February 25, 2021

Organizations unite to develop effective gene therapy treatments by taking aim at accelerating treatments for ten rare diseases

San Sebastian, Spain and Raleigh, NC (February 26, 2021) – Fundación Columbus, an international childhood rare disease foundation with the mission to accelerate access to the most effective gene therapy cures for children suffering from ultra-rare diseases and Viralgen, a leading manufacturer of AAV vectors for gene therapy, announce the Target10 Program with the aim to facilitate and accelerate the advancement of 10 programs to address 10 diseases in order ensure equitable access to potentially curative treatments for all known to have them within 10 years.

Fundación Columbus along with its’ American based sister foundation, the Columbus Children’s Foundation (CCF), is known for its translational role in the acceleration of gene therapy programs bringing hope to children with ultra-rare diseases. In addition to advancing its own programs for ultra-rare diseases. Fundación Columbus develops strategic partnerships with key biotech organizations, like Viralgen, to provide needed expertise, access to highly sought manufacturing capacity, and significantly discounted Adeno-associated viral vector (AAV) manufacturing in order to accelerate curative treatments for ultra-rare programs for which there is limited commercial therapeutic value.

Gene therapy is a type of treatment that uses genes to treat or prevent disease.  The gene is introduced into the body encapsulated in a modified virus (AAV) that acts like a vehicle to deliver the gene it carries to specific organs or tissues. This type of therapy offers a new and realistic potential of healing for rare genetic diseases.

While inpidually, rare diseases have very small patient populations, collectively rare diseases impact over 300 million people worldwide. And, 50% of those diagnosed with rare diseases are children. The impact these diseases have on patients, their families and society is profound, as many are serious, chronic and progressive, leading to limited life expectancy. Three out of ten diagnosed children won't live to see get fifth birthday and Fundación Columbus is deeply driven by the urgency to change those odds through accelerating cures for these children so they are not left behind when science can put a cure in reach.

The Target10 Program announced today by Fundación Columbus and Viralgen is a revolutionary and ambitious program that was created with the aim of developing up to ten new effective gene therapy treatments for ten rare diseases over the next ten years. The Target10 Program emphasizes the need to bring together the right resources and expertise to accelerate development of promising research and clinical programs and aims to focus on tackling ultra-rare program development with greater efficiency. Using a non-profit approach, the Target10 Program will identify and seek to advance programs with the best chance to create successful treatments for ultra-rare diseases where currently there are no known cures. The Target10 Program seeks to remove barriers to manufacturing which are seen as the most significant specific hurdle for small patient population programs poised to translate from the lab to the clinic where children can be treated. The Target10 Program will bring together great science and translational capabilities to deliver curative treatments for children who need them most.

As a major contributor and strategic partner of Fundación Columbus, Viralgen has shown clear commitment and has become a leader in advancing programs for children through lending its knowledge and manufacturing capacity to Fundación Columbus driven ultra-rare disease programs. Because of today’s announcement of the Target10 Program, the AAV vectors produced by Viralgen within this Program, will be even more affordable and more accessible.

"Fundación Columbus is deeply driven by the urgency to ensure equitable access to the most effective treatments for children with ultra-rare diseases. We believe no children should be left behind when science could put a cure in reach and we cannot thank Viralgen enough for his exceptionally generous and valuable contribution that will help with our mission to accelerate treatments for these children who not only fight against a life-threatening disease, but also fight against time," commented on Blanca Garín, Director of the Fundación Columbus.

Currently, Fundación Columbus is already facilitating the development of therapies for seven rare diseases: L-Decarboxylase Aromatic Amino Acid Deficiency(AADC) which is also commonly referred to as pediatric Parkinson's and currently in clinical trials; Spastic Paraplegia Type 50 (SPG50), Mucopolysaccharidosis III (Sanfilippo Type C) and an extremely rare Duchenne Muscular Dystrophy mutation which are all in manufacturing and is supporting pre-clinical programs including Niemann-Pick A, Niemann-Pick C, among others. To advance these programs, Fundación Columbus partners with industry, disease specific foundations and leading research centers to accelerate the development of these critical therapies.

For the selection of diseases to be included in the Target10 Program, Fundación Columbus uses an international panel of experts to help determine program selection according to criteria of scientific excellence, absence of key development initiatives due to low commercial interest, team experience, and quality of the development plan.

Viralgen's participation in the Target10 Program is a cornerstone to the program's success. The production of viral vectors is a major bottleneck in the development of gene therapy projects for these small patient programs and having the support of Viralgen will make it possible to achieve the ambitious goals related to the Target10 Program. The partnership of Viralgen will lead to cost savings of several million dollars per program compared to commercial costs and over 12-24 months of time savings. Manufacturing is one of the most difficult steps of this process and working with an industry leader will help close the rare disease gap for families of children with rare diseases. Javier García, CEO of Viralgen commented, "we are delighted to be able to contribute to this ambitious Target10 Program that will bring so much hope to many families with children suffering from devastating diseases. We believe this is a highly effective and powerful model of collaboration for ultra-rare programs that lack interest through traditional investment by the private sector."

Through key partnerships, like the Target10 Program with Viralgen, Fundación Columbus is working toward establishing an ecosystem that society can use to develop therapeutics for ultra-rare diseases using a nonprofit approach that has both science and compassion at its core. Today, with this announcement, we are one step closer to the goal of working toward having treatments for the many diseases that have no known treatments today.

If you want to read the news in Spanish, click here.

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