Adriana Maggi DOCENTE DI BIOTECNOLOGIE FARMACOLOGICHE CORSO DI LAUREA SPECIALISTICA IN BIOTECNOLOGIE DEL FARMACO Lezione 2 A NOVEL INITIATIVE TO PUT EUROPE AT THE FOREFRONT OF BIOPHARMACEUTICALS INNOVATION IMI STRATEGIC AGENDA 1. reducing medical needs 2. Supporting breakthrough of novel therapeutic avenues 3. Harmonizing reductionistic and systemic approaches to drug research 4. Optimizing R&D process 5. Implementing new techniques and technologies 1. reducing medical needs • Reclassification of the etiology of important diseases and relative drugs on the basis of the molecular target • Coordinate R&D on rare and neglected diseases • Meeting therapeutic needs in gender and age related diseases 2. Supporting breakthrough of novel therapeutic avenues • Nuclear medicine and imaging-based approaches • Extracorporeal therapies (gene surgery, tumor vaccine, stem cell therapy, tissue engineering) • Health technologies • Immunotherapies • Nucleic acid therapies • Productiontechnologies of biologicals 3. Harmonizing reductionistic and systemic approaches to drug research • Receptor-based drug research • Signaling pathways-based drug research (dual action, multi target approach: define complexity of signalling pathways, cell specificity, intr-pathway connectivity…) • Metabolic network-based drug research ( artificial intelligence for data handling and robotics for data generation: nomenclature, data collection, defining systems of pharmacological interest, generate formats of comparable information with old drugs, set up pilot studies) 4. Optimizing R&D process • Reshaping clinical studies • Regulatory measures for novel therapeutics • Personalized medicine • Systematic integration of patients into drug development • Correlate preclinical and clinical studies 5. Implementing new techniques and technologies • Novel approaches in target research • API technology • Advanced formulations • Imaging • Increasing practicability of biobancks • Pharmacoinformatics to correlate in vivo, in vitro and in silico research BIOTECNOLOGIE E FARMACOLOGIA • Identificare nuovi bersagli farmacologici • Screening di farmaci • Nuovi molecole terapeutiche Identificare nuovi bersagli farmacologici • GENOMATICA • GENOMATICA FUNZIONALE • MODELLI DI MALATTIA La GENOMATICA e la ricerca di nuovi farmaci GENOMATICA: studio del genoma* degli esseri viventi attraverso la mappatura o sequenziamento dei geni e lo studio della loro funzione * Per genoma si intende l’intero contenuto in DNA di una cellula, geni e sequenze intrageniche incluse genoma umano Il genoma umano è costituito da 3.000.000.000 di paia di basi Diviso in 24 sequenze lineari : i cromosomi di cui 22 sono autosomi e 2 sono i cromosomi sessuali Il DNA mitocondriale è circolare e ha le dimensioni di 16569 pb Mappare i geni: • Storicamente attraverso la costruzione di mappe fisiche (identificare e ordinare marcatori lungo il cromosoma) • Attualmente con il sequenziamento mappe fisiche costruite con: • restriction mapping •FISH (Fluorescent in situ hybridization) mapping • studi genetici di ‘linkage’ • use of sequence tagget site (STS)