Flagship Pioneering is All Hands on Deck for Cystic Fibrosis

Cystic Fibrosis_Compressed

Flagship Pioneering is uniting a number of its companies for a good and important cause—developing potential therapeutics for the treatment of cystic fibrosis. The prominent life sciences venture capital firm announced a strategic partnership with the Cystic Fibrosis Foundation on Wednesday morning.

This first-of-its-kind collaboration will leverage the talent and innovation at companies such as Tessera Therapeutics to advance multiple candidates to human proof of concept. The CF Foundation has pledged $110 million to help accomplish the mission. Specifically, it will commit $20 million up-front, with an option to invest the remaining funds if promising treatments materialize.

“Despite recent advances, approximately 10% of individuals living with cystic fibrosis have rare or nonsense mutations and do not have any effective treatment options,” stated Paul Biondi, president of Pioneering Medicines and executive partner at Flagship Pioneering. “We are excited to harness the power of multiple Flagship technologies to create novel medicines for these patients in need.”

Cystic fibrosis is an inherited disease that wreaks havoc on patients’ lungs, with implications for the digestive system and other organs. It is defined by thick, sticky mucus that accumulates in these organs and occurs when someone inherits two copies of the defective cystic fibrosis gene, CFTR, one from each parent. According to the CF Foundation, the disorder afflicts more than 70,000 people globally and around 30,000 in the U.S. alone. It often takes the lives of its victims in early adulthood.

The announcement highlighted Tessera’s Gene Writing technology. The company’s RNA-based gene-writing platform attempts to write into or rewrite the genome based on an RNA template. It could potentially fix CFTR, a protein-coding gene that is mutated or missing in cystic fibrosis.

“Tessera is pioneering Gene Writing to cure diseases by making the full spectrum of genetic modifications from changing a single base pair, inserting or deleting multiple bases, to writing entire genes into the genome. Through this partnership, we will apply Tessera’s Gene Writers to correct specific mutations in the CFTR gene, as well as to write the full-length CFTR gene using all-RNA therapeutic compositions,” said Tessera co-founder and CEO Geoffrey von Maltzahn, who is also a general partner at Flagship Pioneering.

Biondi also noted Flagship Executive Partner John Mendlein’s executive leadership, and the scientific leadership of Dr. Luciano Rossetti, M.D., CMO at Flagship Pioneering and head of R&D at Pioneering Medicines, and Luisa Salter-Cid, Ph.D., chief scientific officer at Pioneering Medicines. 

Another Flagship company, Omega Therapeutics, uses its proprietary Epigenomic Programming platform to fix the dysregulation of gene expression at its source. The Cambridge, Mass.-based company aims to biologically engineer a new class of programmable epigenetic medicines to treat various diseases. Flagship’s Sana Biotechnology is developing platforms that can repair and control genes in cells or replace any cell in the body.

Along with combining existing platforms, the aim will be to develop individual asset companies specifically focused on potential CF treatments. While Vertex Pharmaceuticals, often considered the industry leader in cystic fibrosis, has developed and commercialized four transformative drugs—including Trikafta, a combination of three medicines approved by the U.S. Food and Drug Administration (FDA) in 2019—this is not enough for the foundation.

To help the 10% of patients not helped by drugs like Trikafta, it launched a five-year $500 million research initiative called “Path to a Cure” in 2019. If Flagship receives the full $110 million funding, it would be the initiative’s largest investment to date.

“By combining the CF Foundation’s scientific expertise with Pioneering Medicines’ and Flagship Pioneering’s emerging technologies and track record of innovation, we believe this agreement has the potential to push the field forward and accelerate progress toward future treatments for everyone with CF, regardless of their mutation,” said Dr. William Skach, M.D., chief scientific officer of the Cystic Fibrosis Foundation.

To sum up the non-profit’s intentions, CF Foundation President and CEO Dr. Michael Boyle, M.D., said, “We will not rest until every person with CF has a cure.”

This first-of-its-kind collaboration will leverage the talent and innovation at Tessera Therapeutics to advance multiple candidates to human proof of concept.

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