Theme 2.2: Autologous Immunotherapies

Principle of autologous therapy

Objectives

Our objective is to develop a functional DNA vector that addresses the current challenges associated with biologics, aiming to improve patient comfort while maintaining effective disease control. We hypothesize that intramuscular injection of minicircle DNA encoding etanercept could enable autologous production of the drug over several months, thereby significantly reducing the frequency of injections and long-term treatment adherence.

Summary

The context : Biopharmaceuticals are widely used in clinical practice, in particular to treat chronic inflammatory diseases. Despite their efficacy and the accumulated experience since the late ‘90s, biopharmaceuticals have several limits.

Our approach consists on delivering nucleic acid molecules onto the patients’ own cells, i.e. muscular cells, which will, in return, act as « drug-manufacturing-cells ». This innovative approach represents a shift from the current external production of biopharmaceuticals, towards an internal « on-site » manufacturing » paradigm. This shift from external to internal delivery represents an opportunity for European pharmaceutical industry.

The potential benefits of the technology in this project are thus:

  1. A self, proper manufacturing of the drug by the patient himself could lead to a sustained and prolonged effect for several month/years, less constraints and a full adherence/observance and remission maintenance for the patient. « By the patient, for the patient ».
  2. The financial burden could largely be reduced, owing the substantial decrease in reimbursement by the public Health, for the drug itself and for secondary costs in relation with hospitalisation expenses (e.g. nurses, syringes).
  3. Europe could be at the front line in this new paradigm and European pharmaceutical industry could be pioneer in the development of nucleic acid solutions to treat large population of patients.
  4. By avoiding the complex and energy consuming system of production, the technology provided could participate to the environmental protection.

Keywords