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 www.fda.gov.

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.