Coming to KGI 3.1.13: Rare Disease Day

Schedule of events to include special showing of documentary about rare disease family and wide array of talks by industry leaders, top analyst

Drs. Ian Phillips (left) and Tim Coté

Sure there's Commencement, Convocation and TMP Day, but one of the biggest days on KGI's campus is Rare Disease Day, which will be celebrated this year on Friday, March 1. A seminar offering the industry perspective on rare diseases is scheduled from 9 a.m. to noon. Speakers will include CEOs and top executives from some of the world's largest and/or most innovative pharmaceutical and biotech companies, including Pfizer, Amicus and Biomarin Pharmaceuticals, among others. The speakers will share insights into the latest research on rare disease therapies being conducted by their companies and others in the biotech industry. Senior Analyst and Managing Director at J.P.Morgan Chase Geoffrey Meacham will be the lunchtime speaker. Meacham has been ranked for the past five years in the Institutional Investor poll, including a No. 2 ranking for the past four years. His often-cited research and analysis runs the gamut from large-cap biotech companies to small, development stage companies. In a recent interview with "The Life Sciences Report," Meacham discussed his view of hepatitis C and orphan disease drugs as "sweet spots" in the market in terms of therapeutic focus.

The afternoon will be devoted to a special preview of a new documentary film "Here. Us. Now. - A Family's Fight to Bring Medical Innovation Home." The film chronicles Hugh and Chris Hempel's efforts to find an effective drug treatment for their eight-year-old identical twin daughters, who have been diagnosed with Niemann Pick Type C. The rare and progressive neurological disease occurs when the body cannot properly break down cholesterol and other fats (lipids), leading to too much cholesterol in the liver and spleen and excessive amounts of other lipids in the brain.

There is no treatment for Niemann-Pick Type C, so the Hempels did their own research and discovered a simple compound, cyclodextrin, being used in mice. They petitioned the FDA to get permission to use it on their daughters. While not a cure, the Hempels feel that it has slowed the progression of the disease. In 2011, the couple started their own virtual biotech company, Solution Therapeutics, to help jumpstart clinical trials of cyclodextrin. The film was commissioned by the Ewing Marion Kauffman Foundation, the largest foundation in the country to focus on entrepreneurship, and directed by Emmy award-winning filmmaker Rudy Poe. Immediately following the film, there will be a panel discussion with Chris Hempel and Jonathon Bui, MD, neurologist and assistant professor at UC San Diego School of Medicine.

Rare Disease Day at KGI is chaired by Dr. Ian Phillips, director of the Center for Rare Disease Therapies and Norris Professor of Applied Life Sciences, and Dr. Tim Coté, professor of practice. It is sponsored by the Drug Information Association (DIA) ─ KGI Chapter.

For more information or to attend Rare Disease Day at KGI contact Dr. Ian Phillips at Ian_Phillips[at symbol]kgi.edu.