LaunchBio, UNC’s Catalyst for Rare Diseases, the Rare Disease InfoHub and NCBiotech are partnering to hold a two-day forum focused on nonprofit disease foundation investments and the development of rare disease therapeutics on September 25 and September 26. 

LaunchBio’s Invest in Cures, explores how disease foundations are accelerating the pace of commercialization of new therapies and cures for patients. On Wednesday, September 25, LaunchBio welcomes disease foundations to discuss their investment philosophies, explain their fund and deal structures, and describe how they are using deep scientific knowledge to validate early stage technologies and syndicate with other investors.

UNC’s Catalyst for Rare Diseases and the Rare Disease InfoHub are presenting Rare Disease Forum on Thursday, September 26, for stakeholders to discuss the status and potential changes required to overcome the research, clinical, regulatory and economic barriers to effective treatment of rare diseases.


These events represent a significant opportunity for foundations, patient groups, investors and companies to share recent advances and best practices in the emerging field of disease foundation sponsored investing and the ever-evolving field of rare disease drug development. Time for informal networking will be provided throughout both days of the events.

Invited Partnering

Concurrent with the two-day forum, NCBiotech and LaunchBio will be working with the attending disease foundations and investors to arrange invited meetings with companies and entrepreneurs as one-on-one interactions to discuss collaborations and/or funding opportunities.


Presenting Partners


See Day 1 and Day 2 tabs for day-specific information and agendas.



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Day 1: Invest in Cures - $35

Day 2: Rare Disease Forum - $35

Two Day Registration - $60


Day 1 - Invest in Cures

Invest in Cures

Invest in Cures Logo

Invest in Cures explores how disease foundations are accelerating the pace of commercialization of new therapies and cures for patients. LaunchBio welcomes disease foundations to discuss their investment philosophies, explain their fund and deal structures, and describe how they are using scientific knowledge to validate early stage technologies and syndicate with other investors.

The day-long forum combines main stage sessions and panel discussions with intimate gatherings and high-level networking.



3 - 3:45 p.m. | Session 1: Commitment to Cancer Research

Moderator - Derrick Rowe, JD, Ph.D., Wilson Sonsini Goodrich & Rosati

  • John Hallberg, CEO, Children's Cancer Research Fund
  • Annette Baker. Ph.D., President & CSO, Children's Tumor Foundation
  • David Heenan, Director, Operations, BrightEdge Ventures

3:45 - 4 p.m. | Networking Break

4 - 4:45 p.m. | Session 2: The Translational Research Report

  • Kirstie Keller, Ph.D., Milken Institute, Center for Strategic Philanthropy

4:45 - 5:15 p.m. | Networking Break

5:15 - 6 p.m. | Session 3: Foundations, Research and Rare Diseases

Moderator - TBD

  • Sonya Dumanis, Ph.D., Senior Director Innovation, Epilepsy Foundation
  • Amy Gray, CEO, Charcot-Marie Tooth Association
  • Lewis Hsu, MD, Vice Chief Medical Officer, Sickle Cell Disease Assoc. of America
  • Rich Horgan, Founder and Chairman, Cure Rare Disease


Sponsored by

LanuchBio Sponsor logos


Presenting Sponsors

LaunchBio Presenting Partners logos

Day 2 - Rare Disease Forum

Rare Disease Forum

rare disease forum graphic

UNC’s Catalyst for Rare Diseases and the Rare Disease InfoHub has joined NCBiotech to assemble an accomplished group of confirmed speakers and panelists to present interdisciplinary perspectives from bench to bedside probing the barriers to therapeutic development to address the unmet needs of patients within the rare disease community. 

This will be a highly interactive forum for industry, academia, and the investment community to illuminate research, clinical, regulatory and economic challenges and opportunities specific to rapidly advancing effective and affordable therapies for these defined patient groups.

Partnering & Networking

Rare Disease Forum represents a significant opportunity for foundations, patient groups, investors and companies to share recent advances and best practices in the ever-evolving field of rare disease drug development. 


