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Benitec Biopharma's Gene Therapy For Rare Muscle Disorder Shows Encouraging Action In Animal Studies

  • Benitec Biopharma Inc (NASDAQ: BNTCreports an interim analysis of the BB-301 Pilot Dosing study conducted in large animal subjects.
  • BB-301 a genetic medicine directly injected into the pharyngeal muscles. It employs the proprietary platform, which allows for a "Silence and Replace" approach to the treatment of Oculopharyngeal Muscular Dystrophy (OPMD).
  • OPMD is caused by a mutation in the gene encoding poly(A) binding protein nuclear 1 (PABPN1), wherein patients lose the ability to swallow liquids and solids.
  • The interim analysis showed biologically significant, highly-consistent, dose-dependent levels of BB-301 tissue transduction, with BB-301 copy numbers ranging from 1.7 copies per cell up to 8.6 copies per cell achieved after a single administration of increasing doses of BB-301.
  • Durable and biologically significant levels of target gene knockdown (inhibition of the expression of the gene encoding PABPN1) were observed.
  • Low-dose, intermediate-dose, and high-dose BB-301 administration achieved similar inhibition levels, with an average of 74% inhibition of PABPN1 expression observed across all doses.
  • Following an optimized route and method of administration, Benitec demonstrated a 248-fold improvement and a 111-fold improvement in BB-301 transduction of the two key pharyngeal muscles relative to the levels of BB-301 transduction observed in the analogous Beagle dog study conducted by the previous BB-301 licensee.
  • The company has scheduled a Scientific Advice Meeting in France in May to review the interim data and the Phase 1 clinical trial design.
  • The company will provide additional pipeline updates in 2H2021. Plans to initiate human trials of BB-301 in OPMD patients in 2022.
  • Price Action: BNTC gained 5.8% at $3.97 in premarket trading on the last check Wednesday.

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