Proteostasis Therapeutics, Inc., a company developing novel therapeutics that regulate protein homoeostasis to improve outcomes for patients with 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).

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.

“This expanded collaboration will allow us to accelerate our CF programme as we work to advance our most promising series to lead optimization this year,” said Mark Enyedy, chief executive officer of Proteostasis Therapeutics.

Working with the laboratory of William Balch, Ph.D., Professor of Cell Biology at TSRI, scientists at Proteostasis Therapeutics 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 Therapeutics will provide funding for this research and will have exclusive rights to license any technology originating from the research.

“We are excited to deepen our relationship with Proteostasis Therapeutics to develop novel approaches that manage the root cause of the problem of this devastating disease,” added Dr. Balch.

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.

“Dr. Balch’s expertise in CF biology and protein homoeostasis 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 of Proteostasis Therapeutics.

The Scripps Research Institute is one of the world's largest independent, not-for-profit organizations focusing on research in the biomedical sciences.