8:30 a.m. | Registration

8:45 a.m. | Welcome & Opening Remarks

9 - 10:45 a.m. | Session 1: The emerging ecosystem for rare disease research and development

Session Chair - Anthony Hickey, Ph.D., UNC Catalyst for Rare Disease

  • David Ridley, Ph.D., Duke, Health Economist 
  • Sean Ekins, Ph.D., Collaborations Pharmaceuticals
  • Kathleen Miller, Ph.D., US FDA, Orphan Drug Product Development

10:45 - 11 a.m. | Networking Break

11 a.m. - 12 p.m. | Panel: Barriers to rapid development and delivery of affordable therapeutics for rare diseases

  • Sharon King, Taylor's Tale, Batten Disease Foundation
  • Andy Holt, Asklepios BioPharmaceutical, Inc.
  • Joan Levy, Ph.D., Chordoma Foundation
  • Fred Porter, Ph.D., Adrenas Therapeutics
  • Kathleen Miller, Ph.D., US FDA, Orphan Drug Product Development

12 - 1 p.m. | Networking Lunch

1 - 2:45 p.m. | Session 2: Complementary strategies for improving outcomes in rare diseases

Session Chair - Alex Tropsha, Director, UNC/NC State InfoHub

  • Lisa Gehtland, MD, RTI International
  • Jim Hagood, MD, Pediatrics,UNC School of Medicine
  • Rada Chirkova, Ph.D., Computer Science, NC State

2:45 p.m. | Closing Remarks

Sponsored by

UNC Catalyst logo
Infohub logo
NCBiotech logo

Speaker Bios

Day 1 - Invest in Cures


Annette Bakker Photo


Annette Bakker, Ph.D.
President & CSO, Children's Tumor Foundation

In her role at the Childrens Tumor Foundation, Dr. Bakker and her team design and execute innovative business models to accelerate the path from basic discovery to clinical benefit for NF patients, develop partnerships with pharmaceutical and biotech industries, and attract new scientists and funders to the NF field.


Sonya Dumanis Photo


Sonya Dumanis, Ph.D.
​​​​​​Senior Director of Innovation, Epilepsy Foundation

Sonya Dumanis oversees the Epilepsy Foundation Innovation Program, including the Epilepsy Innovation Institute (Ei2), Epilepsy Therapy Project and Targeted Research Programs, and the Next Generation Scientist Programs. Prior to joining the Epilepsy Foundation, Dr. Dumanis worked as a senior associate at the Milken Institute Center for Strategic Philanthropy.


Amy Gray Photo


Amy Gray
CEO, Charcot-Marie Tooth Association

Amy Gray is the Chief Executive Officer of the Charcot-Marie-Tooth Association (CMTA), the leading patient advocacy organization driving CMT research. In her role as CEO, she has worked closely with the CMTA’s scientific, therapeutic, and clinical expert boards to drive the CMTA’s Strategy to Accelerate Research (STAR) which has led to more than 20 new pharmaceutical, biotechnology and industry partners in the last two years. Amy has two decades of management experience in national voluntary health agencies, combined with a strong understanding of mission-driven organizations.


John Hallberg Photo


John Hallberg
CEO, Children's Cancer Research Fund

John Hallberg has served as Chief Executive Officer of Children’s Cancer Research Fund since late 2006. Children’s Cancer Research Fund is one of the nation’s largest private funders of pediatric cancer research, and since its inception in 1981 has provided more than $175 million for research, education and quality-of-life programs dedicated to better outcomes for childhood cancer patients.


David Heenan photo


David Heenan
Director, Operations, BrightEdge Ventures

David began his career working on trade, security, and nonproliferation policy issues for the Chinese Ministry of Commerce in Beijing, China and then at Argonne National Laboratory in Chicago, IL. He then transitioned over to the political side, working on economic development for the Governor of Georgia, before joining a congressional office and running digital media for a US Senate campaign. After the campaign ended he joined the Editorial Staff of the Journal on Preventing Chronic Disease published by the Center for Disease Control and Prevention (CDC) in Atlanta, GA.


Rich Horgan Photo


Rich Horgan
Founder and Chairman, Cure Rare Disease

Rich Horgan is the Founder and President of Cure Rare Disease. Rich is also a Blavatnik Fellow at Harvard Business School with a deep passion for Duchenne muscular dystrophy (DMD) and other rare diseases. With a younger brother impacted by the disease, Rich has a strong interest in accelerating promising treatments for the disease. He formed a collaboration with world-class researchers and clinicians to pioneer the development of customized therapies for Duchenne and other rare diseases.


