Galenea and CHDI Foundation have announced the extension and expansion of their Huntington’s disease (HD) collaboration using Galenea’s proprietary synaptic transmission drug discovery platform, Multiwell Automated Neuro TRansmission Assay (MANTRA).
The collaboration will focus on the characterisation of the synaptic dysfunction evident in HD and extending the findings in order to specifically target the mechanism to discover novel therapies for HD.
Galenea president and CEO Mark Benjamin said the platform is well suited to unravelling the synaptic dysfunction in HD and the discovery of synaptic-based therapeutics.
"The findings in the first phase of work were very compelling, and we see the potential for completely new approaches to this devastating disease," Benjamin added.
The causative defect in HD, mutant huntingtin protein, appears to disrupt normal synaptic function, contributing to the behavioural, cognitive and motor symptoms associated with the disorder.
The MANTRA system directly monitors synaptic events at the cellular level in primary neuronal cultures, and at the network level the system establishes in vivo electroencephalography measures of behaviours in rodent disease models by monitoring brain and behavioural activities in parallel.
The collaboration has employed the two-pronged approach to characterise the synaptic defects that occur in HD at both the neuronal and network levels.
CHDI target assessment director George Yohrling said the second phase of the collaboration with Galenea will focus on determining whether there is a polyglutamine-length dependency to the observed synaptic alterations, and on building a broad HD drug-discovery platform through the application of these novel findings.
"By determining the applicability of these synaptic and network changes, we will develop critical in vitro and in vivo screening tools to identify either small molecules or biological pathways that can ameliorate the synaptic effects of mutant huntingtin," Yohrling added.