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.

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

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.

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Opening Bell – NASDAQ IPO Sep 9th, 2014

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News

BioBlast Receives Notice of Allowance From USPTO Covering Cabaletta for the Treatment of OPMD BioBlast Pharma Ltd (NasdaqGM: ORPN), a clinical-stage, orphan disease-focused biotechnology company, announced today it has received a notice of allowance from the United States Patent and Trademark Office (USPTO) on a composition and method for ...

BioBlast Announces Key Leadership Changes BioBlast Pharma Ltd. (NasdaqGM: ORPN), a clinical-stage biotechnology company committed to developing meaningful therapies for patients with rare and ultra-rare genetic diseases, announced key senior leadership changes designed to support the Company’s global clinical efforts, ...

BioBlast Provides Update on Clinical Trial of Cabaletta(TM) for Oculopharyngeal Muscular Dystrophy (OPMD) TEL AVIV, Israel, Jan. 8, 2015 (GLOBE NEWSWIRE) — BioBlast Pharma Ltd. (ORPN), a clinical-stage biotechnology company committed to developing meaningful therapies for patients with rare and ultra-rare genetic diseases, announced today preliminary findings from ...

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