety of a compound.

Functional genomics

the use of biological experiments and database searches to establish what each gene does, how it is regulated, and how it interacts with other genes.

Functional foods

foods containing compounds with beneficial health effects beyond those provided by the basic nutrients, minerals and vitamins.



the fundamental unit of heredity, a section of DNA which codes for a defined biochemical function. Some genes direct the synthesis of proteins, while others have regulatory functions.

Gene Expression

The transcription and translation of a gene into a messenger RNA and thus into a protein.

Gene therapy

the replacement of a defective gene in a person or organism suffering from a genetic disease.

Generic drug

the version of an approved drug produced by a competitor after a pioneer firm's patents expires.

Genetic code

the language in which DNA's instructions are written. The genetic code consists of triplets of nucleotides (codons), with each triplet corresponding to one amino acid in a protein structure or a signal to start or stop protein production.

Genetic disorder

a condition or mutation that results from one or more defective genes.

Genetic engineering

the manipulation of genes to create heritable changes in biological organisms and products that are useful to people, living things, or the environment.

Genetic predisposition

a susceptibility to disease that is related to a genetic condition, which may or may not result in actual development of the disease.

Genetic screening

the use of a specific biological test to screen for inherited diseases or medical conditions.


The branch of biology that deals with the transmission and variation of inherited characteristics, in particular chromosomes and DNA.


the sum total of an organism's genes.


the study of genes and their function.


A group of organisms having the same genetic constitution.


The reaction of a saccharide with a hydroxy or amino functional group to form a glycoside.

GMO (Genetically Modified Organism)

Organism whose genetic material has been modified in an artificial way.

Good Manufacturing Practice (GMP)

guidelines ensuring the quality and purity of chemical products that are intended for use in pharmaceutical applications, and controls ensuring that methods and facilities used for production, processing, packaging, and storage result in drugs with consistent and sufficient quality, purity, and activity.


It is a nucleic base and pairs with cytosine in DNA and RNA.


Hatch-Waxman Act

contains provisions to foster the development of generic drugs and support pioneer drug development.


A nucleic acid composed of two chains with each derived from a different parent molecule.


A substance produced by a tissue and conveyed by the bloodstream to another to affect the physiological activit.

Host cell

Is a living cell in which a virus reproduces.

Human Genome Project

an international research effort that identified and located the full sequence of bases in the human genome.


The process of generation of a molecule, cell or organism combined with genetic material from different organisms.


Hybrid cell engineered from a lymphocyte and a tumor cell used to generate monoclonal antibodies.


Immune system

the cells, biological substances (such as antibodies), and cellular activities that work together to recognize foreign substances and provide resistance to disease.

In silico ('in computer')

computer-based predictions that can complement in vitro and in vivo procedures.

In vitro ('in glass')

experimental procedures carried out in test-tubes, beakers, etc.

In vivo ('in the living body')

experimental procedures carried out on living cell lines or in living animals.

Initial Public Offering (IPO)

the initial sale of shares of a private company on public markets, turning it into a publicly-traded company.

Institutional Review Board (IRB)

an independent committee of scientists, physicians, and lay people that oversees clinical trials.

Intellectual property

intangible assets such as patents, trade secrets, trade names, etc.


Any of a group of glycoproteins, produced by the immune system that prevents viral replication in infected cells.


A portion of split gene that is included in pre-RNA transcripts but is removes during RNA processing and rapidly degraded.

Investigational New Drug (IND)

an application to pursue clinical trials with an experimental drug that has passed pre-clinical trials.


Kilobase (KB)

A length of single stranded RNA containing one thousand nucleotides (1KB = 1000 bases).


Lead compound

in pre-clinical trials and clinical trials, a potential drug being tested for safety and efficacy.


an agreement to grant rights to a patent or tangible subject.


An enzyme that catalyzes the binding of two molecules.


Any of a group of organic compounds including: fats, oils, waxes, sterols, nucleic acids and triglycerides. Lipids are characterized by being insoluble in water, and account for most of the fat present in the human body. They are, however, soluble in non-polar organic solvents.


an artificial membrane. Can be used to encapsulate drugs and aid in drug delivery.

Lymphocyte B

A type of white blood cell or leukocyte, which produces antibodies in the humoral immune response.

Lymphocyte T

A type of white blood cell or leukocyte which participates in the cell-mediated immune response.


Mass spectrometry

It is an analytical technique that measures the mas/charge ratio of the ions formed when a molecule or atom is ionized, vaporize and introduced into a vacuum.

mRNA (Messenger RNA)

a ribonucleic acid molecule that transmits genetic information from DNA to the protein synthesis machinery in cells, where it directs protein synthesis.


a tool that permits the identification of DNA samples and examination of gene expression in individual tissues and different conditions.


Any microorganism, but specially said of those microorganisms which are harmful types of bacteria.


An organism that is too small to be seen by the eye, especially a single-celled organism, such as a bacterium, virus, algae, fungi, or protozoa.

