There is currently no curative treatment for this rare disease Genetics .

scholarship The European Union has awarded a 3.3 million euro grant to the AIPGENE consortium, led by CIMA and in which Digna Biotech, Clínica Universidad de Navarra, Amsterdam Molecular Therapeutics (AMT), Stockholms Läns Landsting, Deutsches Krebsforschungszentrum and the Madrid Health Service participate, together with Digna Biotech. Specifically, Digna Biotech and CIMA will receive, as members of this consortium, 0.5 and 0.8 million euros, respectively, for the development of a Phase I/II human clinical essay of gene therapy against acute intermittent porphyria. With the support of all AIPGENE partners, Digna Biotech expects to finalize the inclusion of patients with PAI in the clinical essay in the first months of 2012.

By virtue of the co- agreement signeddevelopment , Digna Biotech transfers to AMT all rights for the development and commercialization of the adeno-associated viral vector for the treatment of this disease. In addition, AMT has already obtained Orphan Drug Designation for this vector. 

Disease Genetics rare

Acute intermittent porphyria is a rare disease Genetics in which mutation of the gene encoding the enzyme porphobilinogen deaminase (PBGD) results in insufficient activity of the protein required for the synthesis of group heme. It causes a wide variety of problems, such as acute and severe abdominal pain, as well as psychiatric and neurological symptoms. There is currently no curative treatment for this progressively progressive disease.