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 way in non-human animal models to overcome the inhibitory effects of neutralizing antibodies (NAbs) that block gene delivery when adeno-associated virus (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 in humans successful 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 en nombre de 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 making 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 approach of reducing the risk for inhibitor development in hemophilia patients.
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.
In 2013, sales exceeded 2,740 million euros with a headcount of 13,200 employees. Grifols demonstrates its commitment to advancing healthcare by allocating a significant portion of its annual income to R&D.
The company's class A shares are listed on the Spanish Stock Exchange, where they are part of the Ibex-35 (MCE:GRF). Its non-voting class B shares are listed on the Mercado Continuo (MCE:GRF.P) and on the U.S. NASDAQ via ADRs (NASDAQ: GRFS). For more information visit www.grifols.com