INT41 Gene Therapy for Neurodegenerative Diseases Granted Priority Patent Status


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The patent application for gene therapy candidate INT41, developed by Vybion for the treatment of Huntington’s disease, spinal muscular atrophy (SMA), and other neurodegenerative diseases, was granted Track One status from the U.S. Patent and Trademark Office (USPTO).

The patent application covers the treatment’s composition and methods of use. The Track One designation gives the application priority review, allowing it to get a final decision within about a year. Track One has fewer requirements and doesn’t need a pre-examination search for the application.

“Vybion is pleased to receive Track 1 status on INT41 and anticipates rapid review and allowance,” Vybion CEO Lee Henderson said in a press release.

INT41 gene therapy was specifically designed to target neurologic diseases caused by polyQ expansions, resulting in mutant proteins that accumulate in motor neurons and subsequently lead to cellular degeneration. The gene therapy aims to detect and target the “toxic” sequence, promoting its degradation and thereby preventing its accumulation in neuronal cells.

INT41 belongs to a class of compounds called intrabodies, which are small fragments of antibodies that are functional inside the cells, contrary to “normal” antibodies, which recognize immune response-inducing molecules (antigens) on the surface of cells.

Intrabodies have the potential to contribute to the development of new targeted therapies with the specificity and safety of monoclonal antibodies, one of the most efficient and successful classes of drugs. This new gene therapy may be an opportunity to develop therapies against so-called “hard targets” inside cells that other types of treatments have been unable to reach.

The use of INT41 to treat neurodegenerative diseases was primarily tested on Huntington’s disease animal models. Findings of 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,” were published in the Journal of Neurodegenerative Diseases.

INT41 is considered a potential therapeutic strategy for other neurodegenerative diseases with similar biological features, such as spinal cerebellar ataxia, Alzheimer’s disease, and spinal and bulbar muscular atrophy (SBMA). However, the treatment has so far been more fully developed for Huntington’s, according to Vybion’s drug pipeline, and more studies are required to validate its effectiveness and safety for it and other neurodegenerative conditions.