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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News

BioBlast Pharma Enhances Leadership Team Appoints Industry Veteran, Robert W. Cook, as Chief Financial Officer NEW HAVEN, Conn., Jan. 11, 2016 (GLOBE NEWSWIRE) — BioBlast Pharma Ltd. (Nasdaq:ORPN), a clinical-stage biotechnology company committed to developing meaningful therapies for patients with rare and ultra-rare genetic diseases, announced today that it has appointed Robert (Bob) Cook to the position of ...

BioBlast Pharma Provides Research and Development Update NEW HAVEN, Conn., Dec. 9, 2015 (GLOBE NEWSWIRE) — BioBlast Pharma Ltd. (Nasdaq:ORPN), a clinical-stage biotechnology company developing meaningful therapies for patients with rare and ultra-rare genetic diseases, today provided an update on its research and development programs.Recently, the Company announced positive, open-label, interim HOPEMD Phase 2 clinical data for Cabaletta in ...

BioBlast Pharma Strengthens Leadership Team and Announces Key Organizational Changes Significant Evolution of Senior Leadership Team in 2015 Co-Founders Transition Company to Globally-Experienced Executives NEW HAVEN, Conn., Nov. 16, 2015 (GLOBE NEWSWIRE) — BioBlast Pharma Ltd. (Nasdaq:ORPN), a clinical-stage biotechnology company developing meaningful therapies for patients with rare and ultra-rare genetic diseases, announced the appointment of three new senior executives to its leadership team: ...

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