DESCRIPTION OF THE RESEARCH RESULTS

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1 REF.: QUI_UAH_02 INDUSTRIAL SECTOR RESEARCHER DEPARTMENT CONTACT DETAILS WEB SITE Biochemistry, Pharmacy Fco. Javier de la Mata de la Mata Inorganic Chemistry javier.delamata@uah.es Dendrimers in biomedical applications DESCRIPTION OF THE RESEARCH RESULTS TITLE Synthesis of dendritic systems of nanoscopic size for applications in biomedicine ABSTRACT BIOINDEN research group, from the Inorganic Chemistry Department of Alcalá University has develop a technology consisting in the synthesis and structural characterization of dendritic systems of nanoscopic size with applications in different fields of biomedicine. Among all of them the group launches the development of these systems for the transport and release of drugs as a vehicle of transfection in gene therapy or as therapeutic agents (antibacterial, antiviral or antiprionics). The group is looking for companies from the chemistry, pharmaceutical or biotechnology sector for collaboration in R&D agreements, joint venture agreements and licensing agreements.

2 DESCRIPTION AND SPECIAL FEATURES The developed technology lies in the preparation of molecules of nanoscopic size based on dendritic systems or hyperbranched polymers that contains a carbosilane skeleton and are functionalized in the periphery with groups from different nature (cationic, anionic or neutral), with the objective to provide them with activity focused in different biomedical aims. Among these applications, we can emphasize the development of cationic dendrimers appropriate for the transport of anionic drugs or as non viral vehicles in transfection processes in gene therapy, because of its capacity to join biomolecules with negative charge as oligonucleotides or sirna. These cationic systems can also act as therapeutic antibacterial agents (against gram + bacterial and gram bacterial) or as anti-prionic agents. The group has proved that cationic dendritic systems of carbosilane type are able to join by an electrostatic interaction oligonucleotides or small RNA of interference (si RNA), making complexes that have proved not to be toxic in concentration among 1-5 ım. These complexes are able to internalise the genetic material inside different types of cellular lines and in some cases they have showed a great efficiency in the inhibition of HIV. They have also showed to be useful in processes of transfection of plasmids to different cellular carcinogenic lines in vitro and in vivo, being observed in some cases percentages of transfection bigger to those that are obtained by the use of commercial transfectants. Regarding the antibacterial capacity of these cationic systems, the group has proved that they present a great activity against gram + bacteria and gram bacteria, and in addition there are signs of strengthening of the antibacterial activity of penicillin when it joins one of the dendritic systems. The technology developed allows also the preparation of anionic systems that present an important antiviral activity, mainly against HIV. That s why, these system are proposed as good candidates for the development of a powerful microbicide for external use that avoids the infection for HIV by sexual transmission. Besides this microbicide capacity, these systems have showed antiinflammatory properties what reinforces his inhibitory capacity. The functionalization with neutral groups, has been carried out incorporating oligosacharides into the periphery of the dendritic systems developed what awards to these systems antimicrobial properties. All the synthesized macromolecules have been characterized using different techniques of structural elucidation as elementary analysis, spectroscopy of RMN, IR or UV, mass spectrometry or GPC. INNOVATIVE ASPECTS Regarding the cationic systems developed, the principal innovative aspects are the following: - They are non viral nanoscopic synthetic molecules able to internalise nuclear material inside different cellular lines. This turns them into good candidates to be considered as non viral vectors in gene therapy processes.

3 - They are non toxic in a concentration range adapted for the accomplishment of different biomedical studies. - They present antibacterial properties against gram + and gram- bacteria. - They are able of interacting, in a electrostatic way, with anionic drugs which turns them into drug delivery systems. - They present a hydrophobic carbosilane skeleton that allows the molecules to cross the cellular membranes with major facility. - They are soluble in water. TECHNOLOGY KEYWORDS Dendrimers, dendrons, dendritic systems, nanoscopic, nanomedicine, biomedicine, antiviral, antibacterial, antiprionics, microbicides, gene therapy, nanoscience, nanotechnology. SCIENTIFIC DOMAINS Information and Communication Technologies Industrial Manufacture, Material and Transport technologies Other Industrial Technologies Energy Physical and Exact Sciences Biological Sciences Agriculture and Marine Resources Agrofood Industry Measurements and standards Environment and risk prevention Socioeconomics CURRENT STAGE OF DEVELOPMENT OF THE RESEARCH RESULT Development phase Developed, available for demonstration Already on the market

4 FUNDING RESEARCH European RTD project National project Regional project Private funding COMMERCIAL ASPECTS COMPETITIVE ADVANTAGES Regarding the cationic systems developed, the principal competitive advantages are the following: - Cheaper preparation than other commercial transfection agents. - Minor toxicity than other commercial transfection agents. - Levels of transfection similar or superior to commercial alternatives (depending on the cellular line used) - Antibacterial capacity against to a wide spectrum of bacteria Regarding the anionic systems developed, the principal competitive advantages are the following: - They present a great antiviral capacity, mainly against HIV virus. - They have antiinflammatory activity. - They can be in use in the development of a microbicide gel of topic use. CURRENT STATE OF INDUSTRIAL AND INTELLECTUAL PROPERTY Patent applied Patent granted Software registered Exclusive rights Secret know how Copyright protected COMMENTS Patent granted with reference P

5 TYPE OF COLLABORATION SOUGHT Technical cooperation Joint venture agreement Commercial agreement with technical assistance License agreement Manufacturing agreement COMMENTS The research group is looking for joint venture agreements or licensing agreements with companies from the chemistry, pharma or biotechnology sector. ADDITIONAL INFORMATION (PICTURES) OTRI CONTACT DETAILS UNIVERSIDAD DE ALCALÁ-OFICINA DE TRANSFERENCIA DE RESULTADOS DE INVESTIGACIÓN (OTRI) Edificio Politécnico Campus Externo UAH Alcalá de Henares (Madrid) / otriuah@uah.es

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