A Promising Treatment of WWOX Associated Neurological Disorders

ended

A Promising Treatment of WWOX Associated Neurological Disorders WWOXAID WWOX-related epileptic encephalopathy (WOREE) syndrome, also known as early infantile epileptic encephalopathy, 28 (EIEE28, OMIM: 616211), is a very severe epileptic encephalopathy characterized by absence of language development and acquisition of walking, early-onset drug-resistant seizures, ophthalmological involvement, and a very high likelihood of premature death. Above all, WWOXAID aims to set the basis for bringing into clinical development a treatment for WOREE syndrome affecting children in infancy. Although WOREE syndrome has very limited market prospects, since the number of known patients is low and no current active registry is available, this development is critical because there is no current treatment that could save children with WOREE. In addition to this highly humanitarian purpose, we are confident that this PoC achievement shall be also of importance for broadening the spectrum of applications of this treatment in other WWOX-related disorders. In this PoC, we will achieve several necessary steps including the generation, characterization and delivery of the vectors, securing IPRs, conducting a specific market assessment, targeting regulatory constraints, implementing a business development plan, consulting with professionals in the field and developing contacts with the industry, dedicated organizations and medical doctors.