ProNAi Therapeutics Advances Gene-Silencing Cancer Drug, Marina Biotech Provides Delivery Package

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recognize our idea of silencing genes,” Sooch said. “We’ve actually been working as long as they [RNAi groups] have with probably one-one hundredth of the research dollars.”

ProNAi is developing a drug that uses short pieces of DNA to silence disease-causing genes. A key feature of ProNAi’s treatments is that they are intended to go directly to a region of DNA in cells to block the activity of disease-related genes, while RNA-interference drugs target genetic messenger molecules (or messenger RNA) that play a key role in producing disease proteins. If that doesn’t make sense, think of the firm’s drugs as striking at a different point along a pathway of disease-gene activity than RNA-interference drugs.

While more money has been invested in RNAi than DNAi, both approaches share the same problems of how to deliver the gene-silencing treatments to cells deep in the body. To tackle this delivery challenge, ProNAi has adopted liposomal-particle delivery technology from Marina Biotech. (The Seattle-area biotech, formerly known as MDRNA, acquired the technology in July from Germany-based Novosom for $5 million in Marina common stock.) The drug-delivery technology uses fatty molecules to form liposomes. Those molecules will encapsulate ProNAi’s drug and hopefully carry them into cells, where the drug can travel into the nucleus and silence a tumor gene called Bcl-2. The gene is believed to be a culprit in tumor cell survival, and it’s been shown that it’s overactive in patients with non-Hodgkin’s lymphoma, breast tumors, and other cancers.

The Phase I trial is taking place at South Texas Accelerated Research Therapeutics in San Antonio. ProNAi’s Forgey said that the trial is expected to enroll 20 to 25 patients, and full results from the study are likely to come in the first quarter of 2012.

Meantime, Forgey has nearly finished raising $1 million to pay for the early-stage trial. He declined to disclose who is investing in the financing. Yet the firm’s previous investors include Amherst Fund, Apjohn Ventures, the Michigan Economic Development Corporation, and Sigvion Ventures.

Depending on the success of the firm’s clinical trial, ProNAi might start to attract the kind of attention that Alnylam, Merck-Sirna, and other gene-silencing firms have long enjoyed. If it shows promise, ProNAi’s DNAi won’t be such a quiet cousin to its RNA counterpart.

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