On February 26, Keck Graduate Institute (KGI) will host “Rare Disease Day: Advances in Rare Disease Therapy,” a daylong event dedicated to exploring and discussing the many facets that surround rare disease patients and their therapy.
“Rare Disease Day raises awareness about the plight of 30 million people in the USA who have a rare disease,” said Professor Ian Phillips, director of the KGI Center for Rare Disease Therapies. “At KGI, we have been celebrating the day every year, and on Friday, February 26, we will have our 7th Annual Rare Disease Day. The theme this year is “New Innovations in Rare Disease Therapies.”
Scheduled to present are several exceptional speakers, including scientists, doctors, and patient advocates. The event’s keynote speaker is Dr. Alvin Shih, executive vice president of research and development at Retrophin, Inc., a company dedicated to developing orphan drugs. Other speakers include Dr. Tim Coté, principal of Coté Orphan Consulting, LLC; Tamar Grossman, PhD, associate director at Ionis Pharmaceuticals; Ethan Perlstein, PhD, CEO of Perlstein Lab, PBC; Will Raasch, a KGI alumnus who founded WeHealth; and Nicole Boice, founder and CEO of Global Genes, a global patient advocate organization.
KGI’s tradition is to show a new movie each year related to patients with rare diseases; this year we will screen an extraordinary film, Banner on the Moon, which follows the accomplishments of Cindy Abbot, a patient with a rare disease who has climbed Mount Everest and raced in the 1,000 mile Iditarod Trail Sled Dog Race in Alaska.
The Rare Disease day is supported by the KGI student Rare Disease Club and The Center for Rare Disease Therapies.
Program Details:
• 8 a.m. - 8:45 a.m. Breakfast
• 8:45 a.m. Welcome, president of KGI, Sheldon M. Schuster, PhD
• 9 a.m. - 9:15 a.m. Introduction, Dr. Ian Phillips, director of KGl’s Center for Rare Disease Therapies
• 9:15 a.m. - 9:55 a.m. “Antisense Oligonucleotide Based Therapy for Rare Diseases,” Dr. Tamar Grossman, associate director, lonis Pharmaceuticals
• 9:55 a.m. - 10:30 a.m. “PLab: a therapeutics platform for 5,000+ rare genetic diseases,” Dr. Ethan Perlstein, founder and CEO, Perlstein Lab
• 10:30 a.m. - 11 a.m. Coffee Break
• 11 a.m. - Noon Panel discussion: “New and Innovative Therapies”
Speakers and Panelists
• Timothy Coté, principal, Coté Orphan Consulting, LLC (session chair)
• Tamar Grossman, associate director, lonis Pharmaceuticals
• Ethan Perlstein, founder, Perlstein Lab, PBC
• Nicole Boice, founder and CEO, Global Genes
• Will Raasch, founder, WeHealth
• Alvin Shih, executive vice president, R&D, Retrophin, Inc.
• Noon - 1:30 p.m. Lunch
• Lunch Speaker: Dr. Alvin Shih, executive vice president of research and development at Retrophin, Inc.
• Discussion leaders: Anderson Van and Caroline Kim for the KGI Rare Disease Club
• 2 p.m. - 3:15 p.m. Special Screening: Banner on the Moon, with special guest Cindy Abbott
• 3:15 p.m.- 4 p.m. Discussion of film with special guest Cindy Abbott
• 4:30 p.m. Adjourn
