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Vybion Receives Orphan Drug Designation for INT41 in Huntington's Disease

Vybion Inc., a leading Gene Therapy Company developing Intrabodies to treat serious and rare diseases, today announced that the United States Food and Drug Administration (FDA) has granted orphan drug designation for INT41, the Company’s Intrabody delivered with AAV for the treatment of Huntington’s disease.

“We are pleased to receive orphan drug designation for INT41, which has shown the potential to address an area of high unmet medical need,” said Lee Henderson PhD, CEO of Vybion. “We believe that INT41 has great potential in treating HD and are actively advancing INT41 to the clinic.”

Orphan designation is granted by the FDA Office of Orphan Products Development to advance the evaluation and development of safe and effective therapies for the treatment of rare diseases or conditions affecting fewer than 200,000 people in the U.S. Under the Orphan Drug Act, the FDA may provide grant funding toward clinical trial costs, tax advantages, FDA user-fee benefits, and seven years of market exclusivity in the United States following marketing approval by the FDA. The granting of an orphan designation request does not alter the standard regulatory requirements and process for obtaining marketing approval. For more information about orphan designation, please visit the FDA website at

INT41 will be advancing through the final stages of IND enabling studies in 2019 and 2020 before an Investigational New Drug (IND) is filed to seek approval to move forward with human clinical trials.

About INT41

Intrabodies are small antibody formats that can function within the cell when delivered by appropriate means such as the use of a recombinant adenoassociated virus (AAV). Certain AAV serotypes are neurotropic and provide one approach to delivery that has been used in humans for multiple neurologic diseases. Both animal model and mechanism of action work have been successfully completed for INT41 and Vybion has developed IND enabling study plans following a meeting with the FDA. INT41 also has potential for treating several other diseases within the polyglutamine expansion family of rare diseases.

About Huntington’s Disease (HD)

Huntington’s disease (HD) is a uniformly fatal monogenic neurodegenerative disease characterized by progressive decline in both motor and cognitive function, typically over a 15-20 year period from the time of diagnosis. HD is caused by a successive CAG repeat (encoding the positively charged amino acid glutamine) in the 5’ region of the huntingtin gene. Repeat lengths of greater than 36 CAGs result in clinical adult symptoms and greater than 70 repeats defines juvenile HD. Most patients are diagnosed as adults in their 30s and 40s while about 6% are diagnosed as juveniles. In the US, approximately 150,000 individuals have the genetic mutation and 30,000 are symptomatic.

About Vybion

Vybion is a development stage private company operating virtually by contracting work to laboratories that provide the best expertise for filling preclinical and/or clinical research needs. The Company is focused on neurodegenerative disease using a gene therapy approach for delivery of Intrabody genes subsequently made by the cell to which the gene is delivered.

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Last Updated: 03-Dec-2018