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Biopharmaceutical company SOM Biotech, based in the Barcelona Science Park, has launched its first crowdfunding campaign through the US platform Indiegogo to fund research into five rare pediatric diseases: Duchenne muscular dystrophy, Cystic fibrosis, Gaucher disease, Niemann–Pick type C and Osteogenesis imperfecta (more commonly known as brittle bone disease).

The company hopes to raise at least €275,000 with this campaign (running from 3 December to 2 February) and plans to divide the funds equally among the five diseases it is researching with the aim of identifying new and better therapeutic options and developing new drugs to take to market.

The R&D projects have begun to identify and scientifically assess how drugs already on the market can be repositioned for new medical applications (effective in treating one of these diseases), which has been one of the company’s areas of expertise since it was founded in 2009.

SOM Biotech explains that the idea to launch this campaign came from the positive results they’ve seen in phase II clinical trials on a drug to treat Transthyretin amyloidosis (ATTR) and another clinical program for Huntington disease. Another motivating factor is the fact that nearly 50% of all patients with rare diseases in the world are children.


Studying alternative treatments for amyloidosis

The amyloidosis drug, which was tested with 18 patients in a clinical trial at Vall d'Hebron University Hospital, has been proven 100% effective. The research group discovered that this drug, which is currently marketed to treat Parkinson, is four times more effective than the drug currently available for amyloidosis. 

Amyloidosis is a disease that causes the liver to produce the abnormal copies of the protein transthyretin, which later break up in the blood and create fibers that collect on the nervous system, the heart or the brain and eyes. In some cases, this results in polyneuropathy or cardiomyopathy. Patients tend to develop the disease in their 20s or 30s and, depending on the organ affected, life expectancy is less than 5 to 15 years.

The therapeutic options available for the disease are liver transplant or a drug called Tafamidis, which costs €11,000 per month in Spain (for one box of 30 pills). The drug SOM Biotech is looking at unites the transthyretin protein and stabilizes it so it won’t break up. If it is approved, it will be a lifelong treatment.

After successfully completing phase II, the company must license a new medical indication and sell it to a large pharmaceutical corporation, a process that is currently underway. The pharmaceutical company will take care of phase II (clinical trials with a larger sample of patients to prescribe the drug) and marketing. So far, the project has received public and private funding for three years.

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