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Biomolecules Webinar | An Update on CFTR Drug Discovery: Opportunities and Challenges

14 Oct 2021, 14:00 (CEST)

CFTR, Stability, Trafficking Defect, Modulators, Drug Discovery, Computational Approaches
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Welcome from the Chairs

Biomolecules Webinar Series

An Update on CFTR Drug Discovery: Opportunities and Challenges

Dear Colleagues,
we welcome you to this webinar entitled “An Update on CFTR Drug Discovery: Opportunities and Challenges”.

Cystic fibrosis, the most common monogenic disease in Caucasians, despite the efforts of the scientific community, is still waiting for the development of additional therapeutics. Near symptomatic drugs (aggressive antibiotics, osmotic agents, pancreatic enzyme products citing some), modulators (correctors, potentiators, amplifiers) represent the greatest advance in the treatment of CF in the last decade and are the solid pillars for current therapies, which significantly enhanced the mean survival age of patients. Nevertheless, the burden of CF care and the need of new CF drugs remain very high, the available cures in fact possess severe side-effects to cope with, may determine serious complications and do not target all CFTR mutations.

This webinar, thanks to the contribution of two outstanding scientists in the field, Dr. Isabelle Callebaut (IMPMC, Sorbonne Université, Paris, France) and Dr. Nicoletta Pedemonte (Gaslini Institute, Genova; Italy) will offer you an overview about the most recent experimental and computational findings in the field of CFTR, thus providing a useful tool for pushing forward research on this target and on CF disease.

Prof. Paola Fossa
Dott. Pasqualina D’Ursi
Chairs

Date: 14 October 2021

Time: 2:00pm CEST | 8:00am EDT | 8:00pm CST Asia

Webinar ID: 823 0822 8688

Webinar Secretariat: biomolecules.webinar@mdpi.com

Chair

Department of Pharmacy, Universita degli Studi di Genova, Genoa, Italy

DETAILS
Bio
Paola Fossa got her PhD in Pharmaceutical Sciences in 1992 at University of Genoa, Italy. Senior Researcher since 1997, she has spent some training periods at Department of Chemistry, Leiden University, Dipartimento Farmaco-Chimico, University of Bari and Institut fur Pharmazeutische Chemie, Dusseldorf University, acquiring skills and experience in computational chemistry and drug design. She is Associate Professor since 2005 at the Department of Pharmacy, University of Genoa. Her main research activity concerns the application of computational strategies to medicinal chemistry issues, mainly rational design and identification of CFTR modulators, ligands for GPCRs receptors (serotoninergic, cannabinoid and trace amine receptors), PDE4 inhibitors. She has published about 110 papers and is referee for several medicinal chemistry journals. She has been responsible of different research projects funded by University of Genova and Italian Cystic Fibrosis Foundation.

Institute of Biomedical Technologies-CNR, Segrate, Italy

DETAILS
Bio
Pasqualina D'Ursi is a researcher at the Institute for Biomedical Technologies (ITB) of the Italian National Research Council (CNR). She received a BS degree in Biological Science and a Phd in Molecular Medicine at the University of Milan. Her research activity is focused on understanding the relationship between the molecular structural of a protein and its function. This information is necessary to provide predictions on the effects of disease-causing mutations and positively help drug-design. Computational methods such as molecular modeling, docking, molecular dynamics and bioinformatics have been applied in her research to analyze protein-ligand interactions for drug discovery (Cystic Fibrosis, Covid19, HIV and Endocrine Disruptors) and the study of the structure-function relationship of mutated proteins involved in the coagulation cascade and rare diseases.

