During Benitec's Q3 2018 financial conference call in May 2018, Chief Science Officer David Suhy, talked about BB-301 studies being conducted in sheep. Benitec chose sheep because the size of muscles in the upper digestive system are relatively consistent with humans. Benitec intended to inject the gene therapy directly into the muscles affected by the mutant gene, named cricopharyngeal, and surrounding muscles.
At the time, Benitec was near the endpoint of the study involving 19 sheep. While the study was not complete at that time, Benitec didn't observe any abnormal safety issues with BB-301. Complete analysis was to include toxicology, histology, and studies to verify expression of both of ddRNAi and the protein associated with PABPN1 gene.
Benitec was to initiate an expanded animal study in 54 sheep as a definitive regulatory and toxicology studies to support entry of BB-301 into the clinic. The toxicology studies were said to take approximately 90 days for each animal, once each animal has been treated with a single dose of the therapy. "
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