Late last year, PTC Therapeutics bought up California-based BioElectron Technology in a small deal, nabbing its leading drug, EPI-743, for metabolic and mitochondrial disorders.
Now, it’s also adding the company’s former CEO and chief scientific officer to its top clinical team, as Matthew Klein, M.D., becomes its chief development officer. In this role, Klein “will be responsible for the development of our clinical stage programs,” the biotech said.
This includes its early pipeline work around gene therapy, nonsense mutation, splicing and oncology, according to its site, with targets including Huntingdon’s disease, Angelman syndrome, relapsed/refractory acute leukemias, ovarian cancer and others.
PTC markets Emflaza, a controversial Duchenne muscular dystrophy (DMD) treatment, in the U.S. It sells another DMD drug, Translarna, in Europe, and Tegsedi, which treats polyneuropathy of hereditary transthyretin-mediated amyloidosis, in Latin America.
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It also has a 2020 date with the FDA for a potential approval for its Roche-partnered spinal muscular atrophy therapy risdiplam, which will compete with the likes of Biogen and Novartis but could still be a solid blockbuster at peak.
Klein joined PTC last year, initially as global head of gene and mitochondrial therapies, after the BioElectron deal.