We are a fully integrated, global, commercial-stage biotechnology company focused on the discovery, development, manufacturing and commercialization of genetic medicines to treat diseases with high unmet medical needs. Using our patented gene therapy technology platform that is based on engineered herpes simplex virus-1 ("HSV-1"), we create vectors that efficiently deliver therapeutic transgenes to cells of interest in multiple organ systems. The cell’s own machinery then transcribes and translates the transgene to treat the disease. Our vectors are amenable to formulation for non-invasive or minimally invasive routes of administration at a healthcare professional’s office or in the patient’s home by a healthcare professional, caregiver, or directly by the patient themselves. Our innovative technology platform is supported by two in-house, commercial scale Current Good Manufacturing Practice ("CGMP") manufacturing facilities.
Our first commercial product, VYJUVEK , is now approved in the United States, the European Union ("EU"), and Japan for the treatment of dystrophic epidermolysis bullosa ("DEB"). We launched VYJUVEK in the United States in 2023 and started launching VYJUVEK in Europe and Japan in 2025.
Our development pipeline includes multiple clinical stage product candidates for the treatment of rare and serious diseases, and we are investing in research and development to advance and grow this pipeline. We possess exclusive rights to develop, manufacture, and commercialize VYJUVEK and our pipeline product candidates throughout the world.
While our focus is on the development of gene therapies to treat patients with rare diseases with high unmet medical needs, we are also evaluating the potential of our platform to address more common severe or life-threatening diseases, such as non-small cell lung cancer ("NSCLC"), as well as aesthetic conditions via our wholly-owned subsidiary Jeune Aesthetics, Inc. ("Jeune Aesthetics"), which we incorporated in April 2019.
Quoted from the business section of this company’s 10-K filed February 17, 2026. Read the filing