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.
Opening Bell – NASDAQ IPO Sep 9th, 2014
Bio Blast Pharma Receives Orphan Drug Designation From U.S. FDA for Cabaletta for the Treatment of Spinocerebellar Ataxia Type 3 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 that it has been granted Orphan Drug Designation by the ...
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 ...