Protein substitution therapy for neurodevelopmental diseases.

Scientists at the Georg-August-Universität Göttingen Stiftung Öffentlichen Rechts, Universitätsmedizin, developed a modified MeCP2 protein. This biotechnologically produced protein, which might be used for a substitution therapy of the RETT-Syndrome, crosses the blood-brain-barrier. Thus it reaches the brain as the therapeutical target. First preliminary results could show the success of this strategy in a mice model. A patent application for the novel protein and its uses has been applied for. The corresponding IP rights of the University of Göttingen have been successfully and exclusively licensed to the Swiss biotechnological company IPT Pharma AG, Zürich. The University receives financial payments, which will benefit the scientists as well as their research departments. The agreement has been negotiated by the MBM ScienceBridge GmbH, the technology transfer organization of the Georg-August-Universität Göttingen. The RETT-syndrome is the second most common cause of mental retardation. At least 90% of the cases are due to mutations in a specific gene. The consequence is an inability to produce enough functional MeCP2 protein and the subsequent abnormal brain development is the cause for the RETT-syndrome. The cause and course of this disease including brain atrophy and progressive dementia is yet not fully understood. Clinical manifestations include decelerating head growth, emotional withdrawal, loss of learned skills, truncal and gait ataxia/apraxia, stereotypical hand movements, dementia, and motor deterioration. Young girls show first symptoms after the 18. month. The course of the disease is progressive and results in a lifelong disability and in need of care. Alone in Europe and in the USA there are aprox. 1000 newborns yearly having RETT-syndrome. No successful treatment is yet known for patients suffering the RETT-syndrome. In the last years amazing therapeutical success could be achieved through novel protein therapies for a few genetical diseases. Hence with a biotechnologically produced modified MeCP2 protein a therapeutical or preventive treatment for neurodevelopmental diseases could be tested in coming years, particularly for the RETT-syndrome. Further diseases might include attention deficit disorder (ADD) or the Angelman-syndrome. Due to its expert knowledge with recombinant proteins and development of biopharmaceuticals the licensee IPT Pharma AG selected PharmedArtis GmbH from Aachen as its technological partner. Moreover, the PharmedArtis will provide IPT Pharma its experience developing novel immunotherapeutics for the treatment of cancer and inflammatory diseases, including support with regulatory affairs. The development, GMP-production, realization of the pre-clinical phase and the prearrangements for clinical phases has been assigned to the PharmedArtis. First relevant pre-clinical results are expected for end of 2009. A certified GMP-development of the protein is aimed for the first quarter of 2010. The Universitätsmedizin of the Georg-August-Universität Göttingen includes the school of medicine and its hospital. It is one of the leading educational and research centers for medical doctors in Germany and in Europe and one of the universities in Germany awarded with the title “University of Excellence” www.med.uni-goettingen.de MBM ScienceBridge GmbH is a fully owned subsidiary of the University of Göttingen and is the technology transfer organization for several German Universities and Research facilities. The IP portfolio managed by MBM ScienceBridge covers innovation in the areas of Life Sciences, Chemistry, Physics, Agriculture and Medical Technologies. www.sciencebridge.de / info@sciencebridge.de / +49-(0)551-30724151 The IPT Pharma AG which is located in Zürich/Switzerland is a recently founded biotechnological company. IPT Pharma develops innovative therapies based on recombinant proteins for the therapy, prevention and diagnosis of neurodegenerative and neurodevelopmental diseases. The leading project of IPT Pharma focuses on the development of a protein substitution therapy for the treatment of RETT-syndrom, with the aim of a registration approval. www.ipt-pharma.ch / mail@ipt-pharma.ch PharmedArtis GmbH is a biotechnology company founded in 2004 by experienced scientists of the Fraunhofer Institute for Molecular Biology and Applied Ecology (IME) in Aachen, Germany. Being a specialist in recombinant technologies and Protein Production PharmedArtis is a one-stop-shop offering a wide range of services from gene to product including state-of-the-art GMP facilities. Its knowledge includes production systems in E. coli, yeasts, fungi, mammal cells and plant cells. Besides these services PharmedArtis develops based on four patent families novel immunotherapeutics for the treatment of cancer, inflammatory diseases and autoimmune diseases. www.pharmedartis.de / mail@pharmedartis.de Ihre Ansprechpartnerin: Barbara Fink, M.A. , Öffentlichkeitsarbeit, MBM ScienceBridge GmbH, Hans-Adolf-Krebs-Weg 1, 37077 Göttingen, info@sciencebridge.de Tel.: (0551) 30 724 151 Fax: (0551) 30 724 155