RESEARCH TRIANGLE PARK – About 7,000 rare or “orphan” diseases have been identified, many of them genetic.

The Invest in Cures & Rare Disease Forum, scheduled for September 25 and 26, will showcase the R&D phase and early-stage commercialization activity that North Carolina brings to this large unmet medical need, while bringing together potential investors and collaborators to take this work to the next level.

The combination event, to be held at the North Carolina Biotechnology Center in Research Triangle Park, is the result of a collaboration involving NCBiotechLaunchBioUNC Catalyst for Rare Diseases and the UNC/NC State Rare Disease InfoHub.

Day 1, presented by LaunchBio, features disease foundations for discussion of their investment philosophies, fund and deal structures and how they are using scientific knowledge to validate early-stage technologies and syndicate with other investors. Three sessions will focus respectively on: cancer research, translational research and the role of rare disease foundations.

Day 2, presented by UNC Catalyst for Rare Diseases, Rare Disease InfoHub and NCBiotech, features a highly interactive forum for industry, academia and the investment community to consider the research, clinical, regulatory and economic challenges and opportunities specific to advancing effective and affordable therapies.

It will also include a panel discussion led by Alex Tropsha, Ph.D., director of the UNC/NC State Rare Disease InfoHub, to consider complementary strategies for improving outcomes in rare diseases. These include the advanced computing techniques of the InfoHub to search, organize and present information and data related to rare disorders from multiple resources and to identify relationships that will facilitate development of innovative treatments. The InfoHub was funded by the University of North Carolina General Administration in 2017.

Networking opportunities throughout the two-day event

Throughout the event there will be rich networking opportunities for all who work or have an interest in rare disease – during breaks both days and a networking lunch the second day.

“It’s important to remind people nationally that this important research and technology development is something we do well here,” said Joan Siefert Rose, CEO of LaunchBio, the nonprofit programming arm of BioLabs, Inc. LaunchBio’s mission is to connect entrepreneurs with knowledge, capital and talent across the nation’s leading life science hubs. The organization has presented an Invest in Cures program in other hubs tailored to the specific strengths and needs of each region, so has a bird’s-eye view of how the various hubs stack up against one another competitively and have the potential to collaborate in the development of new therapies through science and investment capital.

Recently, the Raleigh-Durham  metro area was ranked the nation’s fourth-leading life science base among major metro hubs, behind Greater Boston, the San Francisco Bay Area and San Diego. The 2019 Life Sciences Outlook annual report, published by Chicago-based Jones Lang LaSalle, a global financial and professional services firm that specializes in commercial real estate services and investment management, based the rankings on life science employment concentration and growth, company concentration, venture capital funding, NIH funding and patents.

Anthony Hickey, Ph.D., director of UNC Catalyst for Rare Diseases within the UNC Eshelman School of Pharmacy since January of this year, is counting on attendees to bring questions for the panelists during the second day’s Rare Disease Forum.

Seeking audience participation, novel thinking

“What we’re looking for is to elevate everybody’s awareness that rare disease is a serious unmet medical need and then to gather what we learn from the experience being there that day to guide us as we go forward in our efforts,” said Hickey. “It’s going to be the gaps between the words from the speakers and the questions from the audience that will drive novel thinking.”

UNC Catalyst for Rare Diseases is a research group committed to understanding the functional consequences of genetic mutations. It fosters collaboration with patient organizations, partners with foundations to advocate and raise funds, operates as an open access science model and trains post-doctoral fellows.

Vivian Doelling, Ph.D., vice president of Emerging Company Development at NCBiotech, an organizer of the event, noted that while this is the second year offering LaunchBio’s Invest in Cures program, this is the first time offering the two-day combined event.

“The idea is to bring foundations, academics, companies and other rare disease experts together,” said Doelling, who is looking forward to the potential the event offers for investors to learn more about North Carolina R&D and early-stage commercialization activities in rare diseases.

Go to this link for each day’s schedule, list of speakers and to register.

(C) N.C. Biotech Center