Rare Disease Spotlight: Rare form of dementia a gateway indication for PGRN?

With human genetics promising to remove some of the risk from neurology drug development, interest has grown in targeting genetically defined subsets of neurodegenerative diseases, even if that means limiting the target market. GRN-mutant frontotemporal dementia is a prime example.

Various mutations and SNPs in the GRN gene are risk factors for neurodegenerative conditions, including frontotemporal dementia (FTD), amyotrophic lateral sclerosis (ALS), and Alzheimer’s and Parkinson’s diseases. Based on the known functions of the gene’s protein product, PGRN, as a growth factor and anti-inflammatory factor in the CNS, one could make a case for enhancing PGRN function in a variety of disease contexts and patient populations...