Biotech acronyms and terms

Biotech teams lean on dense shorthand across research, clinical and regulatory work. These definitions are short and practical, to help new hires and cross-functional colleagues follow the conversation.

ADC

Antibody-drug conjugate: an antibody linked to a cytotoxic drug so the drug is delivered to cells carrying a target antigen.

ADME

Absorption, distribution, metabolism and excretion: the four processes that determine how a drug moves through the body.

AE

Adverse event: any unfavorable medical occurrence in a trial participant, whether or not it is caused by the treatment.

ANDA

Abbreviated New Drug Application: a US filing to market a generic drug by showing it is equivalent to an approved one.

API

Active pharmaceutical ingredient: the component of a drug that produces the intended biological effect.

BLA

Biologics License Application: the US request for permission to market a biological product.

cDNA

Complementary DNA: DNA synthesized from an RNA template using reverse transcriptase.

CAR-T

Chimeric antigen receptor T-cell therapy: a patient's T cells are engineered to recognize and attack a target such as a tumor antigen.

CDMO

Contract development and manufacturing organization: a company that develops and produces drugs on behalf of sponsors.

CMC

Chemistry, manufacturing and controls: the regulatory section covering how a drug is made, characterized and kept consistent.

COA

Certificate of analysis: a document reporting the test results that show a batch meets its specifications.

CRISPR

Clustered regularly interspaced short palindromic repeats: a family of DNA sequences, and the related Cas-based tools used to edit genes.

CRO

Contract research organization: a company that runs trials or studies for a sponsor under contract.

DMC

Data monitoring committee: an independent group that reviews accumulating trial data for safety and efficacy.

DNA

Deoxyribonucleic acid: the molecule that carries genetic information in cells.

ELISA

Enzyme-linked immunosorbent assay: a plate-based test that detects and quantifies a protein using antibodies and an enzyme signal.

EMA

European Medicines Agency: the EU body that evaluates and supervises medicines.

FDA

Food and Drug Administration: the US agency that regulates drugs, biologics and medical devices, among other products.

FIH

First-in-human: the first study in which a new drug is given to people.

GCP

Good clinical practice: the ethical and scientific quality standard for designing and running trials with human participants.

GLP

Good laboratory practice: quality rules for nonclinical safety studies so their data are reliable and traceable.

GMP

Good manufacturing practice: regulations that require products to be consistently produced and controlled to quality standards.

IC50

Half-maximal inhibitory concentration: the concentration of a compound that reduces a biological activity by half.

ICH

International Council for Harmonisation: the body that issues shared technical guidelines for drug registration.

IND

Investigational New Drug application: the US filing that allows a new drug to be given to people in clinical trials.

IRB

Institutional review board: a committee that reviews human-subject research to protect participants.

mAb

Monoclonal antibody: an antibody made by identical immune cells that all derive from one parent cell, binding a single target.

MTD

Maximum tolerated dose: the highest dose that does not cause unacceptable toxicity.

NDA

New Drug Application: the US request to approve and market a new small-molecule drug.

NGS

Next-generation sequencing: high-throughput methods that sequence many DNA fragments in parallel.

PCR

Polymerase chain reaction: a method that amplifies a specific DNA segment through repeated heating and cooling cycles.

PD

Pharmacodynamics: what a drug does to the body, including its biochemical and physiological effects.

PK

Pharmacokinetics: what the body does to a drug over time; concentration in blood is the usual measure.

PoC

Proof of concept: early evidence that a treatment approach works in the intended setting.

QC

Quality control: testing of materials and products to confirm they meet specifications.

RNA

Ribonucleic acid: a molecule that helps carry out the instructions encoded in DNA, including making proteins.

SAE

Serious adverse event: an adverse event that is life-threatening, causes hospitalization or death, or results in lasting disability.

siRNA

Small interfering RNA: short double-stranded RNA that silences a specific gene by triggering degradation of its messenger RNA.

TPP

Target product profile: a planning document describing the desired characteristics of a future product.

WGS

Whole-genome sequencing: determining the order of nearly all DNA in an organism's genome.