Molecular evolution

the process of making discrete changes in genes to improve the functional characteristics of proteins and enzymes.

Molecular farming

using biotechnology to produce useful products from domesticated plants and animals.

Monoclonal antibody

an antibody that recognizes a single target. Polyclonal antibodies recognize several related targets.


A relatively small molecule which can be covalently bonded to other monomers to form a polymer.


a cell or organism harboring one or more mutated genes.


a change in the base sequence of a gene that results in it not performing its normal task in a cell.



White blood cell.

New Drug Application (NDA)

application filed with the FDA Center for Drug Evaluation and Research (CDER) for approval to market a small-molecule drug.

Northern Blot

A technique used to identify RNA fragments.

Nuclear Magnetic Resonance (NMR)

The absorption of electromagnetic radiation (radio waves), at a specific frequency, by an atomic nucleus placed in a strong magnetic field; used in spectroscopy and in magnetic resonance imaging.


Enzyme capable of cleaving the phosphodiester bonds between the nucleotide subunits of nucleic acids.

Nucleic Acid

Is a macromolecule composed of chains of monomeric nucleotides.


It is an organic molecule in which a nitrogenous heterocyclic base, which can be either a double-ringed (purine) or a single-ringed (pyrimidine), is covalently attached to a five-carbon pentose sugar (deoxyribose in DNA or ribose in RNA).


one of the structural components, or building blocks, of DNA and RNA. A nucleotide consists of a base plus one molecule of sugar and phosphoric acid.


A large organelle found in cells which contains the genetic material.


Off-label use

Use of a drug not in accordance with FDA-approved uses or drug labelling. Physicians are free to use drugs for off-label uses.


Any gene that contributes to the conversion of a normal cell into a cancerous cell when mutated or expressed at high levels.


viruses, chemicals, genes, proteins, etc. that cause the formation of tumors.

Orange Book

also known as the Approved Drug Products with Therapeutic Equivalence Evaluations, the Orange Book contains detailed information on all approved drugs and must list all extant patents.

Orphan Drug

a drug that treats a disease that affects less than 200,000 Americans or for which there is no reasonable expectation that the cost of research and development will be recovered from sales in the United States. The Orphan Drug Act provides special incentives for producers of orphan drugs.



A declaration issued by a government agency declaring someone the inventor of a new invention and having the privilege of stopping others from making, using or selling the claimed invention.


a disease-causing organism.


A class of organic compounds consisting of various numbers of amino acids in which the amine of one is reacted with the carboxylic acid of the next to form an amide bond.


Study of the cost-benefit ratios of drugs.


examination of the genetic basis for variation in response to therapeutics by different individuals.


the process of farming genetically engineered animals and plants to produce drugs.

Phase I

clinical trial designed primarily to determine the safety of an experimental drug.

Phase II

clinical trial that evaluates an experimental drug's safety, assesses side effects, and establishes dosage guidelines.

Phase III

clinical trial designed to verify the safety and effectiveness of an experimental drug. Success in Phase III trials can lead to marketing approval.

Phase IV

post-approval clinical trials used to monitor safety and efficacy or examine additional applications of drugs.


The characteristic of an organism, such as its morphological, developmental, biochemical or physiological properties, or its behavior.

Pioneer (brand-name) drug

the patented version of a drug. Contrast with generic drugs, the competing versions produced when pioneer patents expire.

PIPE (Private Investment in Public Equity)

purchase of discounted shares in a public company in which payment goes directly to the company rather than to existing shareholders.


a mock-treatment used in single-blind or double-blind experiments to eliminate bias from experiment subjects or administrators, respectively.


A circle of double-stranded DNA that is separate from the chromosomes, and which is found in bacteria and protozoa.

Platform technology

a technique or tool that enables a range of scientific investigations. Examples include combinatorial chemistry for producing novel compounds, microarrays for gene expression analysis, and bioinformatics programs for data assembly and analysis.


A long or larger molecule consisting of a chain of many repeating units, formed by chemically bonding together many identical or similar small molecules called monomers.

Polymerase Chain Reaction (PCR)

a method to produce sufficient DNA for analysis from a very small amount of DNA.

Preclinical studies

studies that test a drug on animals and nonhuman test systems. Safety information from such studies is used to support an investigational new drug application (IND).


A single-stranded nucleic acid molecule required for the initiation of replication of a DNA molecule.


a naturally occurring protein that can be converted into a disease-causing form. Prion diseases can be transmitted in the absence of DNA or RNA.


The term prokaryota is applicable to all organisms whose cells do not contain a nucleus (bacteria and viruses).

Prokaryotic Cell

Cell without nucleus.


a DNA sequence preceding a gene that contains regulatory sequences influencing the expression of the gene.

Proof of principle

demonstration of the commercial potential of a discovery or invention.


a long-chain molecule consisting of amino acids that folds into a complex three-dimensional structure. The type and order of the amino acids in a protein is specified by the nucleotide sequence of the gene that codes for the protein. The structure of a protein determines its function.


the study of the protein profile of each cell type, protein differences between healthy and diseased states, and the function of and interaction among proteins.


