Areas of Research




Head and Neck, Squamous Cell Carcinoma (HNSCC)

The Disease

  • Head and neck cancers typically begin in the squamous cells that line the moist mucosal surfaces inside the head and neck, such as the mouth and the throat.
  • HNSCC is more than twice as common in men compared to the rate of occurrence in women.
  • HNSCC accounts for more than 90% of all head and neck cancers
  • More than 50% of HNSCC patients are diagnosed with locally advanced or metastatic disease, which has a higher potential for progression and recurrence.
  • In 2016, approximately 64,000 new cases of head and neck cancer were diagnosed in the U.S., resulting in more than 13,000 deaths.
  • The relative five-year survival rate for metastatic head and neck cancers is <38%, and can be as low as 4% for recurrent or metastatic forms of the disease.


The Benitec Approach

  • The Epidermal Growth Factor Receptor (EGFR) is a well validated oncology target and a key driver of the growth of HNSCC lesions. More than 90% of HNSCC lesions exhibit significantly elevated levels of EGFR versus concentrations found in non-malignant tissues.
  • BB-401 is a DNA construct that produces an antisense RNA that targets EGFR. We believe that a targeted and rationally-designed approach to treating HNSCC could facilitate durable tumour size reductions or complete eradication of malignant lesions and may lead to an improvement in the quality of life and clinical outcomes for patients suffering from this disorder.
  • We are also developing a follow-on compound utilizing our proprietary ddRNAi proprietary technology to silence EGFR. Named, BB-501, early stage iterations of this candidate are currently being tested in preclinical animal testing using xenograft tumour models.


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.


Retinal Disease


Age-Related Macular Degeneration (AMD)

The Disease

  • AMD accounts for 8% of blindness worldwide and has been projected to impact up to 196 million patients by 2020 and up to 288 million by 2040.
  • The wet form of the disease accounts for about 10% of all AMD patients but accounts for up to 90% of all the blindness.
  • Wet AMD is characterised by the growth of new blood vessels into the eye, a phenomenon that has been associated with the expression of abnormally high levels of proteins from the vascular endothelial growth factor (VEGF) family.
  • The most commonly used standard of care treatments for AMD require an intravitreal injection into the eye as frequently as monthly or bi-monthly. These frequent injections may be required indefinitely to be able to halt progression of the disease and stabilise vision.


The Benitec Approach

  • Our AMD product candidate, BB-201, is comprised of a novel adeno associated virus capsid (AAV) and a recombinant DNA cassette, engineered to express steady state levels of three short hairpin RNA that inhibit VEGF-a, VEGF-b and PlGF, three clinically validated targets whose expression is shown to lead to the progression of AMD.
  • Along with our collaborators, we have identified novel AAV capsids for delivery to key cell layers within the retina using direct intravitreal injection, a commercially attractive route of administration.
  • The AMD program is our first program in this space and we anticipate being able to build a ddRNAi franchise for other retinal diseases using the same delivery system.


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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