SOM Biotech, a Barcelona Science Park (PCB), Spain-based clinical-stage biopharmaceutical company, has launched its very first crowdfunding campaign for the purpose of initiating new R&D projects related to the pediatric rare diseases field.

The diseases to be investigated are cystic fibrosis, Duchenne muscular dystrophy, Gaucher disease, Niemann Pick disease type C and osteogenesis imperfecta. The company plans to raise at least $300,000, which would be divided evenly among the five different pediatric rare diseases.

Recently, SOM Biotech has completed a phase IIa clinical trial for Transthyretin Amyloidosis (ATTR) with successful results. Optimistic results obtained from the study, along with another clinical program in Huntington’s disease, were primary motivations in starting the new initiative. In addition, almost 50% of rare disease patients worldwide are children.

Funding will be used to investigate new therapeutic options for those five pediatric rare diseases. The amount will be allocated evenly to each pediatric rare disease program, funding the initial stages of drug repurposing identification to reach the clinical stages of development. At present a list of reference compounds for each pediatric rare disease program has been internally generated and scientifically-evaluated, with plans of identifying new and better therapeutic candidates. SOM's commitment is to advance further in the development of any promising drug to reach the market as soon as possible.

The crowdfunding campaign is taking place within a two-month period that started Dec. 3 and will end Feb. 2, 2016. The chosen platform is the American-based, yet globally-exposed, Indiegogo where funding for all campaigns can be made from public rewards-based donations. Through there, SOM Biotech's campaign will be visible worldwide to various individuals and communities, interested in making a difference for pediatric rare diseases. Given the successes of its clinical programs and the support provided by various organizations, including the Spanish Federation of Rare Diseases (FEDER), senior management is confident of an outstanding performance and the pursuit of long-desired projects.