Potential Huntington’s Disease Gene Therapy Granted Expedited Patent Review

Ana de Barros, PhD avatar

by Ana de Barros, PhD |

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A patent application for Vybion’s potential gene therapy for Huntington’s disease (HD) and other neurodegenerative conditions has been granted Track One status by the U.S. Patent and Trademark Office (USPTO).

Patent applications are ordinarily reviewed in the order of their filing date, with the process taking about three years. The USPTO’s goal with Track One is to provide a final disposition within twelve months of the prioritized status being granted.

The patent for INT41 would cover methods of use and the treatment’s composition.

INT41 is in a new class of drugs called intrabodies, which are antibodies that bind to proteins within cells, Vybion said in a press release.

“Vybion is pleased to receive Track I status on INT41 and anticipates rapid review and allowance,” Lee Henderson, Vybion CEO, said in the release.

The company published a study on the treatment in August in the Journal of Neurodegenerative diseases. The study, “An Intrabody Drug (rAAV6-INT41) Reduces the Binding of N-Terminal Huntingtin Fragment(s) to DNA to Basal Levels in PC12 Cells and Delays Cognitive Loss in the R6/2 Animal Model,” showed that INT41 was able to block gene dysregulation, delaying the loss of cognitive and motor function in an animal model of HD.

Huntington’s disease is caused by mutations in the HTT gene, which provides instructions for making a protein called huntingtin. That protein plays an important role in nerve cells (neurons) in the brain. INT41 works by accelerating huntingtin degradation, stabilizing the N-terminal “toxic” fragment that is produced during the process, and reducing protein aggregation and gene dysregulation — both of which are pivotal in Huntington’s disease.

“Intrabodies may provide opportunities to develop successful therapies against what are considered ‘hard targets’ within the cell,” the company said in the release.