Bioblast Pharma

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Trial-related news archive

Trial-related News Archive

  • March 30, 2015
  • Bioblast Pharma
  • Trial-related news

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, 2015.

Cabaletta was tested in two different animal models of the disease:

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  • March 26, 2015
  • Bioblast Pharma
  • Trial-related news

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 efficacy in penetrating mitochondria in several patient cell lines and in two different and well established animal models of Friedreich’s Ataxia.

In in-vitro studies performed in Friedreich’s Ataxia patients’ cells, BB-FA successfully penetrated the mitochondria and restored the damaged mitochondrial functions to close to normal levels both in studies of oxidative stress and in several biochemical functions that involve critical respiratory chain metabolism in a dose-dependent and time-dependent manner.

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  • January 8, 2015
  • Bioblast Pharma
  • Trial-related news

Bioblast Provides Update on Clinical Trial of Cabaletta(TM) for Oculopharyngeal Muscular Dystrophy (OPMD)

TEL AVIV, Israel, Jan. 8, 2015 (GLOBE NEWSWIRE) — Bioblast Pharma Ltd. (ORPN), a clinical-stage biotechnology company committed to developing meaningful therapies for patients with rare and ultra-rare genetic diseases, announced today preliminary findings from its HOPEMD Phase 2/3 study investigating Cabaletta (trehalose) in patients with Oculopharyngeal Muscular Dystrophy (OPMD).

The multicenter study is testing the safety and tolerability of Cabaletta in patients suffering from OPMD – a progressive, debilitating genetic disease – across three centers in Israel, Canada and the U.S.

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