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

Bio Blast Pharma Announces Initiation of Its Canadian OPMD Clinical Study Center at McGill University, Montreal TEL AVIV, Israel, Sept. 22, 2014 (GLOBE NEWSWIRE) — Bio Blast Pharma Ltd (ORPN), a clinical-stage biotechnology company committed to developing clinically meaningful therapies for patients with rare and ultra-rare genetic diseases, announced today it ...

BioBlast Pharma Announces Preclinical and Clinical Pharmacokinetic Results with Cabaletta, Interim Safety Results from Phase 2/3 HOPEMD Study in OPMD Interim Data on First 11 Patients in the Ongoing Phase 2/3 HOPEMD Study Suggest Cabaletta to be Safe and Well Tolerated with No Drug Related Adverse Events TEL AVIV, Israel, September 17, 2014 – BioBlast Pharma Ltd ...

BioBlast Receives Notice of Allowance from USPTO for Its Novel Fusion Protein-Based Platform for the Treatment of Friedrich’s Ataxia TEL AVIV, Israel, August 18, 2014 – Bio Blast Pharma Ltd, a clinical-stage biotechnology company committed to developing clinically meaningful therapies for patients with rare and ultra-rare genetic diseases, announced today it has received a ...

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