Discovery and R&D activities at MolMed are founded on the company's core technologies to deliver highly effective biological therapeutics, achieved either by targeting peptides or by gene transfer. MolMed focuses its efforts particularly on the development of novel cell and gene therapies, and vascular targeting agents, thanks to outstanding know-how and expertise in related technologies.

Individual-specific genetic engineering of human cells for the introduction of genes with therapeutic functions

MolMed scientists have pioneered the field of genetic modification of haematopoietic stem cells. The experience acquired during almost ten years of developing vectors, gene expression tools, ex vivo gene transfer, and patient conditioning regimens puts the company in a unique position; and demonstrates its capability to apply the cell and gene therapy approach to cancer and to extend it to other severe diseases, such as rare genetic diseases. An important aspect of MolMed's discovery and R&D activities is the development of new gene transfer vectors and packaging cell lines, to increase the safety and efficacy of gene and cell therapy.

Design and development of vascular targeting agents (VTAs)

Peptides are small molecules that are able to interact in a highly selective way with cell surface receptors. Because of this high selectivity and strength of the interaction, it is possible to identify peptides that home to cell surface receptors expressed by tumour tissues. Such peptides can be used to deliver biologically active cytokines specifically to the tumour site, thus enhancing the effectiveness and reducing the toxicity caused by non-specific effects on normal tissues. Furthermore, the cytokine-peptide combination gives origin to a novel molecule with both the cytokine activity and the modulating effects of the peptide; in this manner, synergism is achieved, and the resulting molecule acquires novel properties of its own as well. MolMed's vascular targeting agents - NGR-hTNF, NGR-IFNg and NGR-IL12 - are recombinant therapeutic proteins based on this concept.