CFF Investing Up to $110M, Partnering to Advance Gene Therapy

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by Margarida Maia, PhD |

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The Cystic Fibrosis Foundation (CFF) is partnering with Pioneering Medicines, an initiative of Flagship Pioneering, to support the development of various types of gene therapy for cystic fibrosis (CF).

The foundation is ready to invest up to $110 million to move therapy candidates into clinical testing, starting with an initial $20 million investment. As part of the collaboration, CFF also has the option of making extra money available as promising therapies are developed.

“We will not rest until every person with CF has a cure. 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,” Michael Boyle, MD, president and CEO of the CFF, said in a press release.

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The disease is caused by mutations in CFTR, a gene that codes for a protein of the same name. The mutations lead to the production of a protein that does not work properly.

Overall, gene therapy aims to allow cells to produce a working version of the CFTR protein. As such, it is expected to work similarly in all CF patients, regardless of what specific mutation they carry.

“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 William Skach, MD, chief scientific officer of the CFF.

The collaboration will make use of technologies developed by companies within the Flagship portfolio, including Tessera Therapeutics and its Gene Writing platform.

The intent is to develop a separate company whose specific focus is on CF.

“Despite recent advances, approximately 10% of individuals living with cystic fibrosis have rare or nonsense mutations and do not have any effective treatment options,” Paul Biondi, president of Pioneering Medicines and executive partner of Flagship, said in a separate press release. (Nonsense mutations are those that result in a truncated form of a protein, which is quickly degraded within the cell.)

“We are excited to harness the power of multiple Flagship technologies to create novel medicines for these patients in need,” Biondi added.

The new company’s initial focus will be on developing technologies capable of creating a functional CFTR protein in lung cells, and on a gene writing approach able to simultaneously correct different types of mutations in the CFTR gene. Tessera’s Gene Writing platform is expected to be of help here. The technology is reported to work by writing — or rewriting — the genetic code on DNA based on an RNA template that serves as a blueprint. As such, it has the potential to correct multiple mutations at the same time by making small changes, as well as integrating entire genes into the genome.

“Gene Writers have the potential to revolutionize the treatment of cystic fibrosis and to deliver multiple new medicines that introduce a full length CFTR sequence, or to make true corrections of pathogenic alleles [disease-causing gene copies] in the CFTR sequence,” Biondi said in another press release.

In addition to the lungs, the approach also aims to potentially target other tissues.

“We are eager to address this longstanding unmet need, and ultimately to improve the lives of people living with cystic fibrosis,” said Geoffrey von Maltzahn, co-founder and CEO of Tessera Therapeutics, and general partner of Flagship Pioneering.

The CFF’s investments are part of its Path to a Cure, a $500 million initiative that supports the development of treatments that address the root cause of CF.