Backing will support the firm’s preclinical-stage proteostasis regulator drugs.

Proteostasis Therapeutics (PTI) reports that it has raised $45 million in a Series A financing. The firm has a preclinical pipeline of drugs that regulate the proteostasis network and could have a therapeutic effect on genetic and degenerative disorders.

Investors included HealthCare Ventures, Fidelity Biosciences, New Enterprise Associates, Novartis Option Fund, and Genzyme Ventures.

PTI is focusing on small molecules to treat diseases associated with protein homeostasis deficiencies. Its drug candidates affect the proteostasis network, which comprises over a dozen biological pathways that maintain balance among protein synthesis, folding, aggregation, trafficking, and degradation.

“Our initial funding of PTI over a year ago provided the basis for this emerging area of science to mature so that it now offers the promise of new drug candidates entering the clinic in the next 3-5 years,” points out Christopher Mirabelli, Ph.D., managing director of HealthCare Ventures and chairman of the board of PTI.

“The company has established a map of the proteostasis network, leading to breakthroughs in understanding how stress on the network, including aging, environmental, and cellular stresses, can lead to the onset of multiple serious diseases,” explains Andrew Dillin, Ph.D., cofounder of PTI.

“PTI has identified unique targets as well as small molecules that offer the promise of restoring the balance of the network while preventing the toxicity of damaged proteins associated with disease,” adds Richard Morimoto, Ph.D., cofounder of PTI.

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