080210xf's Blog

L'X fragile sera vaincu | Fragile X will be conquered

SAGE Labs teams on preclinical Autism model development

By Gareth Macdonald |

SAGE Labs will create rodent autism models for preclinical drug discovery and development in collaboration with advocacy group Autism Speaks.

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 the firm 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.”


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