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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BioBlast Pharma Announces Positive Preclinical Proof-of-Concept Results of Its Novel Mitochondrial Protein Replacement Platform in Friedreich’s Ataxia BioBlast Pharma Ltd. (ORPN), a clinical-stage, orphan disease-focused biotechnology company announced today positive preclinical in vitro and in vivo proof-of-concept study results for its mitochondrial protein replacement therapy drug candidate (BB-FA) for Friedreich’s Ataxia. Results ...

BioBlast to Present at the 27th Annual ROTH Conference March 8-11, 2015 The Ritz Carlton 1 Ritz Carlton Dr Dana Point, CA 92629 Webcast: http://wsw.com/webcast/roth29/orpn/

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 ...

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