Salute in Horizon 2020:
Giornata Nazionale di Lancio dei bandi 2014-15
Collaborazione tra Università e Industria in un progetto
europeo "Innovation 2" del Settimo Programma Quadro
(FP7): THINPAD
- Maurizio Botta Università degli Studi di Siena
3 Dicembre 2013, Roma
Prof. Maurizio Botta
- 1974. Laurea cum laude in Chemistry at University of Rome
- 1979. PhD Università del New Brunswick, Canada (Prof. Wiesner)
- 1980-1985 Researcher at University of Rome (Prof. Nicoletti)
- 1985-87. Post-doc at Montreal (Prof. Hanessian)
- 1987 Professore Associato, University of Siena
-2000 Professore Ordinario, University of Siena
-- Member Editorial board of ACS J. Med. Chem., ChemMedChem
-- Adjunt Professor Sbarro Institute for Cancer Research and
Molecular Medicine Temple University, Philadelphia, USA
Google scholar
CITATION REPORT
Number of publications: 430
Number of Patents: 15
Number of citations: 5917
h-index: 37
i10-index: 210
University of Siena
Dipartimento di Biotecnologie, Chimica e Farmacia
-Polo Scientifico San Miniato –
Via Aldo Moro 2 53100 - SIENA, Italy
The Dipartimento di Biotecnologie, Chimica e Farmacia of Siena University was
created in 1982. It is situated within the Scientific Area of the San Miniato
University Campus, where the majority of the scientific and educational
institutions are housed and where to it moved in 1999 from its previous location
at the centre of Siena.
Expertise
Combined application of Molecular Modeling , Combinatorial Chemistry and Analytical
Techniques to Speed-up the Lead Discovery Process
Primary libraries
Molecular Modeling
Disease
selection
Gene to
function
Function
to Target
Combinatorial Chemistry
Solid-phase synthesis
Parallel synthesis
Asymmetric synthesis
Biotransformation using enzymes
Microwave-assisted synthesis
Focused libraries
Target to
Hit
Hit to
Lead
milligrams
Lead
optimisation
Pre-dev.
compound
grams
Phase I
Phase II
Phase III
Phase IV
kilos
Overview of the Botta’s Research Group
Design and Synthesis of Hit Compounds
“Hit to Lead” Optimization
Theoretical Study on Target Proteins
ADMET properties
…
Molecular Modeling
Organic Synthesis
Microwave-assisted
Click-chemistry
Natural compounds synthesis
Combinatorial chemistry
…
Virtual Screening
Pharmacophore modeling
Molecular Dynamics
ADMET prediction
…
Analytical Chemistry
& Toxicology
ADMET properties
Membrane permeation
Drug metabolism (liver)
…
Main Research Areas
Infectious disorders
HIV/AIDS
Tuberculosis
-Reverse Transcriptase
-Zmp1
-Integrase
-PtpB
-ENTRY
-pyrazoles
-NCp7
-pyrazolopyrimidin-7-ones
Kinetoplastid diseases
-T. cruzi Tryp.Reductase
-L. infantum Tryp.Reductase
-T. cruzi G6PDH
-T. cruzi CYP51
Fungal/bacterial infections
-macrocyclic amidoguanidines
-linear guanidines
Cancer
Leukemia
Neuroblastoma
Medulloblastoma
……..
EU Projects involvement
Design of MSAAs. Research Training Network (HPRN-CT-200000018) “Design and Synthesis of Novel Paclitaxel (Taxol®) Mimics
Using a Common Pharmacophore Model for Microtubule-Stabilizing
Agents (MSAAs)” 09/2000 – 08/2004
TRIoH Targeting Replication and Integration of HIV- 6th Framework
Programme LSHB CT-2003-503480, from 01/2004 to 06/2007;
ExCellENT-HIT Exploiting Cellular Transport of Nuclear Transcripts as HIV
Innovative Therapy LSHP-CT-2006-037257 11/2006-10/2008;
THINC Targeting hiv integration co-factors, targeting cellular proteins during
nuclear import or integration of hiv
7th Framework Programme-HEALTH-2007-2.3.2-1 from 03/2008 to 02/2012;
CHAARM Combined Highly Active Anti-retroviral Microbicides –
7th Framework Programme 242135 from 01/2010 to 01/2015;
FLUCURE (HEALTH.2010.2.3.3-4 Novel therapeutics against influenza.
FP7-INFLUENZA-2010), Project reference: 259972
from 01/2010 to 01/2014
COST Action Involvment
CM0602 – Inhibitors of Angiogenesis: Design, Synthesis and
Biological Exploration (ANGIOKEM).
CM0801 – New Drugs for Neglected Diseases
CM0804 – Chemical Biology with Natural Products.
CM1106 – Chemical Approaches to Targeting Drug Resistance in
Cancer Stem Cells.
CM1307 – Targeted Chemoterapy Towards Disease caused by
endoparasites.
