ADDED
(Exact name of registrant as specified in its charter) ______________________________________________________________________________________________________________________________________________________________ Delaware 82-1080209 State or other jurisdiction of incorporation or organization (I.R.S.
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 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.
Regulatory Precedent and Platform Recognition : With the approval of our first platform product, VYJUVEK, in the United States, EU, and Japan, regulatory precedent for our proprietary HSV-1-based platform has been established, and regulator familiarity with HSV-1 and our platform continues to grow.
Reflecting this increasing familiarity, in October 2025, the FDA granted platform technology designation to our genetically modified, non-replicating HSV-1 viral vector used in our product candidate, KB801 for the treatment of neurotrophic keratitis.
The FDA s platform technology designation program is intended to provide efficiencies in drug development, manufacturing, and review processes for drug product applications that incorporate designated platform technologies.
REMOVED
(Exact name of registrant as specified in its charter) ______________________________________________________________________________________________________________________________________________________________ Delaware 82-1080209 (State or other jurisdiction of incorporation or organization) (I.R.S.
Even though we have obtained FDA approval of VYJUVEK and even if we obtain and maintain approval for our product candidates from the FDA, we may never obtain approval for them outside of the United States, which would limit our market opportunities and adversely affect our business.
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.
Our development pipeline includes multiple clinical stage programs for 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 our FDA approved product and our pipeline candidates throughout the world.
While our focus is on the development of gene therapies to treat patients with severe, life threatening, or rare diseases with high unmet medical needs, we are also evaluating the potential of our platform to address more prevalent and/or non-genetic conditions.
To that end, in April 2019, we incorporated Jeune Aesthetics, Inc.
( Jeune Aesthetics ), a wholly-owned subsidiary, for the purposes of undertaking preclinical and clinical studies for aesthetic skin conditions.
First Approval for Platform Builds on Existing Regulatory Precedent : The first FDA and European Medicines Agency ( EMA )-approved oncolytic virus product, Imlygic by Amgen, for treatment of melanoma, a skin cancer, is based on a genetically engineered HSV-1 virus.
Because this product also employs an HSV-1 backbone for chronic administration, it created a regulatory precedent for approval of an HSV-1-based chronic gene therapy.