Augustine Therapeutics Receives Funding to Research Treatments

Marisa Wexler MS avatar

by Marisa Wexler MS |

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PXT3003 for CMT1A

Augustine Therapeutics has received €1.2 million (about $1.4 million) in funding for a research project that will seek to further characterize the biology of Charcot-Marie-Tooth disease (CMT), and to investigate potential treatments.

The funding was awarded by the Flanders Agency for Innovation and Entrepreneurship (VLAIO), in Belgium.

“This funding is not only an excellent boost for our innovative research towards first-in-class medicines for CMT-patients; it is also an external validation of the quality and importance of our pioneering work. We are grateful for the financial support from VLAIO,” Sylvain Celanire, CEO of Augustine, said in a press release.

The project aims to better understand disease processes in CMT and unravel the mechanism of action of HDAC6 inhibitors developed by Augustine.

Acetylation is a biochemical process in which an acetyl group — two carbon atoms, three hydrogen atoms, and an oxygen atom, in a particular configuration — is added to a protein. When an acetyl group is removed, it is termed deacetylation.

Within cells, many proteins become acetylated or deacetylated in particular contexts, which is important for governing a myriad of cellular functions. HDAC6 — short for histone deacetylase 6 — removes the acetyl group from certain proteins.

Prior research has demonstrated that blocking the activity of HDAC6 shows therapeutic potential in preclinical models of different types of CMT. However, it is not clear exactly what effects these therapies exert at the cellular level.

“Inhibitors of HDAC6 hold great promise for the treatment of CMT and other neuropathies [nerve damage]. We already made great progress in understanding its mode of action. The new ambitions described in the project funded by VLAIO are the next steps for us to ultimately provide safe and effective solutions for patients,” said Ludo Van Den Bosch, founding scientist and member of the scientific advisory board at Augustine. Van Den Bosch’s team conducted pioneering work to identify HDAC6 as a relevant target for the treatment of CMT.

The newly funded research project, which is expected to last two-and-a-half years, is a joint effort between Augustine, VIB Discovery Sciences, and researchers at the VIB/KU Leuven Center for Brain & Disease Research.

“The VIB Discovery Sciences team and the VIB lab of Ludo Van Den Bosch have been continuously supporting Augustine in advancing this innovative project,” said Jérôme Van Biervliet, managing director of VIB.

“We are very happy with the financial support and recognition from VLAIO for the ongoing strategic collaboration between VIB and our spinoff Augustine Therapeutics … We look forward to the new treatment options our spin-off will develop to benefit patients suffering from neuromuscular diseases,” Biervliet added.