Drug development for congenital malformations
The drug development process for a congenital condition
Developing a drug to treat a birth defect is an extremely long and regulated process. It globally includes a drug discovery phase, during which drug candidates are identified, and a preclinical phase where drug candidates have to demonstrate their proof-of-concept.
It is often difficult to determine the exact cause of a congenital malformation, whether it has a genetic, environmental or infectious origin. Treatments for such conditions often consist in a pediatric surgery and/or drugs that aim to manage symptoms or functional problems caused by thalassemia, sickle cell anemia or congenital hypothyroidism for example.
To develop such medicines, screening methods in in vitro tests or in vivo studies are commonly used. In the clinical phase, drug candidates that have passed the previous phases are tested on humans. If the drug candidate is judged to be safe and effective, it will have to get market approval.
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In vivo models
In vivo model tests are performed on animals. They may be part of the proof-of-concept validation or may be performed for the regulatory dossier. Typically, in vivo tests will study the physiological and behavioral aspects, as well as the toxicity of the molecules being studied.
In vitro models
Tests on in vitro models are generally part of the pre-clinical study phase of drug development. They are used to test compounds by studying their effects on defined targets and functions. The robustness of in vitro tests is a determining factor for their use in R&D projects.
HCS & HTS screening
High throughput screening (HTS) or high content screening (HCS) are techniques that aims at studying and identifying, within chemical and target libraries, molecules with novel and biologically active properties. Screening consists of using a large number of molecules in a biochemical or cellular test, which must be particularly robust, reproducible, and if possible inexpensive.
The biochemical model is used in the discovery of candidates. The advantage of this type of model is to limit the number of molecular actors, and thus validate a target or mechanism of action. FRET and HTRF techniques applied to biochemical models are particularly effective in studying the phosphorylation and signalling pathways of molecules.
In silico studies
In silico studies correspond to the modelling of biological phenomena, such as the interaction between several molecules or a change in the structural conformation of an active domain. This analysis is relevant upstream of more expensive studies, or when classical chemical methods have reached their limits.
Tools for innovative therapies
ATMPs (Advanced Therapy Medicinal Products) are medicines based on genes, tissues or cells for human use. They offer revolutionary new possibilities for the treatment of diseases and injuries.
Molecules synthesis and optimization
The development of a "small molecule" drug candidate requires tools directly derived from chemistry. In this case, the de novo synthesis of the molecule, the search for candidates in chemical libraries, and the possibility of modifying these molecules by labelling techniques.
Formulation and galenic
Formulation is a critical step in drug development and partly determines the success of a drug's market entry. The aim is to propose the solution best suited to the nature of the pharmaceutical ingredient candidates, their therapeutic target and the route of administration envisaged.
Regulatory studies and assays
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). Different tests, in vitro and in vivo, exist to answer these pharmacokinetic questions.