Gene Therapy Shields Against Deadly Flu

Updated May 29, 2013, 6:53 p.m. ET

Gene Therapy Shields Against Deadly Flu


Researchers said Wednesday they have developed a gene-therapy technique that in animal studies provided broad protection against flu viruses linked to deadly human pandemics.

If verified in people, the approach could become an important tool in the effort to ward off viral infections such as H5N1 that originate in animals and aren’t affected by human vaccines.

Outbreaks of H5N1, a bird flu, have caused 374 deaths around the globe in the past decade, according to the World Health Organization, and provoked concern among public-health officials about the potential for far deadlier pandemics.The advance, described in a study in the journal Science Translational Medicine, involved delivering certain genes into the nasal lining of mice and ferrets. There, the genes produced potent virus-fighting antibodies that provided complete protection against infection from lethal doses of H5N1 and another bird flu called H1N1, said James Wilson, director of the gene-therapy program at the University of Pennsylvania, who led the research.

The technique also protected the animals against a strain of the H1N1 virus that caused the 1918 flu pandemic.


Human deaths in past decade from H5N1 bird flu, a pandemic risk

The accomplishment is an “important proof of concept,” said Anthony Fauci, director of the National Institute of Allergy and Infectious Diseases, who wasn’t involved with the research. He said it would take additional research to determine whether it has similar efficacy in people and to establish its safety.

With an “antibody that neutralizes a whole range of influenza, including potentially pandemic influenza,” said Dr. Fauci, “you would already have in your hand something you can administer to people, rather than having to isolate the virus and starting making a vaccine.”

The strategy also could be used to protect high-risk populations such as the elderly against seasonal flu, Dr. Wilson said.

Infectious-disease experts long have yearned for a universal vaccine that would protect against a variety of flu viruses, especially those with the potential for triggering a global pandemic. But current technology requires developing vaccines against individual strains, which change each year.

It typically takes months to identify strains of seasonal flu viruses and develop and make enough doses to mount public health-efforts against them. Unlike seasonal flu, viruses that originate in animals can’t be predicted, so they can spread for months before any vaccine might be developed.

The gene-therapy approach technically isn’t a vaccine, Dr. Wilson said. “The technology isn’t trying to stimulate the immune system to generate antibodies” in response to an infection, he said. “It simply engineers the cells to make antibodies,” effectively establishing tiny factories in the nasal passages to make them.

If the new approach proves effective in people, it would be available essentially off the shelf, Dr. Wilson said.

Dr. Wilson said nasal administration of the treatment was selected because the nose is the primary entry point for respiratory viruses. Avoiding delivering the therapy systemically potentially would reduce safety worries because cells turn over regularly in the nasal passages.

The advance was made possible in part by the recent discovery of what scientists call broadly neutralizing antibodies that are effective against a variety of strains.

In the technique, genes for the antibodies are loaded into an inactivated virus called adeno-associated virus-9, or AAV-9. Such “vectors”—delivery vehicles for gene therapy—are a focus of Dr. Wilson’s research.

Supporters of the research included the National Institutes of Health, the Public Health Agency of Canada and ReGenX Biosciences LLC. Dr. Wilson is a founder and holds equity in ReGenX, and holds a patent related to adeno-associated viruses.

About bambooinnovator
Kee Koon Boon (“KB”) is the co-founder and director of HERO Investment Management which provides specialized fund management and investment advisory services to the ARCHEA Asia HERO Innovators Fund (, the only Asian SMID-cap tech-focused fund in the industry. KB is an internationally featured investor rooted in the principles of value investing for over a decade as a fund manager and analyst in the Asian capital markets who started his career at a boutique hedge fund in Singapore where he was with the firm since 2002 and was also part of the core investment committee in significantly outperforming the index in the 10-year-plus-old flagship Asian fund. He was also the portfolio manager for Asia-Pacific equities at Korea’s largest mutual fund company. Prior to setting up the H.E.R.O. Innovators Fund, KB was the Chief Investment Officer & CEO of a Singapore Registered Fund Management Company (RFMC) where he is responsible for listed Asian equity investments. KB had taught accounting at the Singapore Management University (SMU) as a faculty member and also pioneered the 15-week course on Accounting Fraud in Asia as an official module at SMU. KB remains grateful and honored to be invited by Singapore’s financial regulator Monetary Authority of Singapore (MAS) to present to their top management team about implementing a world’s first fact-based forward-looking fraud detection framework to bring about benefits for the capital markets in Singapore and for the public and investment community. KB also served the community in sharing his insights in writing articles about value investing and corporate governance in the media that include Business Times, Straits Times, Jakarta Post, Manual of Ideas, Investopedia, TedXWallStreet. He had also presented in top investment, banking and finance conferences in America, Italy, Sydney, Cape Town, HK, China. He has trained CEOs, entrepreneurs, CFOs, management executives in business strategy & business model innovation in Singapore, HK and China.

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