Lexicon Genetics Awarded Grant from the United States Army for Study of Spinal Muscular Atrophy
Lexicon Genetics Incorporated announced that it was awarded a grant from the United States Army Medical Research & Materiel Command (USAMRMC) for the identification of targets that may be important in the development of drugs to prevent or treat spinal muscular atrophy (SMA), a neurodegenerative disorder and the leading genetic cause of death in early childhood. Lexicon will receive $2.0 million in funding for the one-year initial term of the grant.
SMA is characterized by a mutation in the SMN1 gene that leads to neurodegeneration. Under the grant, Lexicon will utilize its proprietary gene knockout technology to identify genes that, when knocked out, lead to increased levels of mouse Smn protein. Genes that regulate Smn protein in mice may be involved in the regulation of SMN2 protein in humans. Identification of these genes may enable the development of drugs designed to increase levels of human SMN2 protein to offset the absence of human SMN1 protein and prevent or treat SMA. Lexicon will study approximately 750 pharmaceutically tractable genes in the research program. Lexicon has also entered into an agreement with the SMA Foundation for the potential development of drugs based on discoveries resulting from the program.
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