Giuseppe Recchia - Direttore Medico Scientifico
GSK, Telethon e Malattie Rare
Terapia Avanzate
modello di ricerca collaborativa
in Italia per il mondo
For discussion purposes only – All proposals are subject to applicable legal and consultation requirements
Indice degli Argomenti
1.
Ricerca e Malattie Rare
5.
Farmaci Orfani 2015
2.
Terapie Avanzate
6.
Ricerca Collaborativa
3.
Terapia Genica
7.
Scoperta Collaborativa
For discussion purposes only – All proposals are subject to applicable legal and consultation requirements
Malattie dell’Uomo – Ricerca
n. Malattie
senza terapia adeguata
Malattie Tr+
Malattie Tr-
n. Malati
senza terapia
adeguata
Malati Tr+
Malati Tr-
For discussion purposes only – All proposals are subject to applicable legal and consultation requirements

Ricerca
Ricerca & Sviluppo del Farmaco
Sperimentazione Clinica
Ricerca
Target
Scoperta
Lead
For discussion purposes only – All proposals are subject to applicable legal and consultation requirements
Sviluppo
Prodotto
Nuovi Farmaci
Ricerca e Malattie Rare - Criticità

Tante

Gravi

Pochi Pazienti

Genetiche

Eterogenee
Tante conoscenze
Nuove categorie di farmaci
Incentivi
For discussion purposes only – All proposals are subject to applicable legal and consultation requirements
Ricerca e Malattie Rare - Specificità

Ricerca

collaborativa
Terapie
Avanzate
Tante conoscenze
Nuove categorie di farmaci
Incentivi
For discussion purposes only – All proposals are subject to applicable legal and consultation requirements
assenza alternative
Evoluzione Tecnologia del Farmaco
 Advanced
bisogni Therapy
orfani
Biologia
Chimica
Medicinal Products

bisogni
2015residuali
– 2016
1982
XIX se
beneficio incrementale
For discussion purposes only – All proposals are subject to applicable legal and consultation requirements
ATMP - Advance Therapy Medicinal Products
For discussion purposes only – All proposals are subject to applicable legal and consultation requirements
Terapia Genica
1.
Farmaci Orfani 2015
For discussion purposes only – All proposals are subject to applicable legal and consultation requirements
ATMP - Advance Therapy Medicinal Products
1^ Terapia Cellulare – ITA
1^ Terapia Genica ex vivo - NDL
1^ Terapia Genica in vivo – ITA
• approvate EMA
• ITA : scoperte Accademia
For discussion purposes only – All proposals are subject to applicable legal and consultation requirements
Farmaci Orfani - 2015
21 Farmaci Orfani su 45 approvati nel 2015
For discussion purposes only – All proposals are subject to applicable legal and consultation requirements
Farmaci Orfani – Sperimentazione Clinica
Italia 2015
For discussion purposes only – All proposals are subject to applicable legal and consultation requirements
Ricerca Collaborativa
21° Century Act - Promuovere la Ricerca Collaborativa
For discussion purposes only – All proposals are subject to applicable legal and consultation requirements
Scoperta Collaborativa - 2016
Dalla Twittosfera ….
For discussion purposes only – All proposals are subject to applicable legal and consultation requirements
Scoperta Collaborativa - 2016
3. Corporate Venture Funds

Spin Off


Academia
Start Up
1. Pharma R&D

Academia
2. Discovery Partnership Programs

Academia
 legal and consultation requirements
For discussion purposes only – All proposals are subject to applicable
Charities
Scoperta del Farmaco - Accademia
Discovery Partnership with Academia
Competizione tra Ricercatori e tra Università
For discussion purposes only – All proposals are subject to applicable legal and consultation requirements
Un Modello Italiano
DPAc – Discovery Partnership with Academia
For discussion purposes only – All proposals are subject to applicable legal and consultation requirements
Un Modello Italiano
1.
Incontro con il Rettore
2.
Programmazione con Delegato del Rettore
3.
Organizzazione con Responsabile Trasferimento Tecnologico
4.
Informazione a tutti i Ricercatori potenzialmente interessati
5.
Incontro con tutti i Ricercatori potenzialmente interessati
6.
Candidature con 1Page Project Insight Template
7.
Selezione dei progetti
8.
Incontro individuale con i ricercatori dei progetti selezionati
DPAc – Discovery Partnership with Academia
For discussion purposes only – All proposals are subject to applicable legal and consultation requirements
Un Modello Italiano
Presentazioni scientifiche
Business 2 Business
DPAc – Discovery Partnership with Academia
For discussion purposes only – All proposals are subject to applicable legal and consultation requirements
Risultati in Italia – Telethon & GSK
For discussion purposes only – All proposals are subject to applicable legal and consultation requirements
Risultati in Italia – Telethon & GSK
5.5.2015
1.
ADA - SCIS
2.
Leucodistrofia Metacromatica
3.
Sindrome di Wiskott Aldrich
4.
Beta Thalassemia
5.
Mucopolisaccaroidosi tipo I (MPS)
6.
Leucodistriofia globoide (GLD);
7.
Malattia granulomatosa cronica (CGD)
For discussion purposes only – All proposals are subject to applicable legal and consultation requirements
Risultati in Italia
> 10% NMEs - Italia
For discussion purposes only – All proposals are subject to applicable legal and consultation requirements
23.10.2015