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Proteostasis, Scripps Research expand collaboration on cystic fibrosis

Friday, July 27, 2012

Proteostasis Therapeutics, a therapeutics developer focusing on regulating protein homeostasis for neurodegenerative and orphan diseases, has 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).

The expansion will enhance the ability of Proteostasis 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 the most common mutation of the cystic fibrosis transmembrane conductance regulator (CFTR), Delta F508, both alone and in combination with agents currently in development or on the market.

The expansion follows Proteostasis’ recently announced collaboration with the Cystic Fibrosis Foundation to research, develop and commercialize therapies to treat patients with Delta F508.

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

“Dr. Balch’s expertise in CF biology and protein homeostasis complements our proprietary technology for characterizing proteostasis network pathways in normal and disease states. The expansion of this collaboration further underscores our commitment to working with leading academic scientists and institutions in our focus areas in neurodegenerative and orphan diseases,” stated Peter Reinhart, chief scientific officer, Proteostasis Therapeutics.

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