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Cystic Fibrosis Foundation, Flagship Pioneering team up to develop new treatments for deadly disease

The nonprofit will provide up to $110 million as part of an effort to create medicines for the 10 percent of patients who don’t respond to existing drugs.

An image showing an example of Cambridge-based Tessera Therapeutics' RNA-based "gene writing."
An image showing an example of Cambridge-based Tessera Therapeutics' RNA-based "gene writing."Microverse Studios

The nonprofit Cystic Fibrosis Foundation has signed a deal with Flagship Pioneering ― the Cambridge venture capital firm behind high-flying Moderna ― to provide up to $110 million to develop treatments for the 10 percent of cystic fibrosis patients who don’t benefit from existing drugs.

The Bethesda, Md.-based foundation said Wednesday it will initially commit $20 million to the effort and has an option to invest $90 million more if promising treatments emerge. The deal is with Pioneering Medicines, a recently established Flagship division that plans to use scientific technology from several of Flagship’s 42 portfolio companies in the collaboration.

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In particular, the partners expect to rely on gene-editing technology developed by the Flagship company Tessera Therapeutics of Cambridge to fix CFTR, a protein-coding gene that when mutated or missing, causes cystic fibrosis. Tessera, founded in 2018, calls the technology “gene writing” and hopes to use it to cure diseases, rather than just treat them.

Cystic fibrosis is an inherited disease in which thick, sticky mucus accumulates in organs, including the digestive system and, most dangerously, lungs. It is often fatal in early adulthood. People with the disease have inherited two copies of the defective cystic fibrosis gene, one copy from each parent. More than 70,000 people live with cystic fibrosis worldwide, including over 30,000 in the US, according to the foundation.

Since 2011, Boston-based Vertex Pharmaceuticals has won approval of four cutting-edge drugs that have made the disease manageable for about 90 percent of patients. They include Trikafta, a three-medicine combination approved in 2019 by the Food and Drug Administration, with a list price of $311,000 a year. Nonetheless, 7 to 10 percent of patients don’t benefit from the treatments because they have rare mutations that cause the condition.

“There have been transformative medicines that have benefited people with cystic fibrosis,” but not everyone, said Michael Boyle, president and chief executive of the foundation. “This is just part of our commitment to develop treatments for everybody with cystic fibrosis.”

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In 2019, the foundation announced a five-year, $500 million research initiative called “Path to a Cure” to develop more treatments and, potentially, a cure for the disease. The initiative now includes 24 programs at drug companies and academic institutions, Boyle said. If the foundation provides Flagship with $110 million, it would be the nonprofit’s single biggest investment.

As it happens, Vertex is collaborating with Moderna on another potential treatment to address the underlying cause of cystic fibrosis by enabling cells in the lungs to produce functional CFTR. That treatment would rely on the messenger RNA technology that the Cambridge company used in its COVID-19 vaccine. Reshma Kewalramani, Vertex’s chief executive, said in a quarterly earnings call Tuesday that the partners plan to ask the FDA to let them start testing the treatment in the clinic next year.

Boyle said the foundation doesn’t view the two dozen efforts as competing against each other.

“We need multiple companies to be applying their technologies to address this challenge,” he said in an interview. “We are focused on finding a therapy, not the success of one company.”

For Flagship, the collaboration with the foundation will be the first big test of its Pioneering Medicines division, created about two years ago. It was established to pool technologies from Flagship companies that might be helpful treating diseases that aren’t the focus of those firms.

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“This is a powerful opportunity to bring together the capabilities of multiple Flagship bioplatform companies, along with the disease expertise and extensive network of the Cystic Fibrosis Foundation,” said Paul Biondi, a 17-year veteran of Bristol-Myers Squibb who heads Pioneering Medicines, in a statement.


Jonathan Saltzman can be reached at jonathan.saltzman@globe.com.