With increasing knowledge of genomics and proteomics, many
new intracellular pharmacological targets will be achieved.
Exploiting these new therapeutic opportunities demands the
sustained delivery of active compounds to the interior of
the cell. A generic platform will be developed that enables
prolonged exposure of intracellular targets to proteins and
small molecules. DIATOS' transport peptides will be
combined with OCTOPLUS' Dextran-based particulate delivery
The project is divided in two phases, a feasibility and a
1. Feasibility Phase:
Four approaches will be followed in a stepwise manner:
1a. Controlled release of therapeutic proteins, linked to
transport peptides, from Dextran microspheres.
1b. Intracellular controlled release of a therapeutic
protein from 500 nm microspheres after loco-regional
1c. Intracellular controlled release of a therapeutic
protein from nanospheres (100-200 nm) or transport-
facilitated liposomes after intravenous administration.
1d. Intracellular controlled release of a gene, coding for
a therapeutic protein, from 150 nm nanospheres.
2. Development Phase:
From the feasibility study, a choice will be made on the
most promising technology. Preformulation and formulation
development studies will be carried out to obtain a
laboratory bench prototype. Freeze drying development and
upscaling will be necessary to prepare for clinical phase 1
and phase 2 production. One of the technological challenges
is the sterilization of these protein-containing
particulate systems. Safety and efficacy of the drug
delivery system will be investigated in a toxicology
programme and clinical phase 1 and phase 2 studies.
Keywords: drug delivery, therapeutic, biotechnology.