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GEN News Highlights : Jul 26, 2012

Cystic Fibrosis Program Expanded

Proteostasis Therapeutics expanded its collaboration with The Scripps Research Institute (TSRI) to encompass an additional funded research project focused on biology and the testing of small molecule modulators of protein folding and trafficking for the treatment of cystic fibrosis (CF). This expansion follows the company’s recently announced collaboration with the Cystic Fibrosis Foundation to research, develop, and commercialize therapies to treat patients with the most common mutation of the cystic fibrosis transmembrane conductance regulator (CFTR), Delta F508.

Working with the laboratory of William Balch, Ph.D., professor of cell biology at TSRI, scientists at Proteostasis Therapeutics have used an integrated platform composed of genomics, proteomics, functional assays, and medicinal chemistry to identify compounds that regulate key folding and trafficking pathways in the cell. To date, according to the company, these compounds have demonstrated significant efficacy in CF-specific cellular models. Under the expanded collaboration, Proteostasis Therapeutics will provide funding for this research and will have exclusive rights to license any technology originating from the research.

The company believes this newly expanded collaboration will enhance the ability of Proteostasis Therapeutics to perform chaperone-based high-throughput screening in multiple disease-relevant cellular models to identify proteostasis regulators that will correct the folding, trafficking, and function of Delta F508 CFTR, both alone and in combination with agents currently in development or on the market.