FP7-HEALTH-2013
Proposal full title: Imaging technologies for detection, diagnosis and therapy of neuroblastoma
Proposal acronym: ImagTechNB
Work programme topics addressed: HEALTH.2013.1.2-1:
Development of imaging technologies for therapeutic interventions in rare diseases
Coordinator: Università degli Studi di Siena (Prof. Maurizio Botta)
Writing collaborator: Dr. Giorgio Maccari
Proposal full title: Targeting Kinetoplastid Diseases
Proposal acronym: TarKinD
Work programme topics addressed: HEALTH.2013.2.3.4-2:
Drug development for neglected parasitic diseases
Coordinator: Università degli Studi di Siena (Prof. Maurizio Botta)
Writing collaborator: Dr. Francesca Moraca
Proposal full title: Targeting the HIV-1 Nucleocapsid Protein to Fight Antiretroviral Drug
Resistance
Proposal acronym: THINPAD
Work programme topics addressed: HEALTH.2013.2.3.1-1:
Drugs and vaccines for infections that have developed or are at risk
of developing significant anti-microbial resistance
Coordinator: Università degli Studi di Siena (Prof. Maurizio Botta)
Writing collaborator: Dr. Mattia Mori
STAGE 1
Proposal full title: Imaging technologies for detection, diagnosis and therapy of neuroblastoma
Proposal acronym: ImagTechNB
Work programme topics addressed: HEALTH.2013.1.2-1:
Development of imaging technologies for therapeutic interventions in rare diseases
Coordinator: Università degli Studi di Siena (Prof. Maurizio Botta)
Writing collaborator: Dr. Giorgio Maccari
Proposal full title: Targeting Kinetoplastid Diseases
Proposal acronym: TarKinD
Work programme topics addressed: HEALTH.2013.2.3.4-2:
Drug development for neglected parasitic diseases
Coordinator: Università degli Studi di Siena (Prof. Maurizio Botta)
Writing collaborator: Dr. Francesca Moraca
Proposal full title: Targeting the HIV-1 Nucleocapsid Protein to Fight Antiretroviral Drug
Resistance
Proposal acronym: THINPAD
Work programme topics addressed: HEALTH.2013.2.3.1-1:
Drugs and vaccines for infections that have developed or are at risk
of developing significant anti-microbial resistance
Coordinator: Università degli Studi di Siena (Prof. Maurizio Botta)
Writing collaborator: Dr. Mattia Mori
STAGE 2
Proposal full title: Imaging technologies for detection, diagnosis and therapy of neuroblastoma
Proposal acronym: ImagTechNB
Work programme topics addressed: HEALTH.2013.1.2-1:
Development of imaging technologies for therapeutic interventions in rare diseases
Coordinator: Università degli Studi di Siena (Prof. Maurizio Botta)
Proposal full title: Targeting Kinetoplastid Diseases
Proposal acronym: TarKinD
Work programme topics addressed: HEALTH.2013.2.3.4-2:
Drug development for neglected parasitic diseases
Coordinator: Università degli Studi di Siena (Prof. Maurizio Botta)
Writing collaborator: Dr. Francesca Moraca
Proposal full title: Targeting the HIV-1 Nucleocapsid Protein to Fight Antiretroviral Drug
Resistance
Proposal acronym: THINPAD
Work programme topics addressed: HEALTH.2013.2.3.1-1:
Drugs and vaccines for infections that have developed or are at risk
of developing significant anti-microbial resistance
Coordinator: Università degli Studi di Siena (Prof. Maurizio Botta)
Writing collaborator: Dr. Mattia Mori
Targeting the HIV-1
Nucleocapsid Protein to fight
Antiretroviral Drug
Resistance
THINPAD website is online
http://www.thinpad.unisi.it/index.html
THINPAD Partners
8th IRNCS – Barcelona 2011
9th IRNPAS – Montreal 2013
SMEs in THINPAD
-IRBM Science Park (http://www.irbm.it/)
-ViroStatics (http://www.virostatics.com/)
• Active collaborations with SMEs, even before THINPAD
 HIV
 Cancer
 Other disease
• SMEs participated to writing and editing the THINPAD proposal
• The THINPAD Business Plan has been elaborated by SMEs and
discussed with other THINPAD parties
THINPAD Budget – SMEs oriented
Academic partner
Academic partner
Academic partner
SME partner
SME partner
INNOVATION-2
SMEs shall request
> 50% of total budget
THINPAD Management & Decision Making Structure
THINPAD WPs Overview
THINPAD Coordination
Research activity
WP2 to WP6
Two coordination workpackages strictly connected with
research workpackages
THINPAD Research Work Plan – Step1: LEAD SELECTION
THINPAD Research Work Plan – Step2: preclinical studies
THINPAD Business Plan - highlights
Step 1
• completion of “first-in-man”
enabling studies
• Phase 1 Clinical study
Managed by THINPAD
SMEs
[1-2 years starting from
the end of the project
(August 2016)]
Step2
• manufacturing,
commercialization
• marketing rights
Out-licensing to
Pharmaceutical
Companies
[starting from the end
of 2017]
in house - SMEs
The Objective is to secure
THINPAD NCIs a codevelopment/license deal by
2018
the economic potential
of the project
Outlicensing to big Pharma