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

Il genoma umano è costituito da 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 pb genoma umano

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)