BOSTON – Governments and nonprofits have hammered for years on the need to find cures or treatments for neglected tropical diseases and other conditions endemic to poverty, such as malaria and tuberculosis (TB).

Some have put their money where their mouth is. The U.S. government, for instance, is the leading funder of research and development in 26 of the 30 most neglected diseases and conditions affecting the developing world, including HIV/AIDS, TB and malaria. Meanwhile, with the Seattle-based Gates Foundation providing nearly two-thirds of its funding, the nonprofit Institute for OneWorld Health is pursuing promising drug candidates discarded by for-profit companies for lack of a viable market. (See BioWorld Today, Dec. 17, 2010, and May 24, 2012.)

Now, biotech and pharmaceutical companies are beginning to participate more fully by lending their expertise and intellectual property (IP) under the auspices of the World Intellectual Property Organization (WIPO). The nascent effort, dubbed WIPO Re:Search, is picking up steam, according to a panel at BIO 2012.

WIPO Re:Search (www.wiporesearch.org) is designed to facilitate R&D partnerships among nonprofits, for-profits, academic researchers and government agencies in a systematic way to "think outside the box" in repurposing IP. Launched in October 2011 by WIPO in conjunction with BIO Ventures for Global Health and several drug developers, WIPO Re:Search is developing a searchable, public database of IP on pharmaceutical compounds, technologies and resources that can be applied to neglected diseases identified by the World Health Organization (WHO), plus TB and malaria.

As a group, those conditions represent a greater health burden than cerebrovascular disease, according to Don Joseph, CEO of BIO Ventures. Although the greatest impact occurs in the developing world, individuals across the globe are affected through travel, immigration, military and other exposure.

The multilateral, voluntary consortium includes three categories of participants. Providers, such as drug and vaccine developers, share their technology, patents, services and intellectual know-how. Users, such as universities, agree to license assets in the database for uses related to neglected diseases and to submit to a strict set of guiding principles. Supporters drive the partnerships through financial and other contributions. For example, the Association of University Technology Managers participates as a supporter, along with the Patent and Trademark Office.

Some organizations participate in multiple categories – for example, as users and supporters.

Though still in its infancy, the initiative has attracted 49 partners, including eight drug developers. WIPO serves as the secretariat, with BIO Ventures as administrator of the information clearinghouse and WHO as the technical advisor.

Because IP has traditionally created a huge barrier to research in neglected diseases, the involvement of drug developers is key to the effort's success. The companies agree to grant users royalty-free licenses to their IP for R&D, anywhere in the world, involving products, technologies or services used solely to address public health needs related to neglected diseases.

Providers also agree to grant users royalty-free licenses to the IP anywhere in the world for the manufacturing, import/export or sale and distribution of resulting products.

Researchers at the university and nonprofit users access data on compounds and patents through the WIPO Re:Search database. When they identify potential leads for vaccines, therapeutics or diagnostics in a neglected disease, they can reach out through the partnership to establish a relationship with the provider.

Each collaboration requires scientific and product development expertise as well as data, materials and IP, Joseph said. At least 15 "conversations" are under way between providers and users in specific targets and initiatives.

Pfizer Inc., of New York, was one of the first pharmas to join WIPO Re:Search and is already engaged in several of those efforts, according to Roy Waldron, the company's senior vice president, associate general counsel and chief IP counsel. The effort is a win-win, he said, because big pharma's traditional drug development engine has been turned on its head at the same time that pipelines are running dry. Expanding the scope of research on existing discoveries – many abandoned as drug candidates in other indications – offers the potential not only to develop treatments for neglected diseases but also potentially to identify new commercial targets, such as orphan drugs.

"The next level of open innovation is about information," Waldron said. "There's a lot we don't know about the mechanisms of disease. This is a start to share information so we can accelerate drug development."

Of course, sharing proprietary knowledge requires a sense of trust, especially among fiercely competitive drug developers. WIPO Re:Search's open innovation structure "is consistent with protecting robust IP," Waldron said. To date, Pfizer has shared animal health screening data and IP for vaccine technologies.

Alnylam Pharmaceuticals Inc., of Cambridge, Mass., has gone a step further. The company is sufficiently comfortable with the license structure that it is sharing all of its technology and 1,500 patents with the consortium, according to Steve Bossone, Alnylam's vice president of intellectual property.

"Being a small company with 120 employees and limited resources, it's difficult for us to participate actively, but we have a $750 million investment in our technology that we can share," Bossone said.

Alnylam was among the first providers, along with GlaxoSmithKline plc, of London, to join the "precursor patent pool," sharing patents that cover fundamental IP, such as siRNA; chemistry and delivery IP; and target-specific IP.

Researchers evaluate the IP for lead selection and optimization as well as delivery mechanisms, formulation, pharmacokinetics and biodistribution. Already, the process has helped to identify or validate malaria host factors, Bossone said, leading to a development collaboration in South Africa.

Peter Ruminski, a former Pfizer executive who now runs the Center for World Health and Medicine at St. Louis University, is excited about the prospects for bringing effective, affordable therapies to neglected diseases, rare diseases and other unmet medical needs.

"A lot of [product development partnerships] are focused on the short-term impact of disease – new formulations, combinations, mass distribution or modifications – but we need a deeper medicine chest with new mechanisms of action," he said.