Funding for a focused approach for gene editing research announced today

How would gene editing therapies work?

Gene editing therapies will literally target the CF gene itself. They work by making a cut in the DNA near the CF mutation that researchers want to repair, correcting the repair, and then using the cell’s natural enzymes to seal up the cut bits of DNA. The overall principle is the same for every CF mutation; the complicated bit is delivering the editing tools to the right cell, working out the best method to do the repair and ensuring that the repair doesn’t have any unintended consequences. It’s a very fast moving field and new, more precise methods are being developed all of the time. In principle, gene editing can be applied to correct all CF mutations, however there is an increased focus on the rare CF mutations that don’t work with the CFTR modifier drugs.

Why do genetic therapies take so long to develop?

The development of genetic therapies for CF is a long way behind the development of CFTR modifiers, as the technology is just so new! While every drug is different and needs to be tested thoroughly, researchers working with CFTR modifiers are building on hundreds of years of scientific knowledge. In contrast, the potential of the ‘CRISPR’ method that researchers are currently using to edit the CF gene in the lab was first reported in 2012. Scientists are working extremely quickly to understand this new technology, but there is still so much they need to learn.

Last month the researchers who developed the CRISPR method of gene editing won the Nobel Prize for Chemistry, one the biggest awards in science. The Prize Committee described gene editing as having ”a revolutionary impact on the life sciences”, going on to say that it ”may make the dream of curing inherited diseases come true”.

Dr Lucy Allen, our Director of Research, commented that:“We are all incredibly excited about the huge effect that CFTR modifiers like Kaftrio are likely to have for many people with CF. However, we have been and will continue to be absolutely committed to funding innovative and world-leading research that will have the same life-changing impact for all people with cystic fibrosis.

“Making gene editing a reality for people with CF will take the coming together of expertise from across the world. That's why we're excited about being able to work in partnership with the CF Foundation to fund our newest, collaborative SRC.”

Speaking about the partnership, William Skach, MD, Executive Vice President and Chief Scientific Officer of the Cystic Fibrosis Foundation, said: “Advancing gene editing therapies is a top priority for the CF Foundation, but it will require collaboration to address challenging basic questions. By working with the UK CF Trust to support this Strategic Research Centre, we are moving one step closer to our ultimate goal of developing a cure for CF.”

What do we still need to know?

Within their current gene editing SRC, Professor Stephen Hart and colleagues have worked out a process for studying gene editing therapies in the lab. This ranges from techniques that can be used to deliver the gene editing tools into cells, to how to test that the edits they’ve made lead to a fully-functioning CF protein in lung cells in the lab.

“Our new SRC focuses on the key questions that we need to answer to bring gene editing therapies closer to the clinic. We have developed a range of tools and gained a lot of experience in applying gene editing in CF, now we need to refine these and work out the best way to proceed towards therapies for cystic fibrosis” explains Prof Hart. “It’s a very exciting area of science to work in and our thanks go to the Cystic Fibrosis Trust and the CF Foundation, and their supporters for the opportunity to continue this research.”

You can read more detail about the techniques they’ll be refining and where this research will take place, in a summary of the SRC on our website.