Quantitative PCR (q-PCR)

Also called Quantitative real time polymerase chain reaction (Q-PCR/qPCR) or kinetic polymerase chain reaction, is a laboratory technique based on the polymerase chain reaction, which is used to amplify and simultaneously quantify a targeted DNA molecule. It enables both detection and quantification of a specific sequence in a DNA sample.



an anti-dilution provision where an investor is granted additional shares of stock without charge if the company later sells the shares at a lower price.

Rational drug design

using the known three-dimensional structure of a molecule, usually a protein, to design a drug that will bind it.

Recombinant DNA

the DNA formed by combining segments of DNA from different sources.

Restriction enzyme

a protein that cuts DNA molecules at specific sites, dictated by the nucleotide sequence.


a type of virus that reproduces by converting RNA into DNA.

Return On Investment (ROI)

profit (or loss) on an investment, often expressed as a percentage.

Reverse transcriptase

Is a DNA polymerase enzyme that transcribes single-stranded RNA into single-stranded DNA.

Ribonucleic acid (RNA)

A molecule with similar structure to DNA, but with a primary function of protein synthesis.


A naturally occurring pentose sugar, which is a component of the nucleosides and nucleotides that comprise the nucleic acid biopolymer, RNA.


Small organelles found in all cells involved in the production of proteins by translating messenger RNA.


Is a fragment of RNA that can act as an enzyme.

RNA interference

using antisense techniques to selectively inhibit expression of a gene.


the payment of a percentage of sales as compensation to product developers, patent licensors, or even investors.


Salami slicing

filing for multiple orphan drug designations on the same drug.

SBIR (Small Business Innovation Research)

a funding program that encourages small business to explore their technological potential and provides the incentive to profit from its commercialization.

Scientific Advisory Board (SAB)

a group of esteemed scientists and business professionals, independent from management that provides objective feedback and guidance on a company's progress and goals.

Single Nucleotide Polymorphism (SNP)

a single base difference in the sequence of a gene which alters the structure and function of the gene product.

Small-molecule drug

a drug produced using defined chemical synthesis or extraction. Contrast with biologics, drugs produced by biological processes.

Southern Blot

A technique whereby biological molecules (especially DNA fragments) are separated by electrophoresis and then transferred from a gel to an immobilizing medium, under the influence of an electric field, for analysis.


separating a smaller unit from an established company, permitting each company to retain focus while shielding the parent from risk and granting the spin-off the administrative benefits of small size.

Stem cell

an undifferentiated cell that can multiply and become any sort of cell in the body.

STTR (Small Business Technology Transfer)

A funding program that encourages public/private sector partnership to develop new technologies and profit from their commercialization.

Surrogate marker

an indirect measure of effectiveness, such as a laboratory test or tumor shrinkage, used to show a strong potential for effectiveness in accelerated drug approval.



Either of the sequences of DNA at each end of a eukaryotic chromosome.

Technology transfer

the transfer of discoveries made by basic research institutions, such as universities and government laboratories, to the commercial sector for development into useful products and services.

Tissue engineering

the production of natural or synthetic organs and tissues that can be implanted as fully functional units or may develop to perform necessary functions following implantation


A base, obtained by applying sulphuric acid to thymic acid; it pairs with adenine in DNA.


The protein responsible for the functional specifity of certain bacteria and is toxic for some organisms.


the synthesis of an mRNA molecule as a copy of a gene. In gene expression, transcription precedes translation.


the synthesis of a protein based on the nucleotide sequence of an mRNA molecule, which corresponds to the sequence of a gene.


Is the process of introducing nucleic acids into cells by non-viral methods.

Transfer RNA (tRNA)

A short-chain version of RNA that transfers individual amino acids to ribosomes during protein synthesis.


an organism with one or more genes that have been transferred to it from another organism.



One of the bases of RNA which pairs with adenine and is symbolized by U.



a preparation of either whole disease-causing organisms (killed or weakened) or parts of such organisms, used to confer immunity against the disease that the organisms cause. Vaccine preparations can be natural, synthetic, or derived by recombinant DNA technology.


Vehicle used to transfer genetic material to a target cell.

Venture capital

money invested by venture capitalists in startup companies in exchange for equity.

Venture capitalist

an individual who invests in start-up companies with the intent of making a large return on investment.


A submicroscopic infectious organism, now understood to be a non-cellular structure consisting of a core of DNA or RNA surrounded by a protein coat that requires a living cell to replicate - often causes disease in the host organism.


Western blot

Is an analytical technique used to detect specific proteins in a given sample of tissue homogenate or extract.


X-ray crystallography

an essential technique for determining the three-dimensional structure of biological molecules.


transplanting a foreign tissue into another species.



A type of single-celled fungus important for their capacity to perform the fermentation.