• Conference spotlights how rare-disease research is pioneering the cutting-edge fields of stem cell, gene therapy and RNAi therapies
• Novel research funding models have evolved through the rare-disease community

The University of California, Davis, Department of Chemistry will spotlight a special focus on rare-disease research with this year’s 15th annual R. Bryan Miller Symposium. Experts in rare diseases will gather at the UC Davis Conference Center March 5 for a conference highlighting the opportunities and challenges in applying cutting-edge technologies and “precision medicine” to better treat conditions that together affect millions of people, especially children. The event is held in conjunction with Rare Disease Day, which this year is Feb. 28.

“On the 15th anniversary of the R. Bryan Miller Symposium, the chemistry department is proud to sponsor this event and aims to continually grow the event every year,” said conference chair Sundeep Dugar, a Ph.D. graduate of chemistry at UC Davis and current chief operating officer of Carderox, a Bay Area biotech company with a focus on rare diseases and associated morbidities. “Our aim with the conference is to be a central and convening partner to the many academia and industry researchers in the area that are navigating the research obstacles to find cures for those with rare disease.”

The Rare Disease Conference at UC Davis will look at the obstacles and novel approaches that are shaping the future of rare-disease research. Speakers include leaders in rare-disease research from academia and industry, as well as from UC Davis. Presentations will also spotlight how patient advocates and foundations play an integral role in driving rare-disease research forward.

Topics will include emerging technologies, such as: stem cell therapy, gene therapy and RNAi therapy, while also emphasizing new research fundraising models and partnerships, the importance of diagnostics, and integrative biologic strategies.

The organizations represented include the Sanford Burnham Medical Research Institute, UC Davis, UC San Diego, NEA Partners, Transderm, Benefunder and rare-disease nonprofit organizations such as EndDuchenne, the Cystinosis Research Foundation, CheckOrphan and RARE Science.

The event will be followed March 6 by the annual Miller Symposium on pharmaceutical chemistry. Both events are organized by the UC Davis Department of Chemistry. More information and registration details can be found at http://chemistry.ucdavis.edu/seminar/miller_symposium/.

About rare diseases

There are over 7,000 rare diseases that have been identified to date. A rare disease is defined as one that affects 200,000 or less people in the U.S. and one out of 2,000 individuals in Europe. Combined, it is estimated that over 25 million Americans are affected by a rare disease.

Developing a treatment for even a common disease takes an average of 14 years at a cost of $2 billion with a 95 percent failure rate. Patients, researchers and clinicians fighting rare diseases face additional problems of restricted resources, small patient populations, and limited specialists and researchers that understand the rare disease. In addition, 50 percent of rare diseases affect children, and 30 percent of patients die before their fifth birthday. These challenges demand urgent and new, innovative approaches for rare-disease researchers.

In his 2015 State of the Union address, President Obama announced details of the Precision Medicine Initiative, which will pioneer a novel model of patient-centered research. Precision medicine takes into account patients' genetic makeup, environment and lifestyles to better understand disease and tailor treatments to a specific individual and condition.

About the R. Bryan Miller Symposium

The annual Miller Symposium honors chemistry professor R. Bryan Miller, whose research laid the groundwork for current thriving programs in pharmaceutical chemistry and chemical biology at UC Davis. Miller died in 1998. This year the symposium has added an extra day to focus on rare-disease research. The symposium is sponsored by the UC Davis Department of Chemistry in partnership with CheckOrphan and RARE Science.

Conference information and registration

Please contact Patricia Chuda, UC Davis Department of Chemistry, (530) 752-6357, pchuda@ucdavis.edu.