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First acute intermittent porphyria patient treated with a gene therapy product


Digna Biotech sets the first clinical trial in Spain to treat acute intermittent porphyria, a rare genetic disease that affects the biosynthesis of heme (a component of hemoglobin) and can cause significant neurological damage. This biotechnology company coordinates a phase I clinical trial in collaboration with CIMA, the Clínica Universidad de Navarra and the Hospital 12 de Octubre in Madrid. The study is funded in part by the European Comission´s Seven framework program under the aegis of the European consortiumAIPGENE, which also involve the Dutch company uniQure and research centers from Germany and Sweden.

The primary objective of this study is to evaluate the safety of the gene therapy product, rAAV2/5-PBGD vector, in patients with severe AIP. Secondary objective includes the evaluation of the efficacy. The trial involved doctors Jesús Prieto, Delia D'Avola and Bruno Sangro from the Clínica Universidad de Navarra and Dr. Rafael Enriquez de Salamanca from the Hospital 12 de Octubre. This first phase will last one year and eight patients will be enrolled who will receive a dose escalation of the therapeutic vector to check tolerability. If the results are positive, Phase II will begin in a larger number of patients. It is estimated that the therapy could be available within five years, approximately.

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