A European media agency broadcasts a report on the research in acute intermittent porphyria of the CIMA
Antonio Fontanellas, Jesús Prieto and Gloria González Aseguinolaza presented the preliminary results of the first gene therapy essay for this rare metabolic disease.
The European media agency youris.com has made a report on the research in acute intermittent porphyria of the research center Médica Aplicada (CIMA ) of the University of Navarra. The report has been uploaded on the youris.com platform.
Acute intermittent porphyria is a rare metabolic disease affecting approximately 5 per 100,000 population. It manifests as acute crises, with abdominal pain, nausea, vomiting, constipation and tachycardia. There is currently no treatment for this disease, which can sometimes be fatal due to severe neurological complications.
The CIMA has developed the first gene therapy product C in the European Union, which is being evaluated in a Phase I clinical trial essay at the Clínica Universidad de Navarra and at the Hospital 12 de Octubre in Madrid. The study is part of the European consortium AIPGENE, which also includes the Dutch business uniQure and research centers in Germany and Sweden.
"Early results tell us that the treatment is safe. Now we have to analyze all the data to determine its efficacy and make decisions on the next steps of the development of this novel therapy. If all goes well, the treatment could reach patients in 2020," explains Dr. Gloria González Aseguinolaza, director of the Gene Therapy and Regulation of Gene Expression Program at CIMA.
youris.com is a non-profit media agency that promotes cutting-edge European innovation through television and web media. It designs multimedia communication strategies for large organizations from research and EU-funded projects.