First Success for Imperial Innovations’ Gene Therapy in Cystic Fibrosis

Good news for patients suffering from cystic fibrosis! Imperial Innovations‘ patented gene therapy could improve the patients’ breathing provoking any side effects. An innovation that could help fulfill this unmet medical need.

Cystic fibrosis is the most common deadly genetic disease in Europe. Gene deficiency leads to thickening mucus that obstructs lungs and results in frequent chest infections. This condition is usually treated with antibiotics and symptom weakening drugs like Mucus-thinning therapeutics or bronchodilators. After 25 years of research, a way to introduce compensating plasmid DNA for the deficient gene CFTR in lung cells has finally been found. And what a challenge it was, as lung cells are highly protected from invading foreign substances. UK Cystic Fibrosis Gene Therapy Consortium (CFGTC), which developed the patented technology, owned by Imperial Innovation, has shown, for the first time ever that frequent gene therapy can have a positive effect on cystic fibrosis.

The clinical trial was conducted on 136 patients aged 12 and above, all inhaled a monthly dose of the plasmid DNA or a placebo for an entire year. Study leader Professor Eric Alton stated: “Patients who received the gene therapy showed a significant, if modest, benefit in tests of lung function compared with the placebo group; there were no safety concerns.” However, he also added that this trial represents the mere foundation for further testing, since patients responded inconsistently to the drug. To improve the impact of the treatment, the team behind the study now wants to pursue trials with “higher, more frequently doses, as well as combinations with other treatments.”

Meanwhile, Imperial Innovations is looking for supporting partnerships, enabling follow-up trials to improve patients’ life conditions suffering from cystic fibrosis.


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