Revolutionary Israeli Genetic Treatment for Cystic Fibrosis to Begin Human Trials

Revolutionary Israeli Genetic Treatment for Cystic Fibrosis to Begin Human Trials

SpliSense “addresses the underlying genetic cause” of cystic fibrosis, offering hope for restored lung function.

By Pesach Benson, TPS

SpliSense, a Jerusalem-based biopharmaceutical company announced on Wednesday that it began human trials of a medicine for treating cystic fibrosis.

Cystic fibrosis is a genetic condition which creates a buildup of mucus in the lungs, pancreas and the digestive system, resulting in malnutrition, breathing difficulties, frequent lung and sinus infections, jaundice and even male infertility.

An estimated 105,000 people around the world have been diagnosed with CF, according to the Cystic Fibrosis Foundation. The American Lung Association estimates that 1 in 30 Americans is a carrier of CF.

“Currently available treatments focus on treating the symptoms of the disease, while we address the underlying genetic cause of the disease, thereby offering hope of restoring the defective protein and generating adequate lung function in patients suffering from CF, with the benefit of an easy-to-use and less frequent treatment approach,” said SpliSense CEO Gili Hart, PhD.

“This program is our first-in-human and will serve as proof of concept of our pulmonary platform for our additional promising programs, including muco-obstructive diseases and IPF, the latter of which is expected to enter the clinic next year,” added Dr. Hart.

The cystic fibrosis disorder originates from various mutations in the CFTR gene, which is responsible for the production of the CFTR protein, a chloride channel expressed in the lungs as well as in other tissues. Current treatments do not offer a cure, or support all patients with CF.

CF patients carrying a genetic mutation called 3849 +10 Kb C->T mutation have no specific approved treatment, but SpliSense’s lead product, SPL84, has shown to have completely restored CFTR activity, suggesting potential cure for these patients, Dr. Hart explained.

People with CF also have a higher risk for osteoporosis, diabetes and liver problems.

The disorder can be identified with prenatal testing and blood tests for adults.

Blood and saliva tests can also detect if adults are carriers of CF.