Lezione 2 2010-11

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)