Exon skipping technology will be used to overcome the effects of certain genetic errors in the dystrophin gene.
AVI BioPharma received $2.45 million from Charley’s Fund, a nonprofit with a focus on Duchenne muscular dystrophy (DMD). The funding will support the company’s development program that uses exon skipping technologies developed along with its partner, Ercole Biotech, to overcome the effects of certain genetic errors in the dystrophin gene.
The focus of the funded program will be to select and develop a lead molecule based on AVI’s Neugene® chemistry that is designed to skip dystrophin exon 50. The company notes that this will restore the proper RNA reading frame and production of functional dystrophin in patients with a particular type of mutation.
“The technologies developed by AVI and its collaborators have incredible promise in addressing the fundamental cause of DMD,” points out Benjamin Seckler, M.D., president of Charley’s Fund. These methods allow manipulation of the RNA splicing process and the production by cells of clinically desirable variants of relevant proteins.