Firms’ technologies allow manipulation of RNA splicing and production of variants of proteins by cells.

AVI BioPharma and Ercole Biotech are working together to develop drugs that are more effective in treating Duchenne muscular dystrophy (DMD) and beta thalassemia.

According to the cross-licensing and collaboration deal, AVI will lead the DMD program and Ercole will head the thalassemia program. Each company has the option of co-funding the program led by the other party and sharing equally in the financial returns from resulting products. AVI will issue Ercole shares of its common stock, and Ercole will issue AVI shares of Ercole Series A-2 preferred stock.

“The potential treatments of Duchenne muscular dystrophy and beta thalassemia are important and compelling applications of the technologies that Ercole and AVI have pioneered,” notes K. Michael Forrest, CEO of AVI. 

The tools developed by Ercole and AVI allow manipulation of the RNA splicing process and the production of clinically desirable variants of relevant proteins by cells. AVI refers to its therapeutic approach as ESPRIT (Exon Skipping Pre-RNA Interference Technology). Ercole’s Splice Switching Oligonucleotide (SSO) drug discovery platform redirects mRNA splicing. AVI believes that its morpholino chemistry is particularly useful in modifying splicing of mRNA because oligonucleotides based on this chemistry do not degrade target RNA and do not lead to down-regulation of the target gene.

“The feasibility of correcting splicing to treat Duchenne muscular dystrophy and beta thalassemia has been well demonstrated in animal models by AVI, Ercole, and our academic collaborators,” points out Ryszard Kole, Ph.D., president and CSO of Ercole. “It is advantageous that Ercole’s splice switching technology and AVI’s morpholino chemistry are so well suited for this purpose.”

This is the second agreement between AVI and Ercole. In December 2006, the companies inked a cross-license and collaboration agreement covering a number of undisclosed therapeutic targets. 

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