Though genetics has fundamentally changed the pace of drug development for many therapeutic areas, the brain has been slow to reveal its inner workings. A new generation of neuroscience-focused biotech companies is poised to change that paradigm.
[R]esearchers at a new generation of biotech firms think the brain can be coaxed into giving up its secrets. Armed with genetic insights, therapeutic technologies, and diagnostic tools only dreamt about a decade ago, they are taking a page from more successful corners of the biopharmaceutical world such as oncology.
Research institutions like Broad, through its Stanley Center for Psychiatric Research, are working to identify genetic risk factors for psychiatric diseases such as schizophrenia through large-scale studies. The Deciphering Developmental Disorders project, undertaken by the Wellcome Trust Sanger Institute, likewise uses genomic tools to uncover the genetic causes of rare developmental disorders.
Disease-focused philanthropic organizations such as the Michael J. Fox Foundation for Parkinson’s Research and the ALS Therapy Development Institute are exploring new endpoints for clinical trials; collecting genetic, clinical, and behavioral data and biological samples from patients; and working with industry partners to exploit these increasingly large data sets.
Because sequencing can generate so much data relatively inexpensively, researchers feel like they’re on the cusp of understanding much more about diseases such as Parkinson’s and ALS.
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