Areas of Research

Orphan Disease

Oculopharyngeal Muscular Dystrophy (OPMD)

The Disease

  • OPMD is a rare progressive muscle-wasting disease caused by a mutation in the poly(A)-binding protein nuclear 1 (PABPN1) gene
  • The disease is characterised by eyelid drooping, swallowing difficulties (dysphagia), and proximal limb weakness.
  • OPMD is typically diagnosed when individuals reach their 50’s or 60’s.
  • Dysphagia is a severe, life-threatening complication of OPMD.
  • There is currently no effective drug therapy for OPMD. Available interventions are limited to temporary palliative care and do not address underlying progressive muscle weakness.

The Benitec Approach

  • We believe our ddRNAi approach to ‘silence and replace’ the mutant PABPN1 protein will result in the correction of the muscular dystrophy and of key clinical features of OPMD including progressive atrophy and muscle weakness associated with nuclear aggregates of insoluble PABPN1.

Infectious Disease

Hepatitis B (HBV)

The Disease

  • Worldwide, 2 billion people have been infected with the hepatitis B virus (HBV) and 400 million people have become chronically infected.
  • An estimated 1 million people worldwide die each year from HBV and its complications
  • Chronic infection with hepatitis causes 80% of all hepatocellular carcinoma and more than 500,000 people die each year from this lethal cancer.
  • There is a need for safe and effective therapeutics that can promote the restoration of a host immune response through targeted hepatitis B surface antigen (HBsAg) knockdown offering HBV patients the potential for ‘functional cures’ by eliminating virus producing cells.

The Benitec Approach

  • We believe that a combination of BB-103 and a nucleoside inhibitor (NUC), a type of drug currently used to treat the HBV in infected individuals, may suppress a number of HBV parameters in humans including HBsAg. The HBsAg is a known contributor to immunosuppression and HBV chronicity.  The ability to suppress HBsAg may thus help spur the patient’s own immune system to produce anti-s-antigen antibodies which is expected to eliminate their need for their daily anti-viral treatments to control the disease.
  • Our pre-clinical results demonstrate that a one-time treatment of BB-103 added on top of a daily dosing regimen of a NUC, results in a far superior suppression of HBV parameters, including a greater than 2 log knockdown of HBsAg, as compared to that NUC inhibitor alone.

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