Drug development for rare and orphan diseases

The drug development process for rare diseases

Orphan diseases do not benefit from viable treatments for their patients. Primarily linked to genetics, rare diseases are often called orphan diseases as a result of the economic difficulties to create new treatments that very few patients will benefit from. Therapeutic care of rare diseases often consists in managing symptoms and limiting the disease’s progression in order to improve the patient’s quality of life. However, progress in gene therapy now allows patients to benefit from biopharmaceuticals.

The production of biodrugs is complex, since it involves manipulation of weak living agents (cells, proteins, DNA…). It globally includes a first phase of cell culture derived from a unique cell line, then a phase of isolation and purification of the wanted therapeutic agents, and finally a phase where the pharmaceutical form is produced.

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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.

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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.

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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.

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Biochemical models

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.

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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.

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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.

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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.

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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.

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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.

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