Benitec Biopharma Inc. – a US biotechnology company focused on the development of novel genetic drugs. Its patented platform, called DNA-directed RNA interference, or ddRNAi, combines RNA interference with gene therapy to create drugs that promote sustained suppression of disease-causing genes after a single administration. The Company is developing ddRNAi-based therapeutics to treat chronic and life-threatening human conditions, including oculopharyngeal muscular dystrophy (OPMD).

The BB-301 drug has been granted orphan drug status in the US and the European Union, which provides opportunities for effective regulatory review and approval of the drug.

On 8 September, the company presented a comprehensive design review of the upcoming clinical trial, a Phase 1b / 2a study scheduled for 2022. In addition to determining the safety and tolerability profiles of BB-301, secondary endpoints of the study will contribute to the accurate and reproducible characterisation of the underlying physiological processes associated with successful completion of the oro-pharyngeal phase of swallowing. The primary analytical methods of the clinical study, will focus on functional measures of swallowing performance in patients with OPMD during the oro-pharyngeal phase of swallowing.