About Us

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 processes, and can potentially offer solutions for several diseases that share the same biological pathology.

BioBlast’s strategy is based upon time and cost-efficient drug development, with the goal of delivering safe and effective solutions to patients in dire need of therapy.

Our business model is based upon three principles:
1. Diversification
2. Strict selection criteria
3. Cost efficient virtual operation mode

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

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BioBlast Pharma Announces Pricing of $6.7 Million Registered Direct Offering NEW HAVEN, Conn., March 17, 2016 (GLOBE NEWSWIRE) — BioBlast Pharma Ltd. (Nasdaq:ORPN) (the “Company” or “ORPN”) announced that it has entered into definitive securities purchase agreements with two healthcare focused institutional investors providing for the issuance of approximately 2.16 million ordinary shares at a purchase price per share of ...

BioBlast Pharma Announces Positive Final Results from HOPEMD Phase 2 Open-Label Clinical Study of Trehalose 90mg/mL IV Solution in Oculopharyngeal Muscular Dystrophy (OPMD) NEW HAVEN, Conn., March 16, 2016 (GLOBE NEWSWIRE) — BioBlast Pharma Ltd. (Nasdaq:ORPN), a clinical-stage, orphan disease-focused biotechnology company, announced positive results from its HOPEMD Phase 2 six-month open-label clinical study in patients with oculopharyngeal muscular dystrophy (OPMD), a rare progressive muscle-wasting disease characterized by swallowing difficulties (dysphagia), leading to the risk ...

BioBlast Pharma to Present Corporate Overview at the 28th Annual ROTH Conference NEW HAVEN, Conn., March 10, 2016 (GLOBE NEWSWIRE) — BioBlast Pharma Ltd. (Nasdaq:ORPN), a clinical-stage, orphan disease-focused biotechnology company, announced today that Colin Foster, President and CEO, will provide a corporate overview and business update at the 28th Annual ROTH Conference, taking place in Orange County, California, March 13-16, 2016. Presentation Details: Title: 28th Annual ...

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