Company intelligence / Profile preview

Bioblast Pharma Ltd.

Tel Aviv, Tel Aviv District, Israelbioblastpharma.com ↗

Ownership: Private
Development stage: Other

Overview

Bioblast developed several clinical candidates for rare diseases. Its lead program, Cabaletta for OPMD, advanced to a Phase 2/3 multicenter study. Trehalose IV solution was studied in over 70 patients with two rare diseases and showed a favorable safety profile and promising efficacy signals. In February 2019, all rights to trehalose were acquired by Seelos Therapeutics[6]. The company later entered into a merger agreement with Enlivex Therapeutics.

Therapeutic areas: Rare DiseasesNeurology
Modalities: Small molecules (notably trehalose)protein stabilization therapiesread-through compounds
Industry: Biotech

Partnerships

Team Sanfilippo Foundation collaboration on trehalose development for Sanfilippo syndrome[6][9]academic collaborations with McGill University/Montreal Neurological Institute for the HOPEMD study on OPMD[8]

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