Invited Speakers

IRCCS Istituto Giannina Gaslini – UOC Genetica Medica, Italy

DETAILS
Bio
Nicoletta Pedemonte obtained her PhD in Human Genetics at the University of Turin, Italy in 2007 and a postgraduate specialization in Clinical Pathology at the University of Genoa, Italy in 2011. She is Senior Scientist at the UOC Genetica Medica of the IRCCS Istituto Giannina Gaslini in Genoa, Italy, where she leads a group of seven researchers. She has a strong expertise in drug discovery strategies and functional genomics. Her research activity is focused on transepithelial ion transport mechanisms in airway epithelial cells, mechanisms associated to mutant protein maturation and degradation, and identification of novel small molecules to rescue mutant CFTR protein in cystic fibrosis. She has authored 79 international peer-reviewed publications with 3,469 citations, H-factor 24 (Scopus). Scopus Author Identifier: 8095389700

IMPMC, Sorbonne Université – CNRS UMR790, Muséum National d’Histoire Naturelle

DETAILS
Bio
Isabelle Callebaut is CNRS Research Director at Sorbonne University (Paris). She is Engineer in chemistry and received her Ph.D. from the Faculty of Agronomical Sciences in Gembloux (Belgium), in 1993. Her PhD thesis was devoted to the development of vaccine strategies against retroviruses. During her post-doctoral training at the University Pierre et Marie Curie, she specialized in the analysis and prediction of protein structures. Her current research is centered on structural bioinformatics, developing and applying original methods for the analysis of sequence-structure-function relationships and for the characterization of the dark proteome. A lot of applications focus on medically important proteins, among which CFTR. Current research interests in the field of cystic fibrosis include the understanding of the function, dysfunctions and modulation of the CFTR protein, in particular by using molecular dynamics simulations. She is the author/co-author of more than 200 peer-reviewed articles (H-factor 58, > 10000 citations (Web of Science)).

Webinar Content

The 5th Biomolecules webinar, entitled “An Update on CFTR Drug Discovery: Opportunities and Challenges”, was held on Thursday 14 October 2021. Chaired by Prof. Dr. Paola Fossa (Universita degli Studi di Genova, Italy) and Dr. Pasqualina D'Ursi (Institute of Biomedical Technologies-CNR, Italy), this online seminar included the scientific contribution of Dr. Isabelle Callebaut (IMPMC, Sorbonne Université, Paris, France) and Dr. Nicoletta Pedemonte (Gaslini Institute, Genova; Italy) which offered an overview about the most recent experimental and computational findings in the field of CFTR research.

The webinar highlighted and discussed the importance to develop mutation-specific modulators in order to have drugs able to properly fit for any of the several CF mutation types. These compounds should possess optimal and well-defined Adme-Tox profiles. Novel cell models and assays are being developed to faster evaluate large libraries of compounds and to best characterize the most promising ones for therapy. A detailed understanding of the structural complexity of the native and the mutated CFTR protein, by means of experimental data combined with computational approaches, is mandatory for designing new modulators. The identification and modulation of new druggable targets, as an alternative to the CFTR protein, is a promising task.


The presentations were followed by a Q&A and a discussion, moderated by the Chairs of the event. The webinar was offered via Zoom and required registration to attend. The full recording can be found here on Sciforum website. In order to stay updated on the next webinars on Biomolecules be sure to sign up for our newsletter by clicking on “Subscribe” at the top of the page.

Program

Speaker/Presentation

Time in CEST/CET

Prof. Dr. Paola Fossa

Chair 1 Introduction

2:00 - 2:15 pm

Dr. Nicoletta Pedemonte

2:15 - 3:00 pm

Q&A Session

3:00 - 3:15 pm

Dr. Pasqualina D'Ursi

Chair 2 Introduction

3:15 - 3:30 pm

Dr. Isabelle Callebaut

3:30 - 4:15 pm

Q&A Session

4:15 - 4:30 pm

Closing of Webinar

Chair

4:30 - 4:35 pm

Relevant SI

An Update on CFTR Drug Discovery: Opportunities and Challenges
Guest Editor: Prof. Dr. Paola Fossa
Deadline for manuscript submissions: 31 July 2021

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