California Stem Cell this week said it will supply BioFocus DPI, the service division of Galapagos, with its Motorplate 96-well assay-ready plates containing motor neuron-progenitor cells derived from human embryonic stem cells.
The plated, assay-ready cells, which debuted earlier this year, will help BioFocus to develop assays and screen knockdown constructs from its proprietary adenovirus knockdown library for the ALS Association’s Translational Research Advancing Therapy for ALS, or TREAT ALS, drug-discovery and clinical trials program.
Specifically, BioFocus will use the cells for target discovery applications with the goal of identifying druggable targets for ALS, Katherine Hilyard, vice president of biological sciences for BioFocus, told CBA News this week.
Once BioFocus identifies potential targets that may affect ALS, “the next step would be to screen small molecules against those targets. The overall goal would be to find a compound that would inhibit the target, which you could then use against the disease,” said Hilyard.
She added that BioFocus has already tested the cells to make sure that “we can handle them in our laboratory, and we are very confident that we will be able to use these cells in the assays that we need to do.”
Chris Airriess, COO of California Stem Cell, told CBA News this week that the company “had already decided that we wanted to make these [motor neurons] available to people for high-throughput and high-content screening and cell-based assays, so we packed them up in 96-well plates.”
Then CSC penned the deal with BioFocus, which “worked with us to actually finalize the product, so we are going forward with it,” said Airriess. “We were developing this product anyway, but we worked with BioFocus to validate it and make sure that, basically, the cells were expressing all of the markers necessary to guarantee that they are motor neurons.”
“We can handle [the cells] in our laboratory, and we are very confident that we will be able to use these cells in the assays that we need to do.”
CSC also worked with BioFocus to develop shipping protocols, so “we can ship these cells around the world and have them arrive in a perfectly healthy state so that they can be used in assays over a period of a few days,” Airriess added.
Irvine, Calif.-based CSC was founded in 2005 and “really started ramping up operations in September 2006,” because the company had raised the necessary funds, said Airriess. “Our primary goal is to develop cell-based therapeutics for several different neuromuscular diseases, including ALS, spinal muscular atrophy, and spinal cord injury.”
Secondarily, the company is “growing cells for sale as an internal business unit that is separate from the cell therapeutics business,” Airriess said, adding that the focus and timelines for the two businesses are quite different.
As a result of developing these cells for therapeutic use, “we realized that we had a potentially viable product that we could sell by plating out our cells in an assay-ready plate to companies that want to do high-throughput or high-content screening,” he said.
CSC currently employs nine people and works with a number of contract research organizations on a consultancy basis. “We will probably double in size over the next year,” Airriess said.
He added that CSC is looking to hire technicians and product managers for the assay-ready cell products, and will be hiring clinical trial managers and scientists for the clinical development side of the business.
In addition, CSC has a GMP facility to manufacture the cells, which it claims “are actually grown according to the same criteria as the cells that we are producing for the clinic.”
Over the next two to three years, “we are really hoping to penetrate the cell-based-assay market quite significantly,” Airriess said. He said the company currently has motor neurons and cardiomyocytes available for sale.
In addition, “everyone also wants hepatocytes, so that is also something that we are thinking about,” he said, adding that CSC is currently developing a number of different cell types.
CSC has sold its Motorplate assay to other companies, as well. “We have some one-off sales to other companies, just a few plates at a time,” said Airriess. He added that CSC is “in the middle of negotiating some larger deals. We will make announcements on those as they come up.”
Airriess said CSC is currently looking to raise an undisclosed amount in a Series A round of venture capital and hopes to close the round by the end of June.
“We are in active negotiations with a number of parties right now,” he said, without elaborating.
Airriess said that the company will use the money to increase its manufacturing capacity for the cell-based assay plates and fund the clinical development of lead compounds.