About Us

BioBlast Pharma is a privately held, clinical-stage biotechnology company 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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BioBlast Pharma Pipeline table

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EURORDIS Lifetime Achievement Award to Marlene Haffner MD, MPH For over 30 years, Dr Marlene Haffner has had an immeasurable impact upon the development of orphan drug therapies. She has dedicated most of her professional life to facilitating the development of therapies for ...

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