The Quoin team is relentlessly focused on developing and commercializing innovative treatments for rare and orphan diseases, for which none exists today. Our pipeline comprises four unique products that collectively have the potential to target a broad number of indications, including Netherton Syndrome, Scleroderma, Peeling Skin Syndrome, Palmoplantar Keratoderma, Epidermolysis Bullosa and others. We are committed to continuously expanding our pipeline of products to target additional rare and orphan diseases.
Other Target Indications
Peeling Skin Syndrome
Peeling Skin Syndrome (PSS) is a rare, genetic skin disorder characterized by painless, continual skin peeling due to a separation of the stratum corneum from the underlying layers of the Epidermis. PSS may be generalized, affecting skin over the patient’s entire body, or localized, affecting only the skin of the hands and feet. Generalized PSS can be categorized as non-inflammatory, and less severe, or inflammatory, which is typically more severe and involves other organ systems. There is currently no approved treatment for PSS, and patients manage symptoms using over-the-counter emollients.
SAM Syndrome, also known as severe skin dermatitis, multiple allergies and metabolic wasting, is a life-threatening rare disease caused by mutations in the desmoglein 1 (DSG1) and desmoplakin (DSP) genes. A compromised skin barrier results in a predisposition to allergies, and other symptoms include severe pruritus, palmoplantar keratoderma, pustular psoriasis and excessive sweating. Quoin is pursuing the development of QRX003 as a potential treatment for SAM Syndrome.
Palmoplantar Keratoderma (PPK) is a group of rare skin disorders with symptoms including thickened skin on the palms of the hands and the soles of the feet, fragile and blistering skin, and excessive perspiration. In its rarer forms, PPK can affect organs other than the skin. Genetic PPKs result in keratin abnormalities in patients.
Scleroderma is a rare, autoimmune disease that affects connective tissue of the skin, blood vessels, internal organs and digestive tract. Symptoms of scleroderma include thickening and tightening of the skin, and inflammation that leads to problems in other organs including the lungs, heart and kidneys. There is currently no approved treatment or cure for scleroderma.
Epidermolysis Bullosa (EB) is a group of rare and genetic skin disorders in which the skin is so fragile, even minor trauma or friction can have devastating results, causing severe pain, blistering, scarring, infections, chronic wounds and immobility. In severe cases, the cost of bandaging alone for EB patients can exceed $10,000 per month. One format of the disease, Recessive Dystrophic EB (RDEB) which is caused by the mutation of the COL7A1 gene, is marked by devastating, progressive, painful blistering. RDEB is diagnosed at infancy and accompanied by a high mortality rate – 76% of patients do not live beyond their 30s.
Genetic and Rare Diseases (GARD) Information Center
GARD is a program of the National Institutes of Health (NIH) that provides free access to reliable, easy to understand information about genetic and rare diseases.