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 Receives Orphan Drug Designation From the European Commission for Cabaletta for the Treatment of Oculopharyngeal Muscular Dystrophy TEL AVIV, Israel, June 25, 2015 (GLOBE NEWSWIRE) — BioBlast Pharma Ltd. (Nasdaq:ORPN), a clinical-stage orphan disease-focused biotechnology company, announced today that it has been granted Orphan Drug Designation by the European Commission for Cabaletta for the treatment of Oculopharyngeal Muscular Dystrophy (OPMD). BioBlast is starting a double-blind, placebo-controlled Phase 3 pivotal study ...

BioBlast Announces Positive Preclinical Proof-Of-Concept Results Of Its Novel Read- through Platform in Spinal Muscular Atrophy (SMA) In-Depth Presentation: Read-through Platform – Spinal Muscular Atrophy TEL AVIV, Israel, June 22, 2015 – BioBlast Pharma Ltd. (NasdaqGM: 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 Read-through drug candidate (BBrm02) for Spinal Muscular Atrophy (SMA). BBrm02 is a proprietary, intrathecal ...

BioBlast Pharma to Present Preclinical Proofs of Concept in Spinal Muscular Atrophy and Friedreich’s Ataxia at Two Leading Scientific Meetings in June TEL AVIV, Israel, June 18, 2015 (GLOBE NEWSWIRE) — BioBlast Pharma Ltd. (Nasdaq:ORPN), a clinical-stage orphan disease-focused biotechnology company, announced today that it will present pre-clinical proofs of concept for two of its therapeutic platforms during June. At the 19th International SMA Researcher Meeting in Kansas City, MO (see details below), BioBlast will ...

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