December Update

Featured This Month:

We have agreed to invest up to $15 million in ReCode Therapeutics to support the development of a gene editing therapy they are working on with Intellia Therapeutics that could be delivered into the lung cells of people with cystic fibrosis. While ReCode Therapeutics concentrates on the delivery system, Intellia Therapeutics is developing the gene editing therapy being delivered. The therapy is designed to target permanent or near-permanent correction of a specific nonsense mutation (also called a “stop” mutation) in the cystic fibrosis transmembrane conductance regulator (CFTR) gene. Other rare mutations that are not eligible for CFTR modulators will be explored as the editing technology matures.   

Clarametyx Biosciences started the Phase 2a portion of a clinical trial of its novel therapy that aims to disrupt bacterial biofilms, which are protective structures that bacteria form to shield them from both the immune system and antibiotics. The drug could help people with CF with difficult-to-treat infections because it potentially makes bacteria more susceptible to antibiotics and the body’s immune response. A Phase 1b trial of the drug in combination with inhaled Tobramycin did not show any safety issues in people with CF with Pseudomonas infections. Earlier this year, we agreed to invest up to $5 million in Clarametyx to support their Phase 1b/2a clinical trials. Use our Clinical Trial Finder to find enrolling clinical trials for anti-infectives. 

The CFTR2 database, which lists CFTR variants (or mutations), was recently updated to 1,085 CF-causing variants — a 35% increase that now includes 367 variants not previously searchable on the CFTR2 website. Most of these additional variants are rare, occurring in 1 to 3 individuals in the database. Additionally, 11 variants previously categorized as being of uncertain significance have been recategorized as: 

A study published in the Journal of Cystic Fibrosis showed that the CF Foundation’s Daily Care Check-In tool was effective in helping people with CF identify factors that prevented them from doing daily care activities such as airway clearance. If you have concerns or face obstacles in doing therapies regularly, ask your care team if the Daily Care Check-In tool could help you identify barriers to sustaining your daily care.  

Want to talk to someone who also has cystic fibrosis or has children with CF, but don't know where to start? You can find support from other people who have had similar experiences as yours through CF Peer Connect. Complete the application or email [email protected] to get started and someone will follow up with you. The program also makes connections to Spanish speakers. To view the application in Spanish, click the language button on the top right of the page and select “Spanish” from the drop-down menu.  

Recently published guidance for CF clinical care provides considerations and best practices for CF care and the number or type of health professionals needed to provide care as people with CF age and face a widening range of health needs. This interim guidance will continue to evolve as research is ongoing.  

Earlier this year, we asked you to share your experiences with health care costs and how those costs may impact you and your family. You generously shared your experiences, and the results showed that though each person’s journey with cystic fibrosis can look different, the burden associated with the disease can be felt regardless of geography, income, and insurance coverage.