Protego Raises $51M to Support Its Protein-misfolding Therapies

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by Marisa Wexler MS |

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Protego Biopharma has raised $51 million to support the development of therapies for diseases caused by protein misfolding, such as familial amyloid polyneuropathy (FAP).

The series A financing was co-led by Lightspeed Venture Partners, Vida Ventures, and MPM Capital. As part of the financing, representatives from these three groups will join the board of directors at Protego.

“We are thrilled to partner with a world-class investor syndicate during this pivotal stage as we accelerate the science from research to potentially delivering impactful medicines for patients,” Richard Labaudinière, PhD, Protego’s CEO and president, said in a press release.

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Proteins, a diverse group of molecules critical to virtually every aspect of life, are made up of building blocks called amino acids, which are strung together into long chains. One or more of these amino acid chains are folded into a specific three-dimensional structure to form the mature protein.

For any protein to work properly, it must be folded into the correct shape. Consequently, when proteins are misfolded, substantial problems may arise. FAP is caused by mutations that lead to the production of a misfolded version of transthyretin, a protein mainly produced in the liver that is responsible for transporting certain hormones and vitamins through the body.

Many other diseases, including Alzheimer’s, Parkinson’s, cystic fibrosis, and chronic obstructive pulmonary disease, are also thought to involve protein misfolding.

“Protego is building on compelling science to develop small molecule therapeutics targeting protein misfolding, which is increasingly recognized as an underlying cause in many chronic degenerative diseases, and an area with enormous unmet medical need,” Labaudinière said.

Specifically, the company is pursuing two broad strategies to correct protein misfolding. One is to use “pharmacological chaperones,” which are molecules that can bind to misfolded proteins and force them into the correct configuration. The other is to modulate cellular processes that normally help to prevent protein misfolding.

“We believe that Protego’s innovative therapeutic approach has the potential to become a game changer for the treatment of protein misfolding disorders,” said Shelley Chu, MD, PhD, and a partner at Lightspeed who has now joined Protego’s board of directors.

Protego was founded by Labaudinière alongside Jeffery W. Kelly, PhD, and Xin Jiang, PhD.

These researchers helped to develop the medication that would become Pfizer’s Vyndaqel (tafamidis), which is approved to treat FAP in Europe. In the U.S., Vyndaqel is approved to treat heart disease related to transthyretin misfolding (TTR cardiac amyloidosis), but not FAP.

“We are thrilled to support the vision of these founders with a proven expertise to tackle this area of unmet medical need in novel ways,” Chu said.