Scientific Contributions

UniQure has just published a study in Science Translational Medicine using a Huntingtin (HTT)-lowering therapy. IRBM is delighted to have been able to contribute to this pivotal work and to be cited on the publication. Huntingtin is the gene that…

Our integrated drug discovery efforts on the Relaxin-2 peptide hormone, in collaboration with our collaborators at global life sciences company Sanofi, has produced a paper just published in the Journal of Medicinal Chemistry. The natural peptide hormone human relaxin-2 has…

In the last few years macrocyclic peptides have emerged as a better class of lead candidates for inhibition of protein-protein interactions with respect to conventional small molecules. The IRBM peptide chemistry team collaborated with the teams at Merck & co. and RaPharma to develop potent macrocyclic peptide inhibitors of PCSK9 a key regulator of plasma LDL-cholesterol.

IRBM used liquid chromatography-high resolution mass spectrometry to explore different methods for the study of catabolism (breakdown) which is a key driver in the design of more stable peptides.

Trypanosoma brucei is a parasite responsible for human African trypanosomiasis, also known as sleeping sickness, a disease endemic in sub-Saharan Africa, with 70 million people at risk of infection. Current treatments are limited by their toxicity, administration in endemic countries and treatment resistance.

Human African trypanosomiasis (HAT), also known as sleeping sickness, is a parasitic disease that causes significant mortality in sub- Saharan Africa. A previous publication from IRBM laboratory reported the identification of 2-(1H-imidazo-2-yl)piperazines as a new series of potent T. brucei growth inhibitors.

IRBM, working with the University of Rome’s Department of Biology, examined signaling pathways involved with the regulation of Fibro/Adipogenic Progenitors (FAPs). FAPs are muscle-interstitial progenitors mediating pro-myogenic signals that are critical for muscle homeostasis and regeneration.

The blood-brain barrier (BBB) is responsible for the homeostasis between the cerebral vasculature and the brain. It has a key role in regulating the influx and efflux of substances, in healthy and diseased states. Stem cell technology offers the opportunity to use human brain-specific cells to establish in vitro BBB models.

The NRF2/ARE signalling pathway is a key mediator in oxidative stress and therefore a potential target for neuroprotective agents. The publication describes the identification of nonacidic tetrahydroisoquinolines (THIQs) that inhibit the interaction between NRF2 and its main negative regulator KEAP1.…