Encouraging preclinical data support rationale of Benitec's BB-301; shares ahead 55%

|About: Benitec (BNTC)|By:, SA News Editor

Nano cap Benitec Biopharma (BNTC +54.7%) spikes on a healthy 28x surge in volume on the heels of its announcement of preclinical data published in Nature Communications that show the potential of a DNA-directed RNA interference (ddRNAi) approach to treating oculopharyngeal muscular dystrophy (OPMD), a rare progressive muscle-wasting disorder caused by mutations in a gene called poly(A)-binding protein nuclear 1 (PABPN1).

Data from a mouse model showed that employing ddRNAi to "silence and replace" the mutant PABPN1 protein corrected the muscular dystrophy and the clinical features of progressive atrophy and muscle weakness.

The company and its collaborators have been working on BB-301, Benitec's next-generation ddRNAi therapeutic for the potential treatment of OPMD that combines the "silence and replace" strategy of mutant PABPN1 into a single vector. A Phase 1/2 study in OPMD is on tap for next year.