UWA licenses genetic drug tech to Sarepta

The University of Western Australia (UWA) has signed a licensing agreement that could lead to the commercialisation of the first genetic drug originating from the state.

The university has arranged to license technology covering a potential treatment for severe muscle-wasting disease Duchenne muscular dystrophy (DMD) to US-based Sarepta Therapeutics.

Under the deal - negotiated by the university’s Office of Industry and Innovation (OII) - the university will be entitled to upfront and milestone payments of up to $7.1 million, as well as royalties on any product sales resulting from the collaboration.

DMD is the most common muscle-wasting disorder in children, affecting an estimated one in 3500 boys worldwide. It is caused by errors in the dystrophin gene, responsible for producing the dystrophin protein, which plays a role in developing normal skeletal muscle tissue.

Without functional dystrophin, DMD sufferers experience severe, progressive muscle loss. Patients are usually confined to a wheelchair by age 12 and succumb to the disease before reaching 30.

The UWA technology involves causing gene translation machinery to skip the dystrophin mutation, allowing the body to produce a functional but shorter form of dystrophin. It achieves this through a process known as exon-skipping.

Sarepta Therapeutics has four exon-skipping DMD treatment programs in development, characterised by which exons they are designed to skip. Its lead candidate, eteplirsen, is based on technology licensed from UWA in 2008. The new agreement grants Sarepta access to UWA’s entire DMD intellectual property portfolio.

“If eteplirsen becomes a marketplace reality, it will be the first genetic drug based on intellectual property from research developed at UWA and in the state of Western Australia,” OII director Dr Andy Sierakowski said.

Besides DMD, Sarepta is also developing more RNA-based product candidates, including potential treatments for hemorrhagic fever viruses Ebola and Marburg.