WG Nanotherapeutics & Targeted delivery

Nanotherapeutics are defined by their specific physico-chemical properties due to their quantum nanoscale, opening new opportunities in the diagnosis, prevention, and treatment of diseases. nanotherapeutics and imaging agents are able to make a difference for patients:

• As drug delivery system to deliver poorly bioavailable and / or highly toxic therapeutic agents
• As therapeutic agents per se: some innovative treatments are now based on the physico-chemical properties of these nanotherapeutics which can be externally activated (by radiation or hyperthermia for example especially in oncology).
• As theranostic agents: imaging therapeutic agent accumulation at the disease site and treatment with the same nanomaterial by coencapsulation of two molecules or using the physico-chemical properties of the nanomaterial (e. g. metallic nanoparticles are suitable for imaging due to their radio opacity and are able to locally enhance energy dose deposition during radiotherapy).
• As pre-conditioning therapy, nanomaterials can be designed to prime the body to receive the treatment: Nanomaterials could support therapeutic agent to overcome biological barriers and increase the benefit /risk ratio of the corresponding treatment. As example nanomaterials could occupy transiently the main pathways involved in the clearance of a therapeutic agent or increase accumulation in the target tissue (e. g. increasing enhance permeability and retention effect (EPR) in solid tumors). Such approaches decrease interpatient variability and enlarge the patient population that could be treated with a same therapeutic agent.
• Nanomedicine can contribute to enhance and optimize other therapies: like tissues engineering (nanoformulated additives, nanostructured biomaterials and scaffolds) and gene therapies (non-viral vectors).
• Diagnosis and patient stratification: Inert nanomaterials can be used as a diagnostic tools and companion diagnostic nanomaterials or generic one is being implemented to identify patients that would benefit from a nanotherapeutic treatment (e; g. visualization of tumor accumulation of the nanotherapeutic).

Benefits and challenges of this working group

The Nanotherapeutics and Imaging WG aims to support the development of these innovative products to the clinic to address unmet medical needs. Our goal is to:

• Support regulatory awareness for successful development.
• Identify and inform about scale-up solutions and parameters of quality according to type of nanoscale-product.
• Support communications across interested parties including manufacturing industry, pharma, patients, clinicians, payers, public domain, and international legal bodies.

Past realizations & accomplishments

During the last years the WG has increased the participation of the different WG members.

We have elaborated a map of capabilities at the EU level, including research and, in a second action, GMP/ISO scale up capabilities, contributing to the generation of common research efforts in the WG. We have also contributed to maintain and define the EU investments in research for nanotherapeutics (i.e. Strategic Research Agenda) and to the better understanding of the regulatory needs for nanotherapeutics.

Key actions 2019-2021:

• In coordination with other WGs, identify potential and applicability of nanomedicine with a patient driven approach: white paper redaction to enlighten the potential of nanotherapeutics and imaging for specific diseases, point out the different challenges to anticipate for a successful product development.
• Continue to contribute to support and define the EU investments in research for nanotherapeutics and imaging (through Horizon Europe): ensure that the industry vision and needs are taken in consideration at the EU level.
• Communication and collaboration within the nanomedicine community: communication from industry on successful stories, bottleneck met during development / creation on networking tools (on ETPN website, during events…).
• Identification and support development of the most promising nanotherapeutics in collaboration with the translation hub TAB.
• Contribute to the creation of guidelines for regulatory development of nanomedicines in collaboration with ETPN WGs and EuNCL.