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Neuroscience

We have had long, productive relationships with many organisations who share our passion for impacting the field of neuroscience, where developing therapeutics is a major scientific and clinical challenge. Our focus has been largely on protein misfolding diseases of the central nervous system (CNS), which cause the brain’s nerve cells to break down resulting in the deterioration of physical, mental, and emotional abilities.

As understanding protein therapeutics is a core expertise, we track down drugs that can reduce the accumulation of mutant proteins, a primary pathological event leading to loss of biological function in several CNS disorders including Alzheimer’s Disease, Machado-Joseph Disease (SCA-3) and Parkinson’s.

Finding biomarkers for, and drugs to treat, the neurodegenerative disorder Huntington’s Disease is a specific neuroscience speciality for IRBM, and we have worked with numerous organisations working in this field to push forward the neuroscience frontier, providing insights into disease biology.

In addition to many Pharma and Biotech, our partners in the neuroscience field include:

  • CHDI, with whom we have had a close and collaborative relationship for well over a decade and our combined research has been published in leading scientific journals.
  • Michael J Fox Foundation, where participated in the Investigating Synuclein Consortium

We cover whole range of neuroscience drug discovery phases from target validation through to candidate nomination. In the tables below we list our toolbox of capabilities we can apply to your program.

Assays

Protein levels (mutant HTT and total HTT, SNCA, ATXN3)

Protein T1/2 (HTT)

Oligomerization/aggregation (imaging and biochemical; HTT)

Subcellular localization (imaging and biochemical; HTT)

Viability and functional readouts in primary rodent neurons (imaging and biochemical)

Neurite outgrowth

Models

Primary neuronal cultures from rodents

Human ES/iPS cells, NPCs and derived neurons

Other relevant tools

MDCK (non endothelial cell based model) a high throughput screen general permeation behaviour of compounds, in a cellular setup and their susceptibility to drug efflux.

Human In vitro BBB model derived from iPSCs which can investigate the permeability of small molecules and biologics as well as the evaluation of delivery strategies.

Neuro-focussed compound library that can be screened together with our diverse compound library containing over 320,000 compounds with fully automated compound management

If you are looking for molecular insights into the mode of action of your potential CNS therapeutic, or whether it crosses the BBB, or a novel target for which you want to develop a therapeutic, talk to us. We have the analytical expertise to help you create the CNS medicine of tomorrow.

Publications:

Cristina Cariulo 1, Lucia Azzollini 1, Margherita Verani 1, Paola Martufi 1, Roberto Boggio 2, Anass Chiki 3, Sean M Deguire 3, Marta Cherubini 4 5, Silvia Gines 4 5, J Lawrence Marsh 6, Paola Conforti 7 8, Elena Cattaneo 7 8, Iolanda Santimone 9, Ferdinando Squitieri 9, Hilal A Lashuel 10, Lara Petricca 11, Andrea Caricasole 11

PNAS 2017 DOI:10.1073/pnas.1705372114

Cristina Cariulo 1, Paola Martufi 1, Margherita Verani 1, Lucia Azzollini 1, Giordana Bruni 2, Andreas Weiss 2, Sean M Deguire 3, Hilal A Lashuel 3, Eugenia Scaricamazza 4, Giulia Maria Sancesario 5, Tommaso Schirinzi 4, Nicola Biagio Mercuri 4 5, Giuseppe Sancesario 4, Andrea Caricasole 1, Lara Petricca 1

FRONTIERS IN NEUROSCIENCE 2019 DOI:10.3389/fnins.2019.00889

Cristina Cariulo 1, Margherita Verani 1, Paola Martufi 1, Raffaele Ingenito 2, Marco Finotto 2, Sean M Deguire 3, Daniel J Lavery 4, Leticia Toledo-Sherman 4, Ramee Lee 5, Elizabeth M Doherty 4, Thomas F Vogt 6, Celia Dominguez 4, Hilal A Lashuel 3, Lara Petricca 1, Andrea Caricasole 1

BBRC 2019 DOI:10.1016/j.bbrc.2019.09.097

Margherita Verani 1, Maria Bustamante 2, Paola Martufi 2, Manuel Daldin 2, Cristina Cariulo 2, Lucia Azzollini 1, Valentina Fodale 1, Francesca Puglisi 2, Andreas Weiss 3, Douglas Macdonald 4, Lara Petricca 1, Andrea Caricasole 5

BBRC 2016 DOI:10.1016/j.bbrc.2016.08.057

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