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Startup focuses on helping the poor
By Helen Branswell
STAT

There’s a new biotech in town. And it doesn’t care about making money.

The Bill and Melinda Gates Foundation has spun out a nonprofit biotech offspring, the Gates Foundation Medical Research Institute. With funding of $273 million for its first four years, the organization is in an enviable position.

“We don’t have to worry about revenue, return on investment. Our bottom line is lives saved. So it’s a pretty exciting place to be,’’ Dr. Penny Heaton, the institute’s CEO, said.

Though the institute opened its doors — in temporary space in Cambridge — in early January, Heaton introduced it to the biotech world on Thursday at the BIO International Convention in Boston.

The institute’s focus is on diseases that disproportionately affect the poor: malaria, tuberculosis, and enteric diseases. The latter are diseases — caused by viruses, bacteria, or parasites — that attack the digestive system. They can cause life-threatening, even fatal bouts of diarrhea, disrupt nutrient absorption, and stunt development.

“Our mission is to develop products that will enable the end of diarrheal disease deaths, eradication of malaria . . . and to accelerate the end of the TB epidemic,’’ Heaton said.

While the institute will have some laboratory space, its staff — which Heaton expects to stretch to about 120 people within three years — won’t be focused on discovery. Instead the plan is to find ideas developed by academic labs or held in the portfolios of other biotech firms and essentially serve as a midwife, seeing projects through the treacherous middle stage of development as far as the proof-of-concept phase. Then the institute will look for commercial partners to take over.

Freed from the need to generate profits, the institute isn’t interested in owning intellectual property. But any products it passes on to commercial partners will come with strings attached — commitments that the products will be made available at affordable prices, in the needed volume, and within a specified time frame.

Heaton, who had been head of vaccine development in the Gates Foundation’s Global Health Program, said the idea for the new research institute was sparked by the realization that the development of tools for the targeted disease was really languishing. During the foundation’s annual strategy reviews, it was clear that some fields were making far greater strides.

“But then you turn and you looked at . . . TB, still 1.7 million deaths every year. You look at malaria, nearly 500,000 deaths every year. You look at enteric disease and while the rotavirus vaccines have done amazing things, we still have 500,000 deaths from enteric diseases every year in children under 5,’’ she noted. “And so we were thinking about: What can we do to start to accelerate finding solutions for these area?’’

Her plan is to break down silos — trying to leverage what fields like HIV vaccine research or the efforts to harness the immune system to fight cancers have discovered that can be applied to gain ground on these neglected challenges.

Heaton’s team already has ideas. It’s exploring whether using monoclonal antibodies in a once-annual shot could drive down rates of malaria. At present, campaigns to mass administer anti-malarial drugs are mounted before the rainy seasons. Would adding a concentrated shot of monoclonal antibodies stretch three months of protection out to six?

And then there’s a tantalizing finding that could pave the way to making big gains against tuberculosis, a massive problem globally.

There is a TB vaccine, called Bacille Calmette-Guerin, or BCG. It is given early in life, but the protection it offers wanes.

In developing countries, it’s known that young adults really drive transmission of the bacterium. A study conducted in South Africa tested an experimental tuberculosis vaccine in adolescents. Participants in one of the arms of the study got a shot of BCG. (All of the participants had received BCG in early childhood, so that new shot was a booster dose.)

To everyone’s surprise, the researchers saw a 45 percent reduction in sustained infection rates among the adolescents who got BCG vaccine. That raises the possibility that giving a booster dose of BCG in adolescence might take a big chip out of the global TB problem.

“Is it as simple as [giving] a boost in these countries with high prevalence that could help really accelerate the end of the TB epidemic?’’ Heaton wondered.

The Gates Foundation Medical Research Institute plans to try to find out.

Helen Branswell can be reached at helen.branswell@statnews.com. Follow her on Twitter @HelenBranswell. Follow Stat on Twitter: @statnews.