Lewis Hsu Photo


Lewis Hsu, M.D.
Vice Chief Medical Officer, Sickle Cell Disease Assoc. of America

Lewis Hsu, M.D., Ph.D. is a pediatric hematologist dedicated to finding more cures for sickle cell disease, and improving treatment and education until cures can be found. His clinical and translational research in sickle cell disease include the landmark STOP study of transfusion to reduce stroke risk, bone marrow transplantation for children with severe sickle cell disease, and clinical research on pain management candidate agents. Dr. Hsu has 23 years of experience in leadership of some of the largest pediatric sickle cell programs in the country. He became Vice Chief Medical Officer of Sickle Cell Disease Association of America in Oct 2016.


Kristie Keller Photo


Kristie Keller, Ph.D.
Sr. Associate, Milken Institute, Center for Strategic Philanthropy

Dr. Kirstie Keller is a member of the Milken Institute's Center for Strategic Philanthropy’s biomedical science team, currently leading the center’s work on single ventricle heart defects, nonprofit drug development and venture philanthropy, and Alzheimer’s disease. With over ten years of experience in life sciences, she brings a unique set of skills and expertise, combining experience conducting scientific research at its highest level with a deep understanding of business conducted in a matrixed environment.


Derrick Rowe photo


Derrick Rowe, JD, Ph.D.
Wilson Sonsini Goodrich & Rosati

Derrick Rowe is a registered patent attorney in the Washington, D.C., office of Wilson Sonsini Goodrich & Rosati, where his practice focuses on patent prosecution and counseling designed to help clients protect their intellectual property rights both domestically and internationally in the life sciences, biotechnology, molecular biology, pharmaceutical, diagnostic, and medical device fields. He regularly works with clients to conduct patentability and freedom-to-operate analyses.


Day 2 - Rare Disease Forum


Rada Chirkova PhD


Rada Chirkova, Ph.D.
Computer Science, NC State

Rada Y. Chirkova is a professor of computer science at North Carolina State University. She is a senior member of the Association for Computing Machinery, served as general co-chair of the ACM International Conference on Management of Data (SIGMOD), and is the editor-in-chief of the SIGMOD Record Journal. Dr. Chirkova has co-authored three books, and has over sixty peer-reviewed publications on topics ranging from query containment and equivalence, query processing, view-based reformulation of data and queries, information security and leakage, to applications of policies to agent behaviors.


Sean Elkins, PhD


Sean Ekins, Ph.D.
Collaborations Pharmaceuticals

Sean is founder and CEO of Collaborations Pharmaceuticals, Inc. which is focused on using machine learning approaches for rare and neglected disease drug discovery. He was a senior scientist at Pfizer and then Eli Lilly, then joined startup companies as Associate Director of Computational Drug Discovery at Concurrent Pharmaceuticals Inc. (now Allergan) and Vice President of Computational Biology at GeneGo (now Thomson Reuters). Dr. Ekins is also adjunct professor at three U.S. universities and Entrepreneur in Residence at the UNC Eshelman School of Pharmacy. 


Lisa Gehtland


Lisa Gehtland, M.D.
RTI International

Lisa M. Gehtland, M.D., is a Research Public Health Analyst in the Center for Newborn Screening at RTI International, where her research interests include newborn screening systems, maternal and child health, and translational science. She is project director of Early Check, a research study designed to develop and evaluate methods to offer free, voluntary screening to up to 120,000 newborns per year in North Carolina. Dr. Gehtland is also the program manager for the RTI International partnership with the North Carolina Translational and Clinical Sciences Institute at UNC-Chapel Hill.


Jim Hagwood


Jim Hagood, M.D.
Pediatrics, UNC School of Medicine

Dr. Hagood is a pediatric pulmonologist specializing in rare lung disease. His “Lung Repair Lab” studies the molecular regulation of fibroblast phenotypes in pulmonary fibrotic disease and lung alveolarization. He also studies the roles and regulation of Thy-1, a critical modulator of cellular phenotype, and epigenetic/epigenomic alterations in lung development and disease, as well as targeted therapy for diffuse lung diseases. Dr. Hagood has joined two collaborative efforts to create a cellular and molecular map of the developing and mature lung, the LungMAP, funded by the National Heart, Lung and Blood Institute, and the HuBMAP, supported by the NIH Common Fund.

