Drug discovery and development
The development of a drug is very long and regulated. Key steps include the preclinical phase, during which the therapeutic molecule is submitted to proof-of-concept validation. For this purpose, it is typically tested in screenings, in in vitro tests on cells or in vivo in animals. During the clinical phase, the molecule is tested on humans. If the molecule of interest is found to be safe and effective, it will require a marketing authorization.
In vivo models
In vivo model assays are tests performed on animals. They can be part of the proof of concept validation or performed for the regulatory file. Typically, in vivo tests will study the physiological, behavioral and toxicity aspects of the molecules studied.
In vitro models
In vitro model assays are part of the pre-clinical study phase of drug development. They are useful to profile compounds by studying their effects on defined targets. The robustness of in vitro tests is critical for their use in R&D projects.
HCS & HTS screening
High throughput (HTS) or high content (HCS) targeting are techniques commonly used for studying and identifying molecules with novel and biologically active properties in chemical and target libraries. Screening consists in using a large number of molecules in a biochemical or cellular test, which must be particularly robust, reproducible, and if possible inexpensive.
Different tests on biochemical models are required to validate a drug candidate. Among these are the FRET and HTRF techniques which make it possible to study the phosphorylation of specific proteins, and the signaling pathways altered by molecules.
In silico studies are carried out using computers, analyzing data and modelling biological phenomena, such as an interaction between several molecules or a structural conformation of an active domain. This type of analysis may be particularly relevant upstream of more expensive studies, or when the drug candidate has reached limits that must be overcome.
ATMPs (Advanced Therapy Medicinal Products) are drugs based on genes, tissues or cells, intended for human use. They offer new possibilities for the treatment of diseases and injuries.
Developing a "small molecule" drug candidate requires tools directly taken from chemistry. For instance, the de novo synthesis of molecules, the search for candidates in chemical libraries, and the possibility of modifying these molecules by labelling techniques.
The galenic is a critical step in the development of drugs that requires a high level of expertise to propose the solution best adapted to the nature of the pharmaceutical ingredient candidates, their therapeutic target and the route of administration envisaged.
Pharmacokinetics studies the fate of an active substance of a drug after its administration in an organism. It consists of four phases: absorption, distribution, metabolism and excretion of the active ingredient (ADME). Multiple tests, both in vitro and in vivo, exist to answer these pharmacokinetic questions.