SOM Biotech is launching a crowdfunding campaign for Pediatric Rare Diseases

SOM Biotech is one of the few biopharmaceutical companies in the world specialized in identifying new therapeutic activities of drugs approved for other indications (reprofiling or repositioning), which considerably reduces the time and investment costs for the arrival of new drugs to the market.

Recently, SOM Biotech has completed a Phase IIa clinical trial for Transthyretin Amyloidosis (ATTR) with successful results. Optimistic results obtained from this study, along with another clinical program in Huntington´s Disease, were primary motivations in starting this new initiative. The diseases to be investigated are Cystic Fibrosis, Duchenne Muscular Dystrophy, Gaucher Disease, Niemann Pick Disease Type C, and Osteogenesis Imperfecta.

With the aspirational goal of raising at least 300,000 $USD, funding will be used to investigate new therapeutic options for these 5 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.

"Given the successes of its clinical programs and the support provided by various organizations, including the Spanish Federation of Rare Diseases (FEDER), we are confident of an outstanding performance and the pursuit of long-desired projects", say Raúl Insa, CEO of SOM Biotech.

Contributions can be made at http://igg.me/at/fightrarediseases

Image: Raúl Insa (behind at the center) with SOM Biotech team.