Gene therapy, CIMA, University of Navarra, AIPGENE, Samulski, Jesus Prieto, Gloria Gonzalez Aseguinolaza

“New clinical trials in gene therapy show the beginning of a new form of molecular medicine”, said Prof. Jude Samulski, director of the Gene Therapy Center of the University of North Carolina (USA), during the International Symposium “Liver directed gene therapy for rare diseases”, celebrated at CIMA of the University of Navarra (Spain).

Prof. Samulski isolated and characterized during his PhD one of the most promising virus in the field of gene therapy, the adeno-associated virus. Since them Prof. Samulski’s group has focused his work in the characterization of the vector, generation of new tools, manipulation of the vector and clinical applications. The main contributions in the science community that facilitated the new field of gene therapy are the sequencing of human genome (that allowed the identification of new gene´s associated with specific disease), specific development of "animal models" that mimic human diseases, and engineering of human viruses as gene transfer systems.

Prof. Samulski explained that the most important advance in gene therapy is the developing of more efficient gene delivery systems. “Ability to harness efficient and accurate gene transfer that is safe to the patient and does not pose a risk to general population has triggered a landslide of new clinical trials that are beginning to show the promise of this new form of molecular medicine. We are observing patients clinically benefiting after experimental gene transfer in many indications such as Factor IX for Hemophilia, Leber Congential Amaurosis -LCA for ocular blindness, Wiscott A syndrome, Adrenolekudystrophy, to name a few”.

Quality of gene therapy research and need of funding

During his visit to the CIMA of the University of Navarra,
said that it is remarkable to see how fast things are moving forward based on the research being carried out in various spanish institutes. “The research teams located in Pamplona (Dr. Jesús Prieto and Dr. Gloria González-Aseguinolaza, to name a few) whom have position themselves to be leaders in gene transfer for liver disorders such as Wilson’s disease, acute intermittent porphyria, and cirrhosis of the liver. If you look beyond the critical mass of investigators located in Pamplona you will find the highest quality of work being carried out on Mucopolysaccharide disorders (Dr. Fátima Bosch, Universidad Autónoma de Barcelona), and inborn errors of metabolism, namely hyperoxaluria (Dr. Eduardo Salido, Universidad de La Laguna -Tenerife), to name a few”.

According to Prof. Samulski, “all of these ground breaking therapeutic initiatives are only possible with strong commitments from the funding communities. In the US, even during hard economic times, it has been important to keep this research engine running at full speed. This area is becoming extremely competitive now that early days of success are on the horizon, and many countries are lining up to exploit what will be the next wave of medical breakthroughs that impacts life long illness associated with genetic disorders.
For me it is a privilege to be part of this amazing journey and similar to the pioneering spirit of the early Spanish explores, to share in discovering new cures to these crippling genetic diseases with the talented colleagues from Spain makes the "hard work" all the more enjoyable”.

AIPGene Consortium

The International Symposium “Liver directed gene therapy for rare diseases”, celebrated at CIMA of the University of Navarra (Spain), was organized by the partners of the European consortium AIPGENE.

AIPGENE is an EU-funded collaborative consortium composed of different groups involved in the preclinical and clinical program for the selection of the best gene therapy vector with a therapeutic application on the acute intermittent porphyria: CIMA (Pamplona); DIGNA Biotech (Madrid); UniQure (Amsterdam); Clinica Universidad Navarra (Pamplona); Hospital 12 Octubre (Madrid); Karolinska Institutet (Stockholm) and Nationales Centrum für Tumorerkrankungen (NCT) (Heidelberg).