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Pioneering research collaboration seeks cure for cystic fibrosis

Backed by the non-profit Cystic Fibrosis Foundation and venture capital group Flagship, Pioneering Medicines will identify and develop genetic therapies for CF

In 1989, a team of researchers in Canada made one of the most significant breakthroughs in human genetics when they identified the first disease-causing gene. It was the cystic fibrosis transmembrane conductance regulator (CFTR) gene, mutations of which cause the genetic disease, cystic fibrosis (CF).

This breakthrough was met with wild optimism that a cure was just around the corner but, as researchers began to identify more and more mutations – more than 2,000 different mutations have now been identified – the goal slipped further from sight.

Now, the search for a cure to CF is back on the agenda with the announcement that the US Cystic Fibrosis Foundation will give up to $110m to Pioneering Medicines to accelerate the development of genetic therapies for CF.

Pioneering Medicines is a strategic initiative within Flagship Pioneering, the venture capital group behind Moderna and an ‘ecosystem’ of more than 40 ‘transformative companies’.

“We will not rest until every person with CF has a cure,” said Dr Michael Boyle, president and chief executive officer of the Cystic Fibrosis Foundation. “This collaboration marks an evolution in our approach to venture philanthropy and expands our footprint to bring the best scientific minds and technologies into CF.”

The funding from the Foundation is part of the $500m Path to the Cure research programme that it launched in 2019. Previously, it worked with Vertex Pharmaceuticals to develop the small molecule drugs that focus on ‘fixing’ the protein in the cells of CF patients, but now has its eye on a cure.

“Despite recent advances, approximately 10% of individuals living with cystic fibrosis have rare or nonsense mutations and do not have any effective treatment options,” said president of Pioneering Medicines, Paul Biondi. He added that the new collaboration was “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, and the drug development capabilities of Pioneering Medicines”.

Initially the new company will focus on two strategies – to develop the technology to create a functional CFTR protein in lung cells and a gene-writing approach to correct mutations in the CFTR gene. These two strategies will be combined with a delivery approach focused on targeting the proper cells in the lung and potentially other tissues, said the CF Foundation.

Article by
Hugh Gosling

4th November 2021

From: Research, Healthcare

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