PER304

PER304 is an investigational oral enzyme replacement therapy (ERT) being developed by Perseo Pharma for the treatment of Maple Syrup Urine Disease (MSUD). The therapy is built upon Perseo's proprietary nanotechnology platform, which employs a "nano-shield" to protect therapeutic enzymes from the acidic and proteolytic environment of the gastrointestinal tract. This technology is designed to facilitate the binding of the enzymes to the mucus of the small intestine, allowing for localized metabolic activity. By delivering protected enzymes orally, PER304 aims to provide a non-invasive treatment option for MSUD, a rare genetic disorder where the body cannot properly process certain amino acids. The program is currently in the preclinical stage of development.