Science and Technology

  • The “art” of gene therapy consists in transferring a gene, or more generally a nucleic acid into a living organism to perform a therapeutic function, whereas the “vectorization” of this element is central in order to protect it from alterations due to enzymatic activities during its transfer to its cellular target. The most efficient vectorization to date for the treatment of genetic diseases relies on the use of viruses. These viruses are rendered non-pathogenic, by the deletion of viral genes implicated in viral replication, which at the same time creates space for the cloning of the therapeutic gene (→ viral vectors).
  • The reason it has taken several decades to convince people that gene therapy should join the therapeutic arsenal is that one of the persistent limiting factors is the difficulty in producing clinical grade batches of viral vectors in a reasonable amount of time. Methods for vector production, purification and characterization are still rather “primitive”, but fortunately they are still compatible with the running of Phase I/II clinical trials.
  • In order to anticipate a wider application of gene therapy it is necessary to develop new and faster production methods, to improve the quality of the gene therapy product, and to industrialize the manufacturing procedures. These objectives are real challenges, because vectorology which is the basis for the technology for production of gene therapy medicines involves numerous parameters which cannot be easily controlled at the moment.
  • The PGT consortium is an assembly of experts in virology, bioprocess, production and purification, analysis and quality control necessary for the development and exploitation of production platforms for recombinant adenovirus, adeno-associated virus and lentivirus vectors for the treatment of rare disorders. At the beginning the main efforts will be concentrated on the improvement of procedures and later on the therapeutic vectors will be made available to the international scientific community.
  • The production and quality control platforms of the PGT consortium will be open to both academic and industrial users.