Treatment of Genetic Skin Disorders with [XX] Inhibitors
Section: For Industry
Category(ies): Other
Reference #: ALDO439501
OTC Contact: Tracy Bruehs (Directory Information | Send a Message)
Description
Skin diseases affect millions of people world wide. Thus far, about 170 skin conditions have been identified as having a monogenetic (skin disorders inhabiting a single gene) basis. Traditional methods of treating genetic skin disorders have included steroids, antibiotics and retinoids. These treatments have many side-effects and limited success. Scientists at Georgetown University have uncovered the benefits of using specific inhibitors to treating monogenetic keratinization skin disorders, including Darier’s Disease, Hailey-Hailey Disease, Tay’s syndrome, eurythrodemic and nonerythrodermic autosomal recessive lamellar ichthyosis, Netherton syndrome, ichthyosis vulgaris, keratosis pillaris, bullous ichthyiosiform erythroderms, Harlequin ichthyosis, and Sjogreen Larsson syndrome, among many, many other monogenic skin disorders identified in this patent.
Applications
The inhibitors identified in the patent treat autosomal dominant or autosomal recessive monogenetic keratinization disorders characterized by hyperkeratosis, keratinocyte hyperplasia or ichthyosis. Also developed are delivery systems for a variety of the identified inhibitors that would inhibit the signal transduction pathway triggered at any point along an identified pathway.
Advantages
- Limited side effects compared to steroids, antibiotics and retinoids (such as Accutane® or Soriatane®), which may result in burning, red, scaling skin and potential bone toxicity with long term use.
- The identified inhibitors in this patent have been shown to be well tolerated; already FDA approved for other indications; used in human populations with little side effects; is less toxic than alternatives; and has more dramatic reduction in skin lesions.
- May be administered to people with known genetically mutated alleles prior to onset of symptoms, to prevent manifestation of disease.
Stage of Development
Research and development stage for skin disorders, but shown effective in a patient being treated for another disorder; already FDA approved and used in general population for other indications.
Inventor: Doru Alexandrescu, MD, Georgetown University
Relevant Publications
No resources available.
Patent Status
Provisional application, 61/022,067 filed January 18, 2008.
