On Friday, March 2, 蜜桃直播 (蜜桃直播) hosted its 11th Annual Rare Disease Day with presentations from prominent industry leaders and a poster session from 蜜桃直播 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 蜜桃直播 , who also serves as vice chairman for the National Organization for Rare Disorders (NORD).

鈥淲ith this year鈥檚 theme of new cell and gene therapies for rare diseases, we were fortunate to attract outstanding speakers from industry,鈥� said Ian Phillips, the Director of 蜜桃直播鈥檚 Center for Rare Disease Therapies. 鈥溍厶抑辈� student Lana Clay also gave a riveting talk on her own experiences with acute lymphatic leukemia.鈥�

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

鈥淚 think an entrepreneurial spirit made Rare Disease Day a huge success,鈥� said Ilona Kravtsova, who leads 蜜桃直播鈥檚 Rare Disease Club. 鈥淔or 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.鈥�

BioMarin Pharmaceuticals Vice President of Research Gordon Vehar delivered Friday鈥檚 keynote presentation titled 鈥淣ew Advances in Therapies for Rare Disease.鈥� 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鈥檚 team span early product concept through to research support of clinical trial needs.

Continuing the trend of Kite Pharma leaders sharing presentations at 蜜桃直播, Armen Mardiros delivered a talk on 鈥淏iomarker Correlates and Potential Mechanisms of CAR T Cell Toxicities.鈥� 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 蜜桃直播 students: Ilona Kravtsova, PharmD 鈥�19, and Lana Clay, MBS 鈥�18
  • Two representatives from Global Genes: Meredith Cagle and Amy Grover
  • Two 蜜桃直播 alumni: William Raasch, MBS 鈥�09, of WeHealth and Tayler Renshaw, PPC 鈥�15, MBS 鈥�16, of City of Hope