ILIT Bio-Ventures, an Israeli start-up company, signed an agreement with the Italian National Research Council (CNR) to develop a drug to treat children with Duchenne Muscular Dystrophy.
ILIT Bio-Ventures, a new start-up company focused on developing innovative technologies and approaches for the treatment of incurable genetic diseases, has signed a research funding and licensing agreement with the Italian National Research Council (CNR) for a unique technology for the genetic treatment of muscular dystrophy on Name Duchenne Duchenne Muscular Dystrophy (DMD).
The technology, developed by Prof. Claudio Passananti and the research team led by him at the CNR laboratories in Rome have already shown the feasibility of restoring muscle function in model animals. The article summarizing the research was recently accepted for publication in the scientific journal - Human Molecular Genetics. 2009 Dec 16 Furthermore, the scientific discovery and its medical application Submitted as new inventions and technologies in applications for patent protection worldwide.
Duchenne muscular dystrophy (DMD) is a severe and debilitating genetic disease characterized by the gradual destruction of muscle tissue. It is one of the most common genetic diseases and is caused by a defect (mutation) in the gene for the production of dystrophin, an essential component of the structural infrastructure in muscle cells. The frequency of the defect in the gene located on the X chromosome is 1 in every 3,500 male births and translates into a worldwide incidence of approximately 200,000 patients, mainly children, for whom there is no hope of a cure. The disease is diagnosed as a lack of muscle function at the age of 1-2 years, which manifests itself and develops into a loss of motor function and a disability requiring a wheelchair at the age of 8-9 years. Ultimately, the disease leads to death from respiratory or cardiac failure at a young age - on average 26 years.
The therapeutic technology that will be developed by Ilit is based on the use of a unique synthetic gene, which was planned and designed in a rational design (Rational Design) and is called Jazz. Transplantation of this gene will allow increased production of another protein called utrophin, which is not defective and identical in its action to dystrophin, which is missing in these patients, thus allowing a natural alternative that functions perfectly and restores its function to the muscle tissue.
"We have a commitment to finding treatments and potential cures for this devastating disease and we are ready and willing to advance this ambitious program." Says Avraham Landa, co-founder of the company Elite BioVentures, and added that "by linking and activating a network of international experts in gene therapy together with leading doctors and researchers in muscular dystrophy, we hope to leverage the scientific excellence of the team of researchers from Italy for research Clinical features in Duchenne muscular dystrophy patients. ".
Elite BioVentures is in the process of presenting the venture to groups of investors for the purpose of financing and developing this technology and other technologies in the field - everything is designed to improve the lives of patients and their families in all existing muscular dystrophy diseases.