SOM Biotech has announced its first crowdfunding campaign to raise at least $300,000 to be used in embarking on a new project to study pediatric rare diseases. The money raised will be divided equally among five diseases to be researched: Cystic Fibrosis, Gaucher Disease, Duchenne Muscular Dystrophy, Osteogenesis Imperfecta, and Niemann Pick Disease Type C.
The main motivation behind this new project was the successful results obtained from the biopharmaceutical company’s recent Phase IIa medical tests for Transthyretin Amyloidosis (ATTR) from which it obtained successful results, as well as the findings acquired from a medical program on Huntington’s disease. Ultimately, the decisive factor behind this initiative is the fact that nearly 50 percent of patients suffering from rare diseases are children.
The money raised will be used to finance the early stages of identifying drug repurposing so that it can be taken to medical stages for further development. Up to now, several reference compounds have been identified for each program of the five pediatric rare diseases. The compounds have been generated internally followed by scientific evaluations. These are carried out in order to recognize new and improved therapeutic compounds. The main goal of SOM Biotech is to enhance development of promising drugs, and to ensure that they are available in the market within the shortest time possible.
The SOM’s crowdfunding campaign is running for 2 months. It started on December 3, 2015 and will conclude on February 2, 2016. The company has invited individuals and communities worldwide to be part of the group that wants to make a difference when it comes to pediatric rare diseases. Based on the support that the company has received from different organizations, such as the Spanish Federation of Rare Diseases (FEDER), during its past medical programs, they are confident that the campaign will be a success by providing the means for the project, and an incentive to expertly put it into effect.