Tom Hickey


Anthony J. Hickey, Ph.D.
Director, UNC Catalyst for Rare Disease

Dr. Hickey is Distinguished RTI Fellow at the Research Triangle Institute and Director of the UNC Catalyst for Rare Diseases of the UNC Eshelman School of Pharmacy. He conducts a multidisciplinary research program in the field of pulmonary drug and vaccine delivery for treatment and prevention of a variety of diseases. Dr. Hickey is a Fellow of the Royal Society of Biology, the American Association of Pharmaceutical Scientists, the American Association for the Advancement of Science and the Royal Society of Biology. He has founded multiple companies including Cirrus Pharmaceuticals, Inc., Oriel Therapeutics, Inc; and Astartein, Inc.


Andy Holt Photo


Andy Holt
Asklepios BioPharmaceutical, Inc. (AskBio)

Andy Holt is the Vice President of Business Development and Manufacturing Support at AskBio. He has been working in gene therapy since 2010, focused on the manufacturing and release of viral vectors and cell therapies in a number of commercial and management roles, until recently in contract testing and manufacturing organizations. His work at AskBio focuses on partnership and manufacturing strategies for AskBio’s portfolio of AAV therapeutics.


Sharon King


Sharon King
Taylor's Tale, Batten Disease Foundation

Sharon co-founded and serves as President of Taylor’s Tale, the public charity named for her late daughter to raise awareness and funding for CLN1 disease (Batten disease) research and advocate for the needs of all rare disease patients. She is a state-appointed member of the N.C. Advisory Council on Rare Diseases, chair of the N.C. Rare Disease Coalition, a member of the Rare Disease InfoHub steering committee, and currently, manager of advocacy and community engagement at Aldevron.


Joan Levy Photo


Joan Levy, Ph.D.
Chordoma Foundation

Dr. Levy joined the Chordoma Foundation as Director of Research in 2017 with 30 years of experience in oncology research in academic, pharmaceutical and disease foundation settings. In her current role, Joan is responsible for oversight of research activities focusing on programs involving identification of new therapeutic targets, further development of the Drug Screening Program, and advancement of new drugs and combination approaches into the clinic. Joan was previously with Bayer Pharmaceuticals and the Multiple Myeloma Research Foundation.


Kathleen Miller Photo


Kathleen Miller, Ph.D.
US FDA, Orphan Drug Product Development

Kathleen L. Miller, Ph.D. is an economist in the Office of Orphan Products Development, in the Office of the Commissioner at the US Food and Drug Administration, and an adjunct assistant professor in the Department of Health Policy and Management in the Gillings School of Global Public Health at the University of North Carolina at Chapel Hill. Previously, she was an economist on the Economics Staff in the Office of the Commissioner. Dr. Miller’s primary research interests include pharmaceutical innovation, rare diseases, and policy incentives for drug development.




Fred Porter, Ph.D.
Adrenas Therapeutics

Frederick W. Porter, Ph.D. is Senior Vice President of Technical Development Gene Therapy and Manufacturing at BridgeBio Pharma and leads the development and industrialization of novel gene therapy treatments for rare diseases. Previously, Dr. Porter held key positions at Duke University, GlaxoSmithKline and Novartis Vaccines. His R&D expertise includes production and characterization of vaccines, gene therapy vectors, nucleic acid and recombinant protein therapeutics.                      




David Ridley, Ph.D.
​​​​​​Duke, Health Economist

David Ridley is the Dr. and Mrs. Frank A. Riddick Professor of the Practice of Business and the Faculty Director of Duke's Health Sector Management program. Dr. Ridley examines innovation and pricing in health care in his research. To encourage the development of new treatments for neglected diseases, Dr. Ridley and his co-authors proposed the priority review voucher program, which became law in the United States. His research appears in economics journals (Journal of Public Economics), medical journals (Journal of the American Medical Association), and scientific journals (Nature Reviews Drug Discovery).


Alexander Tropsha Photo


Alexander Tropsha, Ph.D.
Director, UNC/NC State InfoHub

Alexander Tropsha, PhD. is K.H. Lee Distinguished Professor and Associate Dean for Pharmacoinformatics and Data Science at the UNC Eshelman School of Pharmacy. His research interests are in the areas of computer-assisted drug design, computational toxicology, cheminformatics, (nano)materials informatics, and structural bioinformatics. Dr. Tropsha is an Associate Editor of the ACS Journal of Chemical Information and Modeling. His research has been supported by multiple grants from the NIH, NSF, EPA, DOD, foundations, and private companies.



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