Shared expertise

Cross-functional project management

This expertise, made available to projects led by the laboratories of the Institute of Biotherapies and/or leading teams supported by AFM-Telethon, ensures the effective implementation and coordination of projects for the discovery and development of new medicines, with the aim of initiating a clinical trial.

This support begins at the scientific proof-of-concept stage and continues through to the regulatory non-clinical toxicity studies of the drug candidate. It encompasses: selection and optimisation of the drug candidate; evaluation of its activity in experimental disease models (in vitro and in vivo); validation of its mechanism of action; assessment of its biodistribution; and toxicology studies aimed at identifying target organs and toxic doses.

All these studies must make it possible to determine the dose that can be tested in humans.

Cross-functional project management includes:

  • developing non-clinical development plans in compliance with applicable regulations;
  • coordinating the various stages of preclinical development within the required timeframes;
  • identifying service providers and acting as an interface;
  • establishing budgets and allocating resources.

Artificial intelligence is now used across the Institute of Biotherapies: capsid design for gene therapy, analysis of omics data, and high-throughput drug screening to identify molecules of therapeutic interest.

 

Clinical development and regulatory affairs

The clinical development of drug candidates developed by the laboratories of the Institute of Biotherapies and/or leading teams supported by AFM-Telethon requires specialised skills and expertise to prepare, design and conduct clinical trials in collaboration with hospital investigators, in accordance with both French and international regulatory frameworks.

Drug candidates are also submitted for “Orphan Drug” designation to the European Medicines Agency (EMA) and/or the Food and Drug Administration (FDA).

The clinical development team, based at Genethon, works across all entities of the Institute of Biotherapies.

Partnerships and technology transfer

Involved in international projects with private partners (pharmaceutical and biotech companies), institutional partners (Inserm, CNRS, AP-HP, etc.) and various funding bodies (NIH, European Community, Oseo, etc.), the Institute of Biotherapies develops a partnership and technology transfer strategy enabling it to seize opportunities for co-development or joint exploitation.

It has the expertise required to implement:

  • a strategy for in-licensing technologies, know-how and patents required for the development of Institute of Biotherapies projects;
  • a strategy for out-licensing projects and technologies developed within the Institute of Biotherapies;
  • scientific legal expertise;
  • expertise in cross-functional project management;
  • a strategy for developing international collaborations.

This strategy contributes to the international visibility of the Institute of Biotherapies and its entities. It also helps secure the development of its resources in order to advance its programmes and launch new ones.