Written by Gianluca Quaglio and Nada Alkhayat, Therapies for the futureThe purpose of this workshop is to report on the status of nanomaterial-based and advanced therapy medicinal products, as well as on possible pathways for the future development of these new therapies in Europe. The event will offer participants an opportunity to develop a range of options for supporting patient access and transparent information on these therapeutic treatments across Europe. Nanomedicine (the application of nanotechnology for the treatment, diagnosis, monitoring and control of biological systems) and advanced therapy medicinal products have great innovative potential. The implications for a number of diseases – for which no adequate diagnostic and therapeutic tools exist today – are significant. Both areas may also represent a vital boost for the future development of the pharmaceutical industry in Europe.

Join the discussion by registering for the workshop by 9 October 2017. Web-streaming is available.

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Research into the rational delivery and targeting of pharmaceutical, therapeutic and diagnostic agents is at the forefront of projects in nanomedicine. These involve the identification of precise targets (cells and receptors) related to specific clinical conditions and the choice of appropriate nano-carriers to achieve the required responses, while minimising side effects. Specific cells (e.g. dendritic, endothelial or tumour cells) are key targets. Today, nanotechnology and nano-science approaches to particle design and formulation are beginning to expand the market for many drugs. Advanced therapy medicinal products constitute an innovative group of heterogeneous, research-driven biopharmaceuticals. This group encompasses gene therapy and somatic cell therapy medicinal products, as well as tissue-engineered and combined products (tissue or cells associated with a device):

  • Gene therapy medicinal products are products of biological origin containing recombinant nucleic acids and having a therapeutic, prophylactic or diagnostic effect related directly to the recombinant nucleic acid sequence.
  • Somatic cell therapy medicinal products are biological products that contain or consist of cells or tissues that have been subject to substantial manipulation (or that are not intended to be used for the same essential functions) in the recipient and the donor; the recipient and the donor could be the same person.
  • Tissue-engineered products contain or consist of engineered cells or tissues and are presented as having properties for, or are used in, or administered to, human beings with a view to regenerating, repairing, or replacing human tissue.

Potential candidates for advanced therapy medicinal products include severe, untreatable or chronic diseases, and many clinical trials are currently underway in conditions varying from cancer, cardiovascular diseases, musculoskeletal conditions and immune system disorders. If advanced therapy medicinal products are to fulfil their promise of providing innovative treatments for patients, then the regulatory environment should encourage innovation, safeguarding public health and, ultimately, facilitating timely patient access to new therapies.