CFF, Deep Science Ventures Team Up to Advance Gene Therapies

Marisa Wexler, MS avatar

by Marisa Wexler, MS |

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A collaboration between the Cystic Fibrosis Foundation and Deep Science Ventures aims to identify technologies that might overcome the challenges inherent in developing gene therapies for cystic fibrosis (CF).

The effort is part of the foundation’s Path to a Cure initiative, a $500 million research program whose focus includes advancing therapies that target the disease’s underlying cause to, ultimately, arrive at “a cure for every person with CF,” according to a press release.

“Our goal is therapies and a cure for 100% of people with CF. By aligning the Foundation’s CF expertise and scientific capabilities with Deep Science Ventures’ track record of uncovering innovative technologies, this agreement has the potential to push the CF field forward and significantly accelerate progress toward future therapies for CF,” said Kara Foshay, PhD, director of biopharma and medicine programs at the CF Foundation.

CF is caused by mutations in the gene CFTR (cystic fibrosis transmembrane conductance regulator), which encodes instructions for a protein of the same name. The CFTR protein normally functions like a “gate” on the surface of cells, helping to regulate the movement of water and salts in and out of cells. CFTR mutations affect the protein’s ability to work as it should, leading to a buildup of thick and sticky mucus in organs like the lungs and pancreas.

The overarching aim of gene therapies for CF and other genetic diseases is to correct the underlying genetic deficit. In the case of CF, this would mean delivering a non-mutated version of the CFTR gene to cells, allowing them to produce a working version of the key protein.

Recent years have seen substantial advances in gene therapy development and use. However, translating this knowledge into therapies for CF poses particular challenges.

In CF, cells in the lungs are among those most affected — and, as such, are a leading target for a gene therapy.  Delivering gene therapies to the lungs, however, requires bypassing the body’s natural defense mechanisms, such as physical barriers in the lungs. These mechanisms evolved to protect lung cells from potentially harmful substances in the air, but they also can lessen a therapy’s effectiveness by preventing it from reaching target cells.

Once such mechanism is the protective natural layer of mucus that surrounds lung cells and works to trap germs and other invaders from reaching the cell, the foundation notes on a webpage devoted to gene therapies and CF. Because patients’ mucus is much thicker than that of other people, their lung cells are even more difficult to reach.

Deep Science Ventures and the CF Foundation will collaborate to assess this and other obstacles to gene therapies for CF, explore potential solutions, and design proof-of-concept studies.

“Deep Science Ventures has a novel approach to innovation and company creation. … We are excited to apply our expertise together with the CF Foundation to significantly accelerate progress towards future therapies for cystic fibrosis,” said Kerstin Papenfuss, PhD, associate director of pharmaceuticals at Deep Science Ventures.