Advances in research and medicine have improved the life expectancy for people with cystic fibrosis. But this doesn’t mean that we should stop searching for a cure. Most likely, the cure lies in gene therapy: a process that allows scientists to manipulate gene expression. Cystic fibrosis results from CFTR gene mutations. Normally, CFTR regulates the movement of salt in the body. When this gene isn’t working properly, mucus becomes thick and sticky, accumulating in the body, clogging airways, preventing digestive enzyme release, and causing multiple other health issues.

Drugs.com provided an update on the Phase 1 CORAL-1/US clinical trial which is evaluating KB407 for cystic fibrosis. KB407 is an investigational, redosable gene therapy that delivers two functioning copies of CFTR directly to the lungs through nebulation. It received Orphan Drug designation from the U.S. Food and Drug Administration.

Ideally, this treatment could be used to treat a wide variety of patients regardless of their specific gene mutation. Recently, the first participant within the CORAL-1/US trial received a dose of KB407.

Ultimately, the trial will enroll 20 participants with cystic fibrosis. These individuals will be split into three separate cohorts for the course of the study. Each cohort will receive different administrations of the drug. The study hopes to understand how safe KB407 is, but also how well it addresses the symptoms and manifestations of cystic fibrosis.

The Details of Cystic Fibrosis (CF)

Cystic fibrosis is a rare genetic condition which can cause progressive (worsening) damage to your respiratory and digestive systems. This condition is more common in Caucasian infants, while rarer in other communities. For example, 1 in every 2,500-3,500 Caucasian newborns in the United States has cystic fibrosis, but only 1 in every 31,000 Asian Americans does. Current treatment options include CFTR modulators, bronchodilators, anti-inflammatory medications, antibiotics, chest physical therapy, vest therapy, and exercise therapy. However, there are some people whose condition remains refractory (does not respond well to treatment).

Symptoms relating to cystic fibrosis can include:

• A chronic cough that may be dry or produce mucous
• Persistent wheezing
• Exercise intolerance
• Fatigue
• Nasal polyps
• Very salty-tasting skin
• Abdominal or chest pain
• Frequent lung or sinus infections
• Slowed growth and poor weight gain
• Stuffy nose
• Constipation or diarrhea
• Infertility in males