Roche and Isis Pharmaceuticals have formed a potential $392 million alliance to develop antisense drugs for Huntington’s disease, a fatal, neurodegenerative disease.

The alliance, which provides an upfront payment to Isis of $30 million, will combine Isis’ antisense oligonucleotide technology with Roche’s expertise in CNS diseases and its brain shuttle technology, which Isis says can lead to the development of antisense drugs that can be delivered systemically and cross the blood-brain barrier.

Isis will take responsibility for development of drugs through the first phase 1 study and Roche will have the option at that point to license and continue development of drugs. If Roche exercises its option, it will be responsible for global development, regulatory and commercialization activities for all drugs arising out of the collaboration.

“We believe our mature antisense drug discovery platform is a perfect fit for Roche’s neuroscience franchise, and we anticipate a fruitful collaboration to advance our pre-clinical compounds,” says Frank Bennett, senior vice president of Research at Isis.

Huntington’s disease is a fatal, inherited genetic brain disorder that results in the progressive loss of both mental and physical capabilities. Symptoms usually appear between the ages of 30 to 50, and worsen over a 10 to 25 year period. Presently, there is no effective treatment or cure for the disease, and current treatments focus on reducing the severity of some disease symptoms.

In people with the disease, there is a copy of both the normal gene for producing huntingtin protein and an abnormal version of the gene that produces the disease causing form of the huntingtin protein. Isis has shown the ability in animal models to inhibit either production of all forms of the protein or just the abnormal form. The company says there is some debate whether inhibiting just the abnormal form of the protein or all forms of the protein will be the most effective means of treating the disease and said it is pursuing both options.