Characterisation of cell and gene therapy products

Oligonucleotides, short nucleic acid sequences, play a crucial role in the development of targeted therapies, particularly for genetic diseases and cancers. In biopharmaceuticals, their detailed analysis is essential to guarantee their purity, sequence and integrity. Using advanced chromatography and mass spectrometry technologies, the characterisation of oligonucleotides makes it possible to verify their molecular identity, detect any impurities or degradation, and ensure their therapeutic efficacy.

Antisense oligonucleotides (ASOs) are synthetic molecules designed to modulate gene expression by binding specifically to the target messenger RNA, thereby preventing the production of proteins responsible for certain pathologies. Their development requires sophisticated analytical methods to assess chemical stability, sequence and bioavailability. The analysis of these large molecules in biopharmaceuticals focuses on the detection of post-synthetic modifications and the precise quantification of these molecules in complex biological environments.

Vaccines are complex biological compounds that require meticulous analysis to guarantee their safety and efficacy. Whether they are protein-based vaccines, attenuated viruses or mRNA-based vaccines, their biopharmaceutical development relies on advanced techniques for characterising large molecules, such as mass spectroscopy and chromatography. These analyses are used to verify the structure of antigens, assess the potential immune response, and ensure the absence of contaminants or impurities that could affect the safety of the final product.

mRNA-based vaccines and therapies represent a revolutionary advance in biopharmaceuticals, using RNA sequences to induce the production of therapeutic proteins by the patient's cells. Their analysis requires advanced expertise in the characterisation of large molecules. The tests include verification of the mRNA sequence, the stability of the lipid envelope, and the efficiency of translation into target proteins, ensuring that the treatment is effective while minimising the risk of side effects.

CAR-T (Chimeric Antigen Receptor T-cell) therapies use a patient's own T-cells, genetically modified to target and eliminate cancer cells. As a complex cellular treatment, CAR-Ts require rigorous analyses to ensure the quality, safety and efficacy of the final product. This includes characterising the chimeric antigen receptors, verifying the purity of the cells, and assessing their ability to specifically attack cancer cells. These tests are crucial to ensure that CAR-T therapies meet safety and performance standards before they are administered to patients.