U.K. green lights study of pioneering gene therapy against cystic fibrosis

Posted under Blog, Clinical Trials, Companies, cystic fibrosis, Diagnostics, drug delivery systems, Funding, gene therapy, genetic mutation, Medical Devices, Medical Supply, Pharmaceuticals, Startups, Universities, Videos by Ryan McBride

Researchers in the U.K. plan to launch a mid-stage clinical trial this month for an inhaled gene therapy to combat cystic fibrosis (CF). The U.K.'s National Institute for Health Research and Medical Research Council have ponied up £3.1 million ($4.9 million) in funding to get the trial off the ground.

U.K. Cystic Fibrosis Gene Therapy Consortium, a nonprofit research group, will coordinate the 130-patient trial, which the consortium called the largest of its kind and will include patients aged 12 and up. Patients in the double-blind, placebo-controlled study will get a dose of the gene therapy or a placebo once per month over the course of a year, with results expected in spring 2014, according to the group's release.

Cystic fibrosis is a genetic disease that causes a buildup of thick, sticky mucus in the lungs and leads to respiratory infections and other debilitating symptoms. In the U.K. the only available treatments address only the symptoms of the chronic condition, and CF patients have an average life expectancy of 41 years, the PharmaTimes reports. The consortium has been working for more than a decade on their gene therapy, which is inhaled into the lungs to deliver molecules of DNA into cells to replace the defective gene that causes CF.

The Medical Research Council has also come up with £1.2 million ($1.9 million) to fund research of a modified virus as a vehicle to provide improved delivery of the gene therapy.

CF patients are forced to take a battery of treatments to control the symptoms of their disease, yet in recent years, companies such as Vertex Pharmaceuticals ($VRTX) have advanced treatments that address the root genetic cause of the disease rather than just the symptoms. The FDA in January stamped an approval on Vertex's Kalydeco, a targeted drug for about 4% of CF patients in the U.S. who have the G551D mutation. And the drug developer is now working on other targeted approaches to address the root cause of the disease.

- get the consortium's release
- read the PharmaTimes article

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MIT scientists tout first human data on drug-delivery chips

Posted under Blog, Companies, Diagnostics, drug delivery systems, Eli Lilly, Funding, Medical Devices, Medical Supply, MicroChips, MIT, Pharmaceuticals, Startups, Universities, Videos by Ryan McBride

After overcoming bumps in the road and millions of dollars in development costs, venture-backed startup MicroCHIPS has evidence that its wirelessly controlled implant can release drugs into humans. And the company's MIT founders believe the technology could overcome major hurdles to getting drugs into patients properly.

The Waltham, MA-based company reported its implant delivered a generic version of Eli Lilly's ($LLY) Forteo in 7 women with osteoporosis without any adverse immune reactions for a month. The implant was inserted into the women in a half-hour procedure, and the company believes the data show promise that the implant could last for up to 12 months. It's a major step forward for the company and its technology, which helps patients avoid repeated needle injections of drugs and doesn't require them to remember to take their meds.

“This is for drug therapies that can't be delivered orally, so we're replacing multiple daily injections with a single device under the skin,” MIT's Michael Cima, a co-founder of MicroCHIPS, told Bloomberg.

The startup, which Xconomy reports has raised $33 million through three rounds of venture capital, faces more tests before its drug-delivery implant reaches the market. And, as The In Vivo Blog points out, there were some technical glitches in the osteoporosis trial that will need to be worked out before the delivery system is ready for prime time.

- here's the release
- check out Bloomberg's article
- see The In Vivo Blog item
- and Xconomy's report

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