SOM Biotech announced the start of its first crowdfunding campaign to initiate new R&D projects into rare pediatric diseases, including Duchenne muscular dystrophy (DMD).
The company, headquartered at the Barcelona Science Park (PCB), aims to raise at least $300,000 to be divided evenly among five rare childhood diseases: DMD, cystic fibrosis, Gaucher’s disease (a genetic disease in which fatty substances accumulate in cells and certain organs), Niemann-Pick disease type C (a lipid storage disease), and osteogenesis imperfecta (an inherited, brittle bone disease).
Recently, SOM Biotech reported promising results from its completed a Phase 2a clinical trial for transthyretin amyloidosis, a slowly progressive condition characterized by the buildup of abnormal deposits of a protein called amyloid in the body’s organs and tissues, especially the peripheral nervous system. Data from this study, together with a clinical program in Huntington’s disease, were the company’s primary motivations for the R&D project, according to a press release.
The global crowdsourcing campaign, “Fight against Pediatric Rare Diseases” using the Indiegogo platform, began Dec. 3, 2015, and will run through Feb. 2, 2016. The $300,000 goal will be equally distributed to investigate novel therapeutics for each disease, funding the initial stages of drug repurposing identification so that the drugs might reach clinical development. A list of reference compounds for each pediatric rare disease program has been internally generated and evaluated, to identify newer and better therapeutic candidates.
The company is confident of success for this campaign and has the support of several organizations, including the Spanish Federation of Rare Diseases (FEDER).
Muscular dystrophies cause progressive weakness and loss of muscle mass, where genetic mutations interfere with the production of proteins necessary to produce healthy muscle. Duchenne muscular dystrophy is a recessive X-linked dystrophy that leads to muscle degeneration, complications in walking and moving, and compromised pulmonary and cardiac function. Science has yet to find a cure for DMD, but therapies available can help to minimize symptoms and maintain patients’ quality of life.