According to a story from Fierce Biotech, there have been some impressive advances in the treatment of cystic fibrosis recently. The latest milestone was in developing and approving the drug Trikafta, which was developed by Vertex and has the potential to provide benefits for most cystic fibrosis patients. However, for a small number patients with the disorder that is linked to rare mutations, Trikafta has no effect. Clearly a new medication is needed for this group. Recently, ReCode Therapeutics has announced that is successfully raised $80 million towards the creation of a new medication for these patients.
About Cystic Fibrosis
Cystic fibrosis is a type of genetic disorder which can have impacts throughout the body, but it is most characterized by the build up of abnormally thick, sticky mucus in the lungs. This mucus becomes a fertile breeding ground and habitat for potentially infectious bacteria. Many patients must take antibiotics for much of their lives. This disorder is caused by mutations of the CFTR gene. Symptoms of cystic fibrosis include progressive decline in lung function, lung and sinus infections, coughing up mucus, fatty stool, poor growth, infertility in males, clubbed digits, and digestive problems. Treatment includes antibiotics and medications or procedures intended to maintain lung function. Lung transplant is an option when lung function declines severely. Life expectancy ranges into the 40s and 50s with good care. To learn more about cystic fibrosis, click here.
Interfering with RNA
ReCode is developing an RNA based treatment for cystic fibrosis linked to nonsense mutations and for a similar disorder called primary ciliary dyskinesia. For these treatments, each disorder is linked to a specific mutation that the treatments will attempt to rectify. The cystic fibrosis drug uses transfer RNA which will hopefully counteract the nonsense mutation.
ReCode says that the mechanisms for these treatments are very similar and plans trial designs for them that are largely comparable. If the drug wind up being successful, the company hopes to translate the same approach for other genetic diseases as well, laying the groundwork for a new wave of RNA-based therapies. Only continued testing and developing will determine how effective this method ultimately will be for treating genetic disorders.