Partnership aims to advance disease modifying therapy for Huntington’s disease

A company specializing in therapeutics for rare and neurodegenerative diseases has announced a partnership with a provider of innovative solutions in early-stage drug discovery to develop a BRD9 inhibitor to treat Huntington’s disease.

According to a joint release issued by Rumi Scientific and X-Chem, bromodomain 9 (BRD9), an epigenetic reader, was selected as the novel therapeutic target for HD with the potential for disease modification.

Rumi successfully validated this target in the R6/2 model of HD and in cellular models that replicate various aspects of the HD phenotype. A key result from these studies, the company said, is that lowering of BRD9 levels can restore striatal volume, which strongly indicates disease-modifying potential.

“BRD9 inhibition is a novel target for the treatment of HD identified by our proprietary high throughput organoid phenotypic screening platform,” Rumi CEO Allen Fienberg, PhD, said in the release. “We look forward to working closely with the team at X-Chem on the development of the lead molecule, and to advancing it toward clinical studies.”

Rumi and X-Chem plan to design a screening cascade for BRD9 that maximizes speed, efficiency, predictive accuracy and translational validity, harnessing Rumi’s expertise in HD assays at the cellular and tissue levels.

“We are excited to be partnering with Rumi Scientific in their pursuit of a lead candidate iBRD9 for [Huntington’s disease],” X-Chem CEO Matt Clark, PhD, said in the release. “We are particularly excited about the unique promise of the Rumi platform and its potential to discover additional novel targets that we can take to the next level.”