Boston’s Autism Mini-Cluster: New Drugs and Diagnostics Target Mysterious Brain Disorders
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this hub of medical research. Scientists at multiple local research institutions—Massachusetts General Hospital, the Broad Institute of Harvard and MIT, and others—are affiliated with Harvard or its Autism Consortium. For example, Harvard scientists were behind the recent discovery of genetic and chromosomal patterns behind certain cases of autism and related brain disorders.
At MIT, there are researchers from multiple departments advancing autism research. Perhaps most notably, MIT professor and Nobel laureate Susumu Tonegawa led a study that showed that blocking a certain enzyme in mice reversed the symptoms of Fragile X syndrome, thought to be a cause of autism. The San Diego-based biotech startup Afraxis is in human testing of a drug that targets such enzymes as a potential treatment for Fragile X and autism.
This biotech startup, co-founded by the MIT neuroscientist Mark Bear in 2005, is also advancing treatments for autism and Fragile X syndrome. The company’s lead drug, STX209, is in mid-stage clinical trials for both Fragile X and autism spectrum disorders, according to a government website that tracks clinical trials. In September 2009, Luke covered the firm’s $30 million round of financing from a family investment fund. At that time, the firm had raised $66 million from the family fund, the National Institutes of Health, Autism Speaks, and the Fragile X Research Foundation, company CEO Randy Carpenter told Xconomy.
If I were asked to make a short list of the Boston area’s top diagnostics entrepreneurs, Stan Lapidus would definitely be on it. Lapidus—who is a founder of Cytyc, a Massachusetts provider of women’s health diagnostics that was acquired by Bedford, MA-based Hologic (NASDAQ: HOLX) for $6.2 billion in 2007—has recently formed SynapDx to develop blood-based tests for autism. In June, the firm announced that it had raised $9 million in a Series A funding round. Lapidus is president and chief executive of the firm.