The Food and Drug Administration (FDA) has granted Orphan Drug designation to AL001 (Alector), an investigational treatment for patients with frontotemporal dementia (FTD). 

AL001 is a human recombinant monoclonal antibody designed to increase levels of progranulin, a secreted glycoprotein, in the brain; mutations within the progranulin gene (GRN) can lead to frontotemporal lobar degeneration which presents as FTD.

According to the Alzheimer’s Association, “there may be 50,000 to 60,000” people with FTD in the US. There are currently no treatments available for the condition. 

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“FTD is caused in part by a dysfunctional brain immune system, and AL001 is one of our immuno-neurology drugs designed to repair that immune system to elicit a therapeutic benefit.” said Arnon Rosenthal, PhD, CEO, Alector. “We believe that our immuno-neurology approach could potentially have as much impact on brain disorders as immuno-oncology drugs have had on cancer patients.” 

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