GENETHON

Development of therapeutic approaches involving the genetic modification of cells (e.g., T cells, stem cells, macrophages) or the use of viral/non-viral vectors to deliver genetic material for disease treatment.

Therapeutic strategies employing nucleic acids (DNA, RNA, oligonucleotides) to modulate gene expression, deliver genetic material, or interfere with disease-causing pathways. Includes gene therapy using viral vectors.

Development and application of therapeutic proteins or peptides produced through recombinant DNA technology, including fusion proteins and modified growth factors.

Methods and apparatus specifically tailored for the expansion, differentiation, or genetic modification of cells for therapeutic applications (e.g., CAR T cells, progenitor cells) or for the production of specific biological products (e.g., cultured fat, RNA).

Modification of protein or peptide sequences, structures, or post-translational modifications (e.g., glycosylation, lipidation) to enhance their stability, solubility, delivery, or therapeutic efficacy.

Design and synthesis of acyclic or carbocyclic organic compounds that selectively modulate specific biological targets or pathways for the treatment of diseases.

Therapeutic approaches involving the use of living cells, often genetically modified or ex vivo activated, to treat diseases, particularly cancer, by modulating immune responses or replacing damaged cells.