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In Deals, BioFocus, CHDI to Co-Develop Mouse Assay to Screen Huntington Rxs

Galapagos service division BioFocus and the Huntington's Disease Initiative Foundation this week announced a pair of drug-discovery alliances that focus on cell-based assays.
In the first collaboration, BioFocus and CHDI plan to co-develop novel drug-discovery assays to evaluate compounds as potential new therapies for Huntington’s disease.
Terms of this deal, for which CHDI will pay BioFocus $1 million over 18 months, call for BioFocus to develop a high-throughput screening assay using mouse neurons to identify compounds that prevent HD-associated neuronal dysfunction, a BioFocus official said this week. 
“It involves quite complex cell biology to culture the neurons and keep them alive,” Kate Hilyard, vice president of biological sciences for BioFocus, told CBA News this week.
To develop the assay, BioFocus also has to transduce the huntingtin gene mutation into these neurons. It will do that with its adenoviral platform.
Hilyard also said that BioFocus does not already have a mouse neuron assay and is “developing that for [CHDI].”
However, she said she does not think BioFocus will use this assay for projects with other clients. “This is something that we are developing specifically for CHDI, with the huntingtin phenotype, the huntingtin readout,” she explained. If the assay development is successful, BioFocus could use the assay to test compounds sent by CHDI.
For the second project, BioFocus will test compounds known to inhibit enzymes that play a key role in HD development. CHDI will perform all of the disease modeling studies in animals.
“It’s looking at known compounds that target those enzymes, to examine the properties of those compounds, and then using those compounds to determine if that enzyme is a good target for the disease,” Hilyard said.
She declined to elaborate regarding the identity of the enzymes or the compounds, except to say that the alliance “is primarily a biology program, but we will do some chemistry as well to make the compounds.”
Besides funding BioFocus’ work, CHDI will provide the company with certain reagents, and, as part of its work, BioFocus will provide reagents back to CHDI, said Hilyard.
‘Between a Partnership and a CRO’
BioFocus and CHDI have been collaborating since August 2005, and have partnered in the area of target discovery. The two new programs, therefore, “will expand on that, and use some of the capabilities we have in drug discovery to work with CHDI,” Hilyard said.
A number of CHDI’s research programs begin with target validation. The organization wanted to see what druggable targets would be relevant for HD. “So we worked with BioFocus there,” said Bonnie Lee La Madeleine, communications director for CHDI. She explained that the relationship was something “between a partnership and a CRO.”
“They helped us develop that program early on, and helped us determine how we would work through the target-identification and -validation stages of our program,” La Madeleine said. She added that CHDI’s target validation program will stay operational. 
“Once we know a bit more about the compounds, for example, that they actually do inhibit our enzymes of interest, those compounds will be tested in animal models of HD to try and validate that target,” Hilyard said.
If the target is validated, then it would become very interesting for drug discovery programs, she said.

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