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Publications 2021

IRBM cited in UniQure publication on using HTT-lowering therapies to slow down neurodegeneration in Huntington’s Disease

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 causes Huntington’s and therapies attempting to lower the levels hold the potential to slow down neurogeneration. UniQure’s approach uses recombinant adeno-associated viral vector serotype 5 expressing a microRNA targeting human HTT (rAAV5-miHTT).

The study demonstrates widespread biodistribution, strong and durable efficiency of rAAV5-miHTT in disease-relevant regions in a large brain. These results pave the way to future studies which look to advance treatment options for Huntington’s Disease.

Identification of Potent and Long-Acting Single-Chain Peptide Mimetics of Human Relaxin-2 for Cardiovascular Diseases

IRBM and Sanofi publish in J. Med. Chem. on optimizing the Relaxin-2 peptide hormone in cardiovascular diseaseOur 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 a complex heterodimeric insulin-like structure that makes its chemical synthesis tractability quite challenging.
The goal of our collaboration was to reduce the chemical complexity and optimize the hormone to a highly potent single B-chain Relaxin-2, with sustained duration of action and an improved half-life suitable for once daily administration.
The new class of RXFP1 agonists described in J.Med. Chem. paper have long-lasting properties that are compatible with once daily sub-cutaneous administration in patients. This approach opens the door to new treatments for chronic fibrosis and cardiovascular diseases.

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