The humble safflower seed is looking like it could be the next protein-producing factory after SemBioSys Genetics produced commercial levels of an atherosclerosis treatment.
The Canada-based biotechnology company has successfully modified safflower seed lines, and expressed and collected apolipoprotein A1 and its variant apolipoprotein A1 (Milano), collectively referred to Apo A1,which is a next-generation cardiovascular drug that targets the removal of atherosclerotic plaque from arteries.
The development represents a move into the $35bn cardiovascular drug market, but the seed lines have also proved successful in producing about 43 other therapeutic proteins at a fraction of the cost of conventional methods, SemBioSys chief scientific officer Dr Maurice Moloney told US-PharmaTechnologist.com.
The transgenic technology, which is looking as an increasingly popular tool for producing proteins, was the brainchild of Moloney, who developed the genetically modified "Roundup Ready" crops.
In the safflower seed case, the Apo A1 gene is only expressed in the seed. The technology, which can be used in any oil seed, has been further developed so that the protein attaches itself to oil bodies in the seed making the recovery of the protein much easier as the oil floats out of the solution when the seeds are crushed to extract the protein.
"This dramatically reduces the cost of purification," Moloney said.
The company decided to focus on safflower because it was a less common crop, compared to canola, so it was easier to biologically isolate the crop reducing the chances of cross pollination or the accidental mixing of seeds with non-transgenic ones, he said.
Meanwhile, developing the seeds as the protein-producing factories instead of another part of the plant, meant the company could utilize the long-term storage capabilities that seeds have as well as being able to adjust production of the protein depending on the market demand.
"This is difficult to do if you are dealing with conventional methods like fermentation," Moloney said.
The cost advantages were also very promising over conventional methods, he said.
The cost to grow a ton of seed, about an acre of safflower which would then produce about 2kg of Apo A1, would be about $800. This is compared to the current cost which sits in the range of about $400 per gram of Apo A1.
Moloney estimated the technology could be developed to reduce the cost by 90 per cent.
As a result, the safflower seed technology would be particularly beneficial in the production of Apo A1, as large amounts of the protein were required for treatment, he said.
SemBioSys president and chief executive Andrew Baum said in a statement: "We believe manufacturing capacity and cost are major commercialization issues for pharmaceutical companies developing Apo A1. Today's announcement demonstrates that safflower seed is an enabling production vehicle for Apo A1 with the scale and economics necessary to allow for commercialization of this potentially transformative therapy."
Apo A1 has been described as a promising new therapy for the treatment of cardiovascular disease. It is the major component of the good cholesterol, high density lipoprotein (HDL), which naturally removes atherosclerotic plaque from arteries.
Currently, Pfizer is developing Apo A1 via E.coli, with positive clinical trial results, while CSL and Borean Pharma have both recently confirmed the strong therapeutic potential of their respective Apo A1-based drug candidates, CSL-111 and Trimeric Apo A-I, in clinical and preclinical trials.
SemBioSys intends to scale up production of safflower-produced Apo A1 and perform the necessary preclinical work in 2008 in order to initiate clinical trials in 2009.
The company has also produced authentic human insulin in safflower, which is expected to enter clinical trials in early 2008.
SemBioSys is now planning to initiate development program partnerships.