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Monday, November 2, 2009

Seaside launches trial for autism treatment

By Julie M. Donnelly

Seaside Therapeutics LLC has begun a Phase 1 clinical trial for STX-107, a drug target that is designed for the treatment of Fragile X Syndrome. Fragile X Syndrome is the most common inherited form of mental impairment and the most common known cause of autism.

“Individuals with Fragile X and other related brain development disorders need effective therapeutics directed towards the underlying disease pathophysiology, rather than just the associated symptomatic behaviors,” said Thomas R. Insel, M.D., Director of the National Institute of Mental Health, in a statement.

Fragile X syndrome, which affects 90,000 Americans, is caused by a mutation of a gene that prevents the body from making a protein key to normal brain function, according to the company. Seaside said research has shown that neurological and psychiatric symptoms of the syndrome have been linked to excessive signaling of a separate gene, and scientists have identified more than 400 chemical compounds that could inhibit that gene.

There is currently no approved drug for the treatment of Fragile X Syndrome. Drugs currently used primarily focus on treating specific symptoms of autism such as anxiety, or improving and controlling behavior.

Seaside has won grants from several agencies at the National Institutes of Health and patient advocacy groups to support the development of STX107, which is intended to “normalize” the exaggerated neurological signaling that is linked to Fragile X.

Seaside was founded in 2005 by Mark Bear, Picower Professor of Neuroscience at MIT, who developed the research behind the drug candidate.







 

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