Benitec Biopharma Limited is pleased to announce it has developed a new single vector (gene therapy construct) system which uses DNA directed RNA interference (ddRNAi) to silence expression of the mutant gene associated with oculopharyngeal muscular dystrophy (OPMD), while simultaneously adding back a copy of the normal version of the same gene to restore gene function. This next generation single vector system, termed BB-301, represents the clinical candidate that Benitec intends to advance into human clinical trials in the second half of calendar year 2018.
Simultaneous silence and replace modalities are required for an effective gene therapy for OPMD and other orphan diseases. By combining both ‘silence and replace’ functions into a single vector, Benitec can focus its manufacturing efforts for the program on a single product, which vastly simplifies the regulatory process and reduces the complexity of the clinical strategy. The innovative Benitec vector design which integrates both ‘silence and replace’ modalities into a single vector is not readily achievable with other gene therapy and gene editing technologies.
Greg West, Chief Executive Officer, commented on today’s news: “This is an important development in our OPMD program. The single vector system shows the same excellent activity as the earlier generation dual vector system where the ‘silence and replace’ constructs were delivered in separate vectors. Similar application of the single vector technology may allow development of novel therapeutics to treat other orphan diseases. OPMD is a significant commercial opportunity for Benitec and we are working with the regulators and key opinion leaders in this field to advance BB-301 into the clinic as quickly as possible.”
Background information:
OPMD is a rare progressive, muscle-wasting disease caused by mutation in the poly(A)-binding protein nuclear 1 (PABPN1) gene, that is characterized by eyelid drooping, swallowing difficulties, and proximal limb weakness.
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