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U.K. CF gene therapy trials to test inhaled and viral delivery options

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A cadre of U.K. researchers will begin a mid-stage trial later this month for a cystic fibrosis gene therapy delivered to the lungs with a nebulizer. They'll also begin a laboratory trial using a more advanced version of the same drug designed to ride in a modified virus to reach its target.

These latest milestones hit after a decade-long quest by the U.K. Cystic Fibrosis Gene Therapy Consortium to deliver a groundbreaking replacement gene therapy in order to beat back the disease. Cystic fibrosis, which is inherited, is lethal and eventually destroys the lungs with a thick, sticky mucus. Most patients live an average of just 41 years, according to a PharmaTimes story on the research news. And many existing treatments focus on CF-related respiratory symptoms and not the root genetic cause.

As FierceBiotech's coverage of this news notes, companies including Vertex Pharmaceuticals ($VRTX) either have or are pursuing targeted, genetic-focused treatments for the disease, but Vertex's newly-approved drug, Kalydeco, only helps about 4% of cystic fibrosis patients with a certain gene mutation.

The Consortium's Phase II clinical trial using a nebulizer for delivery will involve about 130 patients ages 12 and older. Funded by a $4.9 million grant, this will be a double-blind, placebo-controlled trial set with a dose once each month over a year with results due by 2014. More than 30 patients received similar dosing in previous studies. Patients inhale the treatment through a nebulizer. Once in the respiratory system, fat globules wrapped around DNA molecules carry the replacement gene to its target, the group explains.

Scientists will test the modified virus/treatment in the lab, with the idea of eventually developing a better way to deliver their drug. They'll be backed by a $1.9 million grant.

The Imperial College London, the Universities of Oxford and Edinburgh, Royal Brompton & Harefield NHS Foundation Trust and the NHS Lothian are the Consortium's members.

- here's the PharmaTimes article
- check out the release

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