Taking the mystery out of biotech: Local companies explain their sophisticated science in plain English (really).
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Stemina would gather additional clinical data from patients at the CLIA lab to make an FDA submission and eventually place a diagnostic test on the market. The strategy is to get the buy-in of thought leaders in the Mind Institute and the Waisman Center, build a database of clinical results, and move to pediatricians and other first-line providers who would prefer a test they can deliver in the clinic.
Stemina has raised $3.25 million in state and federal grants, $4.6 million in angel funding, and is in the process of raising another $1 million while it waits to hear from the Environmental Protection Agency, which is soliciting proposals for its ToxCast program. Under ToxCast, the EPA is trying to build a computer model of human developmental toxicity, and Stemina is vying for a contract of between $5 million and $50 million.
“The proposal is to screen the ToxCast library of compounds using our devTox assay, and provide those biomarkers to their ‘virtual embryo’ program,” Donley said. “Right now, there are roughly 1,800 compounds enrolled in that program, and there may be as many as 10,000 over the five-year length of the contract.”
Cancer is another disease in Stemina’s crosshairs. The company has a $1 million contract with the National Cancer Institute to examine the biomarkers of cancer stem cells. In cancer research, cancer stem cells are thought to be the seeds of recurrence and metastases, so while existing treatments reduce overall tumor size, “we’re not as good at killing the cancer stem cell, so it sort of just smolders there,” Donley said. “The idea is to identify whether or not we are being effective at killing the cancer stem cell with therapy, and to monitor biomarkers in blood or urine to see if the cancer stem cell is still lurking somewhere.”
This study is being conducted in collaboration with the UW’s McCardle Cancer Center and with Drs. John Kuo of the UW and Jann Sarkaria of the Mayo Clinic, and the eventual goal is to create a personalized medicine approach to treatment. Donley said researchers might find effective therapies in drugs that didn’t reduce tumors as rapidly as chemotherapy but could be more effective in killing cancer stem cells.
Harry Rossiter Horn uses the term “ruggedized antibodies” in reference to proteins that have been engineered to be stable and work in elevated temperatures and other harsh conditions. Since they are being used for bio-threat detection in support of the Department of Defense’s Advanced Research Projects Agency, they better be tough.
Horn, CEO and COO of StableBody Technologies, or SBT, a privately held protein engineering company, noted that SBT’s products will be counted on to uncover bio-threats in very small amounts in the environment – soil, water, and air – and in the human body. What SBT hopes to become is an industry leader for the stabilization and optimization of proteins referred to as next-generation stable proteins (NGSP).
Whether they are next-generation or old-school, they must provide an added dimension over antibodies, which are proteins produced by the body’s immune system. Antibodies generally are found in the blood, and they detect and destroy bacteria and viruses, but while they probably are the best detection systems, they have not been stable enough to use in extreme conditions.
SBT’s proteins have shown protein stability at temperatures of over 80 degrees Celsius for many days; at room temperature, this protein stability has been calculated to more than three years. This provides the business case for SBT’s product platform, which combines science and bioinformatics in the design stable reagents (chemical substances used to create a reaction in combination with another substance).
“We are working to reduce the amount of cold or cool chain storage and distribution costs for protein reagents,” Horn stated. “These costs are in the hundreds of millions of dollars for the biotech, pharma, and diagnostic industries.”
In addition to licensing its products to major pharmaceutical companies and the Department of Defense, which provide revenue and long-term royalty streams, SBT has a broad enough platform to branch out. The company is subcontracting on a grant from the National Institutes of Health that will add to its environmental detection capability by providing what Horn called a unique activity in the bioremediation field. “Our reagents cannot only detect environmental toxins, they can be used to capture those toxins and get rid of them,” he stated.
SBT also has the opportunity to generate revenue through corporate partnerships. As part of these relationships, the company would provide its core technology for the creation of new compositions of matter. In addition to bio-threat detection and bio-threat countermeasure programs with defense contractors, its target markets include the health care industry.
In health care, it would sell directly to the developers of therapeutics, diagnostic, and detection firms. “We have a number of collaborations underway with revenue expected in the next 12 months,” Horn indicated.
In addition, SBT has applied its technology to protein reagents in the cancer field. The program is focused on the development of diagnostic, therapeutic, and prognostic reagents for use in fighting aggressive solid tumor growth. Among its high-value proteins are ones that potentially can be used for the detection and treatment of vascularized tumors, a market that is approaching $50 billion.
As a result, the company is seeking private equity via angel investors. “We have been in business for three years now, and we’re totally self-sufficient through contracts,” Horn noted. “We now have a major cancer treatment and detection program that we want to accelerate and get to market quicker for the benefit of the cancer field.”
The products will have to go through clinical trials for the health care market, but the company will not provide for that. That’s where partnering is important.
“We believe our first cancer diagnostic reagent will be available within 12 to 16 months – available for acquisition or licensing to a diagnostic company,” Horn stated. “We will not have to go through the Food and Drug Administration approval process. We just develop the reagent, prove that it works, and then the company [partner] will take it through regulatory clinical testing.”
Stem cell technology plays a role in product development. The cancer program is based on embryo-genic protein biomarkers (small molecules) produced in human embryonic stem cells – the “Nodal” protein is a potent inducer of blood vessel growth – and there also is potential for induced pluripotent stem cells, derived from adults, to aid the development of SBT’s products.
“We’re working on many targets and reagents at this time with various clients in the health care market, bio-threat defense, as well as environmental testing,” Horn summarized. “We tend to refer to the old BASF slogan: We don’t make the product [reagent], we make the product better.”
The mission of BellBrook Labs is to accelerate drug discovery, and in assembling a portfolio of proprietary tools for that purpose, the business has commercialized two technology platforms and found other ways to build value for the angel investors who have placed a total of $3.7 million of good faith in the organization.
President and CEO Robert Lowery has a good deal to show for his 10 years at the helm of BellBrook. It starts with the two technology platforms: Transcreener for biochemical high-throughput screening, or HTS, which enables researchers to screen thousands of different enzymes with just a few assays; and the iuvo micro-conduit array, another HTS device that uses special micro-channel geometries instead of wells to enable more realistic cellular models.
Transcreener is focused on specific protein drug targets because, in many diseases, researchers are able to identify a specific protein that acts aberrantly. To intervene in that disease, they want to develop a drug molecule that disrupts the function of that protein. “When the pharmaceutical companies or academic labs are screening these huge compound libraries, our assays tell them when they are getting a hit when one of those compounds is interacting with the target of interest,” Lowery explained. “The main advantage of our platform is that it streamlines screening kits. They have four different assays, and between those four different assays they can screen thousands of different protein targets.”
Whereas Transcreener provides screening for defined biochemical targets, iuvo is used to screen whole cells. The idea behind iuvo is to advance cellular assays’ high-throughput screening by enabling people to develop cell models that are representative of the tissues in your body. Nearly all assays, or tests, are run with cells growing flat on a single layer of plastic, but that’s not how cells grow in the human body. “What we’re doing with iuvo is giving people a flexible platform that they can use to produce cell models that are much more physiologically relevant,” Lowery stated.
Providing validation, BellBrook has entered into a deal with Thermo Fisher, one of the world’s largest life science companies, to distribute iuvo.