Press Releases

Proteostasis Therapeutics Presents Data at Multiple Scientific Conferences Highlighting Novel Approach to Neurodegenerative Diseases and Cystic Fibrosis

August 14, 2012 at 5:19 PM EDT

Cambridge, Mass., August 14, 2012 — Proteostasis Therapeutics, Inc., a company developing novel therapeutics that regulate protein homeostasis to improve outcomes for patients with neurodegenerative and orphan diseases, announced today that company scientists, founders and co-founders made the following presentations at scientific conferences in the last month highlighting progress with the Company’s lead programs and the potential of its approach to developing disease modifying therapeutics:

  • Alzheimer’s Association International Conference (AAIC) held in Vancouver, British Columbia on July 14–19, 2012: The Company presented a poster titled “Small-Molecule Inhibition of Usp14 Enhances the Proteolytic Degradation and Clearance of Misfolded Proteins Associated with Neurodegenerative Diseases.” The data highlighted the efficacy of the Company’s compounds in lowering neurotoxic protein aggregates in cells and documented the progress towards lead nomination. Scientific Founder of the Company, Professor Jeffery Kelly, Ph.D. of the Scripps Research Institute, also gave a plenary lecture titled “Is Amyloid a Tractable Target?” His lecture further highlighted the therapeutic potential of modulating protein folding and cellular quality control mechanisms to achieve meaningful clinical outcomes.
  • 4th Annual Ubiquitin Drug Discovery Conference 2012 held in Philadelphia, PA on July 23-25, 2012: Proteostasis Therapeutics gave a podium presentation on the Company’s protein clearance program. The data shown described recent progress on identifying small-molecule regulators of protein degradation pathways, particularly proteasomal protein processing, which can lead to an enhanced rate of removing disease-related cellular protein aggregates. Scientific Co-Founder of the Company, Professor Alfred Goldberg, Ph.D. of Harvard Medical School, also presented a lecture on novel approaches for altering the processing of -synuclein by ubiquitin ligases and how such strategies may lead to the lowering of -synuclein levels in cells. Proteostasis Therapeutics was also a co-sponsor of the conference.
  • FASEB Science Research Conference on “Protein Folding in the Cell” held in Saxtons River, VT on July 29 – August 3, 2012: The Company provided updates on its protein folding program by presenting a poster titled “Identification of Proteostasis Regulators Providing Functional Rescue of CFTRF508.” These data described the development of Proteostasis Network (PN) transcriptional signatures associated with improved folding and trafficking of the CFTRF508 protein in cellular models of Cystic Fibrosis (CF) and the use of these PN signatures to discover compounds that improve the folding and trafficking of CFTRF508, the most common genetic mutation leading to CF. In addition, the Chairman of the Company’s Scientific Advisory Board, Professor Ulrich Hartl, M.D., D. Med of the Max Planck Institute of Biochemistry in Germany, gave the keynote address titled “Chaperone-mediated protein folding in health and disease.” This address focused on the role of chaperonins and chaperones in maintaining the conformational integrity of the cellular proteome, and how deficits in the cellular folding machinery can lead to protein folding and aggregation diseases such as Parkinson’s disease and CF. Other founders and co-founders that presented at this conference were Professor Richard Morimoto, Ph.D. of Northwestern University, Andrew Dillin, Ph.D. of The Salk Institute for Biological Studies, Professor Daniel Finley, Ph.D. of Harvard Medical School, and Professor Jeffery Kelly, Ph.D. of The Scripps Research Institute. Proteostasis Therapeutics was a co-sponsor of this meeting.

“These data further demonstrate our Company’s commitment to developing novel therapeutics for neurodegenerative and orphan diseases and also show the promise of a drug discovery and development approach based on our Proteostasis Network Platform. We look forward to advancing our most promising chemical series to lead optimization this year and providing updates on our programmatic progress as we approach the clinic,” stated Peter Reinhart, President and Chief Scientific Officer of Proteostasis Therapeutics.

About Proteostasis Therapeutics

Proteostasis Therapeutics is developing disease-modifying therapeutics for orphan and neurodegenerative diseases. The Company’s lead programs in Cystic Fibrosis (CF) and protein aggregation disorders such as Parkinson’s disease modulate protein chaperone and proteasomal degradation pathways within the cell. These pathways are part of the cellular ‘quality control’ machinery, called the protein homeostasis network or Proteostasis Network (PN) that regulates protein folding, trafficking, and clearance. By enhancing the function and capacity of the PN, the Company’s product candidates correct for imbalances in the PN resulting from the cumulative effects of disease, genetic mutations, environmental factors, and aging. For more information, please visit

Investor Contact:

Lilian Stern

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