Cambridge Healthtech Institute’s 2nd Annual

Drug Discovery for Rare Diseases

Exploring New Technologies, Targets and Drug Modalities

March 28, 2019


Rare diseases, or diseases that affect only a small percentage of the population, have grown in significance and prominence in recent years. According to the National Institutes of Health, there are nearly 7000 rare diseases and more than 25 million Americans who are affected. Approximately 80% of these rare diseases are genetic in origin. Cambridge Healthtech Institute’s symposium on Drug Discovery for Rare Diseases will bring together leading scientists, clinicians, executives and experts who are involved in finding new drug targets and drug modalities for treating rare disorders. This unique one-day event will encourage people from diverse backgrounds to discuss potential opportunities, as well as existing challenges in this field. It’s an opportunity for scientific and technical experts to come together to exchange ideas, experiences and to set up collaborations for rare disease research.

Preliminary Agenda

Rare Diseases: From Simple to Complicated, Complex and Cure

Michael Liebman, PhD, Managing Director, Strategic Medicine, Inc.

Talk Title to be Announced

James J. Hickman, PhD, Founding Director, NanoScience Technology Center; Professor, Nanoscience Technology, Chemistry, Biomolecular Science, Material Science and Electrical Engineering, University of Central Florida

Considerations for Late-Stage Gene and Cell Therapies

Timothy J. Miller, PhD, President & CSO, Abeona Therapeutics, Inc.

Apelinergic Therapy in Pulmonary Arterial Hypertension

Hyung Chun, MD, FAHA, Associate Professor of Medicine and Pathology, Yale School of Medicine

Talk Title to be Announced

Philip John (P.J.) Brooks, PhD, Program Director, Office of Rare Diseases Research, National Center for Advancing Translational Sciences (NCATS), National Institutes of Health (NIH)

Gene Editing Cancer Cells: A New Frontier in Cell Therapy for Cancer

Khalid Shah, MS, PhD, Director, Center for Stem Cell Therapies and Imaging, Harvard Medical School; Vice Chair of Research, Brigham and Women's Hospital

Disrupting a BCL11A Enhancer with Zinc Finger Nucleases for the Potential Treatment of Sickle Cell Disease and Beta Thalassemia

Alexandra Hicks, PhD, Executive Director, Hemoglobinopathies, Bioverativ, a Sanofi Company

Talk Title to be Announced

Mark J. Osborn, PhD, Assistant Professor, Department of Pediatrics, Division of Blood and Marrow Transplantation, University of Minnesota

Paving a Legal Path for Translational Genome Editing

Paul Enríquez, JD, LLM, PhD Candidate, Structural and Molecular Biochemistry, North Carolina State University

For more details on the conference, please contact:

Tanuja Koppal, PhD

Conference Director

Cambridge Healthtech Institute

For partnering and sponsorship information, please contact:

Carolyn Cooke

Business Development Manager

Cambridge Healthtech Institute

Phone: (+1) 781-972-5412