BioBlast Pharma is a publicly traded, clinical-stage biotechnology company (NASDAQ – “ORPN”) committed to developing clinically meaningful therapies for patients with rare and ultra-rare genetic diseases.

Founded in 2012, the company is rapidly building a diverse portfolio of product candidates with the potential to address unmet medical need for incurable rare diseases.

Our therapeutic platforms are based on deep understanding of the disease-causing biological mechanism, and can potentially offer solutions for several diseases that share the same biological pathology.

Our business model is based upon three principles:

1. Diversification: Our 3 platforms currently address 6 different diseases with the potential to address tens of additional diseases.

2. Strict selection criteria: we have been very thorough is selecting diseases that ensure timely and cost efficient development process suitable to our resources.

3. Cost efficient virtual operation mode: We have a highly professional core team and outsource all our works to leading laboratories and CRO’s, thus avoiding costly learning curves and set up time. BioBlast’s business operation model enables us to develop multiple programs timely and cost efficiently.

The company is led by a management team experienced in the development and commercialization of disease therapeutics.