Rare Disease Day Focuses on Cellular and Genetic Therapies

On Friday, March 2, Keck Graduate Institute (KGI) hosted its 11th Annual Rare Disease Day with presentations from prominent industry leaders and a poster session from KGI students.

The event serves as an opportunity for students, faculty, and guests to collaborate, discuss, and raise awareness for rare disease therapies. Attendees were officially welcomed by KGI President Sheldon Schuster, who also serves as vice chairman for the National Organization for Rare Disorders (NORD).

“With this year’s theme of new cell and gene therapies for rare diseases, we were fortunate to attract outstanding speakers from industry,” said Ian Phillips, the Director of KGI’s Center for Rare Disease Therapies. “KGI student Lana Clay also gave a riveting talk on her own experiences with acute lymphatic leukemia.”

Also featured among Friday’s speakers was Timothy Coté, who helped start KGI’s Rare Disease Day tradition with Phillips in 2008. Coté is an accomplished expert on rare diseases and prior to his current entrepreneurial ventures, he was Professor of Practice at KGI and director of the FDA Office of Orphan Products.

“I think an entrepreneurial spirit made Rare Disease Day a huge success,” said Ilona Kravtsova, who leads KGI’s Rare Disease Club. “For the first time, 21 posters made by Rare Disease Club members decorated the room and added texture to the event. The patient panel was engaging, and Lana Clay’s story was extremely touching. This year’s theme attracted and benefited a lot of students who want to stay on the forefront of medicine and the orphan product space.”

BioMarin Pharmaceuticals Vice President of Research Gordon Vehar delivered Friday’s keynote presentation titled “New Advances in Therapies for Rare Disease.” Starting his role at BioMarin in 2007, Vehar directs the company research platform focused on treatments for rare genetic diseases. Leveraging a multi-technology platform of drugs, biologics, gene therapy, and oligonucleotides, the efforts of Vehar’s team span early product concept through to research support of clinical trial needs.

Continuing the trend of Kite Pharma leaders sharing presentations at KGI, Armen Mardiros delivered a talk on “Biomarker Correlates and Potential Mechanisms of CAR T Cell Toxicities.” Serving as a senior scientist in the translational sciences group at Kite Pharma, Mardiros is the clinical biomarker lead for the ZUMA-3 and ZUMA-4 clinical trials in adult and pediatric B-ALL, respectively.

Another featured presenter was Paul Oh, who is a professor and Barrow Aneurysm Center co-director at the Barrow Neurological Institute. Oh is a scientist dedicated to studying two rare genetic vascular diseases: hereditary hemorrhagic telangiectasia and pulmonary arterial hypertension.

To conclude the day, a panel answered questions from the audience about advocacy for patients and families with rare diseases. The panel included:

• Two KGI students: Ilona Kravtsova, PharmD ’19, and Lana Clay, MBS ‘18
• Two representatives from Global Genes: Meredith Cagle and Amy Grover
• Two KGI alumni: William Raasch, MBS ’09, of WeHealth and Tayler Renshaw, PPC ’15, MBS ’16, of City of Hope