Adriana Maggi

DOCENTE DI BIOTECNOLOGIE FARMACOLOGICHECORSO 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 needs2.Supporting breakthrough of novel therapeutic avenues3.Harmonizing reductionistic and systemic approaches to drug research4.Optimizing R&D process5.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 3.000.000.000 di paia di basiDiviso 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

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)