Grifols reinforces its commitment to research in the 7th edition of the Martín Villar Awards for Research on Hemostasis


24.09.2014
 
  • Created in 2007, the awards recognize progress made in the field of Hemostasis. The awards are endowed with 20,000 euros for each of the two prizes: basic research and clinical research.
  • For the first time, the awards ceremony was held during the congress of the National Hemophilia Foundation (NHF) in Washington DC, United States.
  • The Basic Research prize was awarded to Dr. Federico Mingozzi for demonstrating a novel and elegant technique in non-human animal models to overcome the inhibitory effects of neutralizing antibodies (NAbs) that block gene delivery when adeno-associated viruses (AAV) are used as vectors for gene therapy.
  • The Clinical Research prize was awarded to Dr. Yohann Repessé and Dr. Ivan Peyron who have identified for the first time polymorphisms in the HMOX1 promoter that are associated with the development of factor VIII (FVIII) inhibitors in hemophilia A patients.

 

Washington DC, September 18, 2014 - For the seventh consecutive year, Grifols awards the Martín Villar Prizes for Research on Hemostasis, with which the company promotes and reinforces its commitment to scientific research. Since the year 2007, these International prizes have recognized the work of scientists and researchers that conduct innovative studies in the field of blood coagulation disorders. The award also honors and pays tribute to Dr. José Martín Villar, a medical doctor and surgeon from Valencia who passed away in 2011. Dr. Martín Villar devoted much of his career to the advancement of knowledge of hematology and, especially, of hemophilia.

The awards ceremony for this seventh edition took place during the congress of the National Hemophilia Foundation (NHF) in Washington DC, United States.  A jury composed of internationally recognized experts in the field of Hemostasis  awarded prizes for the original contributions developed by Dr. Federico Mingozzi during his time at the Children's Hospital of Philadelphia, USA, and by Dr. Yohann Repessé and Dr. Ivan Peyron during their stay at the INSERM in Paris, France.

 

About the award-winning research

Basic Research Prize

The publication "Overcoming Preexisting Humoral Immunity to AAV Using Capsid Decoys" by Mingozzi F, Anguela XM, Pavani G, Chen Y, Davidson RJ, Hui DJ, Yazicioglu M, Elkouby L, Hinderer CJ, Faella A, Howard C, Tai A, Podsakoff GM, Zhou S, Basner-Tschakarjan E, Wright JF, High KA. (Department of Pediatrics and Center for Cellular and Molecular Therapeutics, Children's Hospital of Philadelphia, Philadelphia, PA, USA).

The authors describe a novel and elegant technique in non-human animal models to overcome the inhibitory effects of neutralizing antibodies (NAbs) that block gene delivery when adeno-associated viruses (AAV) are used as vectors for gene therapy. The prevalence of NAbs to AAV in humans is 30-60% and constitutes a major complication. In their study, the authors demonstrate that by introducing empty viral capsids to serve as a decoy for antibody binding along with the gene therapy vector, NAbs to AAV are absorbed. In addition, the authors improved the safety of this procedure by mutating the empty capsids to prevent binding to target cells.

This approach could allow successful human gene therapy with this vector for inherited and metabolic diseases, even in the presence of preexisting NAbs.

 

Clinical Research Prize

The publication "Development of inhibitory antibodies to therapeutic factor VIII in severe hemophilia A is associated with microsatellite polymorphisms in the HMOX1 promoter" by Repesse Y, Peyron I, Dimitrov JD, Dasgupta S, Farrokhi Moshai E, Costa C, Borel-Derlon A, Guillet B, D'Oiron R, Aouba A, Rothschild C, Oldenburg J, Pavlova A, Kaveri SV, Lacroix-Desmazes S on behalf of the ABIRISK consortium. (INSERM, UMR S 872, Centre de Recherche des Cordeliers, Paris, France; Université Pierre et Marie Curie- Paris6, UMR S 872, Centre de Recherche des Cordeliers, Paris, France; Université Paris Descartes, UMR S 872, Centre de Recherche des Cordeliers, Paris, France).

The authors have identified for the first time polymorphisms in the HMOX1 promoter that are associated with the development of factor VIII (FVIII) inhibitors in hemophilia A patients. Development of inhibitors against FVIII occurs with a prevalence of 30% and represents the major complication of the disease as inhibitors block the activity of FVIII and make the replacement therapy with FVIII concentrates ineffective. In their study, the authors identified 6 genotype repeats of which the genotype group including L alleles was found more frequent among inhibitor patients than in non-inhibitor patients.

This important finding represents a step forward towards the goal of reducing the risk for inhibitor development in hemophilia patients.

 

Jury Members

  • Dr. Manuel Campos, Director of Clinical Haematology at the Hospital Geral de St. António, Porto, Portugal.
  • Dr. Elbio D'Amico, Hospital das Clínicas da Faculdade de Medicina da Universidade de São Paulo, São Paulo, Brazil
  • Dr. Augusto Federici, Associate Professor of Hematology, Department of Internal Medicine, University of Milan, Milan, Italy
  • Dr. Craig Kessler, Professor of Medicine and Pathology, Georgetown University Medical Center, Washington DC, US
  • Dr. Guy Young, Director of Hemostasis and Thrombosis Center , Children's Hospital Los Angeles, Los Angeles, CA, US
  • Dr. Johannes Oldenburg, Chairman and Director of the Institute of Experimental Haematology and Transfusion Medicine, University Clinic Bonn, Bonn, Germany
  • Dr. Victor Jiménez Yuste, Head of the Thrombosis and Haemostasis Unit at the University Hospital La Paz, Madrid, Spain
  • Dr. Eva Bastida, Corporate VP of Scientific & Medical Affairs at Grifols, S.A. acts as Secretary of the panel but is not a voting member

 

About Grifols

Grifols is a global healthcare company with a 70-year legacy of improving people's health and well being through the development of life-saving plasma medicines, diagnostics systems, and hospital pharmacy products.

The company is present in more than 100 countries worldwide and is headquartered in Barcelona, Spain. Grifols is a leader in plasma collection with a network of 150 plasma donor centers in the U.S., and a leading producer of plasma-derived biological medicines. The company also provides a comprehensive range of transfusion medicine, hemostasis, and immunoassay solutions for clinical laboratories, blood banks and transfusion centers, and is a recognized leader in transfusion medicine.