Bioblast Pharma

Hope should not be rare

Pipeline updates archive

Pipeline Updates Archive

  • November 19, 2014
  • Bioblast Pharma
  • Pipeline updates

Bioblast Pharma Receives Orphan Drug Designation From U.S. FDA for Cabaletta for the Treatment of Spinocerebellar Ataxia Type 3

Bioblast 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 U.S. Food & Drug Administration (FDA) for Cabaletta for the treatment of Spinocerebellar Ataxia Type 3 (commonly known as SCA3 and Machado Joseph disease). This is the second indication for which Bioblast’s Cabaletta has received such designation.

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  • August 18, 2014
  • Bioblast Pharma
  • Pipeline updates

Bioblast Receives Notice of Allowance from USPTO for Its Novel Fusion Protein-Based Platform for the Treatment of Friedrich’s Ataxia

TEL AVIV, Israel, August 18, 2014 – Bioblast Pharma Ltd, a clinical-stage biotechnology company committed to developing clinically meaningful therapies for patients with rare and ultra-rare genetic diseases, announced today it has received a notice of allowance from the United States Patent and Trademark Office (USPTO) on a composition-of-matter patent for its novel fusion protein for Friedrich’s Ataxia (referred to as BB-FA) that is built on its mitochondrial protein replacement (mPRT) platform. The Company’s breakthrough platform technology has produced drug candidates that have demonstrated efficacy in several mitochondrial protein deficiencies in cell and animal models.

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