Details of our out-licensed programs

Several biotechnology companies have licensed Benitec's ddRNAi technology for use in their own therapeutic programs for human diseases.

1.HIV/AIDS with Calimmune


In March 2012, Benitec granted a non-exclusive licence to US-based biotech company, Calimmune, to use ddRNAi for treating HIV/AIDS. The agreement covers use of ddRNAi to target up to three key viral and cellular genes, which have been identified as significant therapeutic targets able to inhibit HIV/AIDS infection.

Calimmune’s approach to treating HIV/AIDS is highly innovative, and developed with core technology from the lab of Dr David Baltimore, a Nobel Laureate in the area of HIV/AIDS. The approach involves extraction of T cells from HIV patients, silencing of the gene that codes for the CCR5 receptor protein associated with low HIV loads, and re-injecting the modified cells back into patients, thereby conferring resistance to HIV. Calimmume's HIV/AIDS treatment is known as Cal-1.


Calimmune has commenced a Phase l/ll clinical trial of Cal-1 with current status:

  • The first cohort of four HIV-positive participants has been infused with their own T cells and hematopoietic stem cells, modified to silence the HIV receptor CCR5
  • Review of trial data by the DSMB confirmed that none of the patients had experienced serious adverse affects
  • The FDA has approved the next cohort dosing of three patients, who will also receive a preconditioning regime designed to make the treatment more effective.
  • Data from this study are expected in 2015.

2. RETINITIS PIGMENTOSA with Genable Technologies


Rhodopsin-linked autosomal dominant retinitis pigmentosa (RHO-adRP) is an orphan disease which affects around 1 in 30,000 people. There are around 19,000 patients in the European Union and there is no current treatment.Sufferers experience severe visual dysfunction due to the death of rod and cone photoreceptor cells. The disease is caused by any of one of over one hundred inherited defects in the rhodopsin (RHO) gene, which has made a single treatment very difficult until now.

Genable Technologies Limited (Genable) is a privately held, venture-backed bio-pharmaceutical company based in Dublin, Ireland. It is developing new gene medicines to treat 'dominant' genetic diseases based on the pioneering work of Professor Jane Farrar, Dr Paul Kenna & Professor Peter Humphries. 


In July 2012, Benitec granted Genable an exclusive worldwide agreement to license ddRNAi for development of a single treatment for patients with RHO-adRP, regardless of which RHO mutation(s) causes the disease. The treatment involves suppression of the mutant and normal genes, and replacement with a normal RHO gene that has been modified to be resistant to ddRNAi gene silencing. The program has established proof of concept studies in an in vivo model of the disease, which was reported at the World Gene Therapy Congress in May 2012 in London.

Genable utilises well-established, clinically safe and effective AAV vectors to cause expression  of  RNA interference (RNAi) molecules that suppress expression of both faulty and normal gene copies, and replaces them with a gene that becomes refractory to  suppression but still encodes a  normal wild type protein. The combination of suppression and replacement (S&R) overcomes the significant hurdle of mutation variability, by eliminating the need to target specific mutations. Genable’s technology is protected by a broad suite of granted patents and patent applications in the USA, EU and worldwide.

In April, 2013 the US Food & Drug Administration (FDA) granted Genable orphan drug designation for GT308, its gene therapy product. GT308 is a two-part therapy: the first is suppression by ddRNAi of the defective rhodopsin gene (RHO) which is responsible for the loss of vision, and the second is replacement of the defective RHO with a healthy form of the gene. Benitec is using a similar “suppression and replacement” strategy in its oculopharyngeal muscular dystrophy (OPMD) program, in collaboration with the Royal Holloway, University of London. 



Huntington's Disease (or HD, formerly known as Huntington's Chorea) is a neuro-degenerative disease which affects muscle coordination and leads to cognitive and behavioural decline. It is associated with involuntary writhing movements (known as chorea), which usually become apparent in sufferers between 35 and 44 years of age.  

The disease affects both men and women, and is more common in people from Western European than those from Asia or Africa. HD is caused by an autosomal dominant mutation in either of an individual's two copies the 'Huntington gene', so offspring of affected persons are expected to have a 50% chance of inheriting the disease. 


In December 2012, Benitec granted a non-exclusive sub-licensing agreement with Amsterdam-based uniQure BV (uniQure) to develop a treatment for Huntington’s Disease using Benitec's ddRNAi gene silencing technology. 

Benitec’s licence to uniQure covers the application of ddRNAi to target, and thereby silence, key genes identified as significant therapeutic targets in the treatment of HD. Under the agreement, uniQure has an option to convert the non-exclusive licence to exclusive pending achievement of certain pre-clinical milestones, and also to acquire additional licences to Benitec’s ddRNAi technology for other diseases. 

At the same time, uniQure granted Benitec a non-exclusive licence to use its AAV 5 (based on Adeno-Associated Viral vectors) technology to assist Benitec’s development of treatment for Hepatitis B.

5. CANCER VACCINES with Regen Biopharma


In August 2013, Benitec granted a license to US based Regen BioPharma Inc. (Regen), a subsidiary of the Bio-Matrix Scientific Group, to use ddRNAi technology for the development of cancer vaccines. 

Benitec's licence to Regen covers the application of ddRNAi to silence indoleamine 2,3 - dioxygenase (IDO) in dendritic cells. IDO is associated with immune-suppression and is over-expressed in cancer. Regen has produced pre-clinical evidence that modification of these cells using ddRNAi targeting the silencing of IDO should significantly enhance their efficacy as anti-cancer vaccine agents.

6. INTRACTABLE PAIN with Circuit Therapeutics


In November 2014, Benitiec granted an exclusive licence to US-based biotech company, Circuit Therapeutics (Circuit), for the use of ddRNAi to target intractable pain. This is severe, constant pain which has not responded to current therapies. If untreated, intractable pain can lead to death.  

Circuit plans to use ddRNAi to block Nav1.7, a sodium channel produced in certain sensory nerves, which is is critical for the generation of pain. By blocking Nav1.7, Circuit plans to control pain without the side-effects of less specific therapies.