The firm, part of Sigma Aldrich, will use its CompoZr zinc finger nuclease (ZFN) platform to create knockout rat strains representing various symptoms of autism and related disorders like Fragile X and Rett syndrome.
Unit director Edward Weinstien told Outsourcing-pharma SAGE anticipates a strong response from the preclinical research sector when the new strains become commercially available next year.
“While there are many mouse models available for Autism research, I believe the overwhelming consensus is that the mouse is a suboptimal model for this condition.
“Our conversations with the research community, greatly facilitated by the members of Autism Speaks, make us believe that genetically engineered rats will be extremely valuable.”
Research funding
But despite SAGE’s positive prediction, quite what the level of pharmaceutical industry demand for rodent autism models remains to be seen.
According to a recent PhRMA report, at present, only nine of the 313 drugs currently being developed to treat all forms of mental illness are intended as therapies for autism spectrum disorders.
The reason, according to Life Science Analytics president Sarah Terry is risk. In an interview with Psychiatric News She explained that autism “is not very well understood [and] it's somewhat seen as a syndrome and constitutes different symptoms.
“Very few [pharmaceutical] companies are [developing autism treatments at present] because it is high risk, and we're still in the bench-science stage of understanding the disease.”
Weinstein acknowledged all these factors, admitting that while some drug firms are making progress, efforts are “hampered by a number of factors, including an uncertain genetic basis, and inadequate animal model systems.”
“However,” he continued “I think Autism is definitely a treatable disorder, anything is, if there is sufficient funding and resources. SAGE Labs simply hopes to make our contribution to the process by providing quality reagents.”
So, while risk aversion may be stifling development of autism treatments at present, the advent of improved models may yet prove to be the tipping point that sees the therapeutic niche hungry pharmaceutical industry finally invest in developing treatments for this complex disorder.
Zinc finger
SAGE licensed CompoZr from Sangamo BioSciences in 2007 and has already developed models for a number of central nervous system and cardio vascular diseases as well as for a number of toxicology testing applications.
The growing importance of the technology, marketed as the SAGEspeed platform, and SAGE’s wider research models’ business was highlighted back in March when the firm bought rodent breeder Ace Animals.