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.

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 Announces Granting of Fast Track Designation by the FDA for Cabaletta in Oculopharyngeal Muscular Dystrophy (OPMD) BioBlast Pharma Ltd. (NasdaqGM: ORPN), a clinical-stage, orphan disease-focused biotechnology company announced today that the United States Food and Drug Administration (FDA) has granted Fast Track designation to Cabaletta for the treatment of patients with Oculopharyngeal Muscular Dystrophy (OPMD). Cabaletta is being developed to treat OPMD, a rare and debilitating muscular dystrophy ...

BioBlast Pharma Announces Positive in vivo Preclinical Proof-of-Concept Results of its Cabaletta Platform in Spinocerebellar Ataxia Type 3 (SCA3) TEL AVIV, Israel – BioBlast Pharma Ltd. (NasdaqGM: ORPN), a clinical-stage, orphan disease-focused biotechnology company announced today positive in vivo proof-of-concept results for Cabaletta for Spinocerebellar Ataxia Type 3 (also known as SCA3 or Machado Joseph disease). Results were presented at the International Ataxia Research Conference held in Windsor UK, March 25-28, ...

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 will be presented at the International Ataxia Research Conference held in Windsor UK, March 25-28, 2015.” BB-FA demonstrated ...

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