Date: 2015-03-19
Type of information: R&D agreement
Compound: new treatments for orphan disorders including Translarna™ (ataluren)
Company: PTC Therapeutics (USA - NJ) University of Pennsylvania (USA - PA)
Therapeutic area: Rare diseases
Type agreement: R&D collaboration
Action mechanism: inducer of ribosomal readthrough on nonsense mutation mRNA stop codons.
Disease: orphan diseases including mucopolysaccharidosis I
Details: * On March 19, 2015, PTC Therapeutics announced a collaboration with the Orphan Disease Center at the Perelman School of Medicine at the University of Pennsylvania focused on translational research for discovering and developing new treatments for orphan disorders. The partnership reflects both parties\' commitment to developing treatments and improving the lives of those who suffer from rare diseases. The first collaborative initiative will focus on Translarna™ (ataluren) for the lysosomal storage disease Mucopolysaccharidosis I (MPS I) due to a nonsense mutation. PTC is initiating a multi-center, Phase 2, proof-of-concept study which will evaluate the safety and pharmacokinetics of Translarna™ in patients with nonsense mutation MPS I. Pharmacodynamic activity will also be explored by an assessment of GAG levels in cerebrospinal fluid, urine and blood. Initial data from this study is expected by the end of 2015.
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