The Columbus Foundation, international organisation with the mission of accelerating access to the most effective gene therapies to treat children diagnosed with rare diseases, and Viralgen, leaders in the production of AAV vectors for gene therapy, based at the Gipuzkoa Science and Technology Park, have launched the Objetivo10 programme. The aim is to facilitate and accelerate ten treatments for treating ten rare diseases over the next 10 years, guaranteeing equal access to these treatments for all patients
The Columbus Foundation, together with its sister foundation based in the United States, the Columbus Children’s Foundation, is known for its role in the acceleration of the development of gene therapy programmes which offer hope to children with rare diseases. In addition to promoting its own programmes for rare diseases, the Columbus Foundation has established strategic alliances with organisations such as Viralgen, who provide the necessary experience and access to the capacity to manufacture adeno-associated viral vectors (AAV) at cost. All with the aim of accelerating treatments for rare diseases with a limited commercial therapeutic value.
Gene therapy is a type of treatment which uses genes to treat or prevent diseases. The gene is introduced into a modified virus (AAV) which acts as a vehicle for transporting the gene to the specific organs or tissues. This type of therapy offers new potential and a realistic possibility of curing rare genetic diseases.
Although individually rare diseases have very small populations of patients, together they affect more than 300 million people all over the world, of which 3 million alone are in Spain. And 50% of patients diagnosed with rare diseases are children. The impact of these rare diseases on the patients, families and society runs deep, as many are serious, chronic and progressive, resulting in limited life expectancy. Three of every ten children diagnosed will not live to the age of five. The Columbus Foundation is deeply driven by the urgent need to change these probabilities by accelerating the treatment for these children, to ensure they are not left behind when science could place a cure within their reach.
Objetivo10, announced by the Columbus Foundation and Viralgen, is a revolutionary and ambitious programme which highlights the need to bring together the relevant experience and resources for accelerating the development of promising clinical and research programmes. It seeks to address the development of effective treatments for rare diseases quickly and efficiently. Using a non-profit approach, the Objetivo10 programme will identify and seek to advance in programmes with the best chance of creating successful treatments for rare diseases where effective solutions are not currently available. Objetivo10 seeks to eliminate barriers to manufacturing, considered the most significant obstacle in programmes with small patient populations, for those developments ready to move from the laboratory to the clinic. Objetivo10 will bring together major scientific capacities and the development of a pharmaceutical product to offer treatments to the children most in need.
As a major collaborator and strategic partner of the Columbus Foundation, Viralgen has proven their clear commitment and become a leader in the advancement of programmes for children by offering their knowledge and manufacturing capacity to the rare disease programmes promoted by the Columbus Foundation. The AAV vectors produced by Viralgen for Objetivo10 will be even more affordable and accessible.“We can’t thank Viralgen enough for their exceptionally generous and valuable contribution. This will help us with our mission to accelerate treatments for kids who are battling not only a life-threatening disease, but also time”, commented the Executive Director of the Columbus Foundation, Blanca Garín.
Viralgen’s participation in Objetivo10 is key for the success of the programme. The production of viral vectors is a huge bottleneck in the development of gene therapy projects. The support of Viralgen will make Objetivo10 possible. The association with Viralgen implies savings of several million Euros per programme in comparison to commercial costs, and between 12 and 24 months saved in time. Manufacturing is one of the most difficult steps in the process and working with a leader from the industry will help to close the rare diseases gap for the families of children suffering from these diseases. Javier García, chief executive office of Viralgen remarked, “we are honoured to be a part of the Columbus Foundation’s work, which will bring much hope to many families with children suffering from devastating diseases”.
In addition, the launch of the Objetivo10 programme is accompanied by a campaign on social networks #todossomosraros #weareallstrange, to provide greater visibility to rare diseases and promote their inclusion in society.