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Entrepreneurship and Rare Disease Day Helps KGI Raise Awareness

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Since its founding in 1997, Keck Graduate Institute has established plenty of impactful traditions. Rare Disease Day continues to be one such important KGI event, as students, faculty, and guests gather to collaborate, discuss, and raise awareness for rare disease therapies.

Friday, Feb. 17, 2017 marked the 10th Annual Rare Disease Day at KGI, featuring a theme of entrepreneurship. Five industry leaders shared presentations and Q&A sessions, providing opportunities for KGI students to connect and lead the conversation about rare diseases.

“The rare disease space offers opportunities for those looking to start new companies,” KGI President Sheldon Schuster said in his welcome to the crowd in building 517. “It is important for us to recognize that there is tremendous strength in all of us working together to ensure that everyone with a rare disease knows they are part of a compassionate community.”

Following Schuster’s introduction, Director of the Center for Rare Disease Therapies Ian Phillips spoke about the event’s history and the key contributors throughout the years. KGI’s Rare Disease Day began in 2008 with an on-site Food and Drug Administration (FDA) workshop led by Tim Cote, who previously served as the FDA’s director of the Office of Orphan Products Development.

Phillips also commended several companies on their progress in orphan therapies with gene therapy. Among the highlights mentioned by Phillips were Ionis’ Spinraza therapy for spinal muscular atrophy, Spark Therapeutics’ gene therapy for retinal blindness, GSK’s Strimvelis that treats severe combined immunodeficiency, and Sarepta’s Eteplirsen for Duchenne muscular dystrophy.

The first featured guest of the event, Ryan Bethencourt, shared his stories about the impact of biotechnology on rare disease therapies and the exponential momentum behind biotech investments and startups. Betehncourt serves as the program director and venture partner at IndieBio, an incubator company focused on accelerating biology. He also is an advisor, angel investor, and board member at multiple biotech companies.

“Entrepreneurship and Rare Disease Day granted me the opportunity to meet those that inspire me to think bigger,” said KGI School of Pharmacy student Ilona Kravtsova, who serves as president elect of the Rare Disease Club.

“The day was a great example of KGI’s collaboration between science, business, innovation, and compassion,” she said. “From the giants of biopharma and innovation to patient advocates affected by rare disease, all have come together to advance treatments and spread awareness for rare diseases.”

Joining Bethencourt in the morning Q&A panel was William Raasch, a KGI Master of Bioscience (MBS) alumnus. Raasch is the founder of WeHealth, a web platform that harnesses the power of social networks to find patients and raise awareness for rare diseases.

Later on during the event, the attendees and presenters took part in breakout sessions and that gave guests an opportunity to meet Tamar Grossman of Ionis Pharmaceuticals along with Shannon and Rob Grandia of the Tuberous Sclerosis Alliance (TSA).

Within Ionis, Grossman serves as the associate director in the Antisense Drug Discovery Department. She is a group leader, and her lab is focusing on the identification of novel antisense therapeutics to various genetic and rare diseases.

“The passion from our guests and the creative energy in the room reinforced my interest in pursuing a career in pharmaceutical industry and being on the forefront of scientific breakthroughs for rare diseases,” Kravtsova said.

For the Grandia family, the effort to help raise awareness of tuberous sclerosis complex (TSC) is a personal mission. Rob and their children, Rylee, Jake, and Luke are diagnosed with TSC. Up to 40,000 Americans and over 1.2 million people worldwide suffer from the condition, according to the National Institute of Neurological Disorders and Stroke.

“Although it was heart-wrenching to hear about the myriad medical problems that tuberous sclerosis complex (TSC) can cause, I am incredibly grateful that Rob and Shannon shared their story with us,” said KGI School of Pharmacy student Russle Benson, who is the president of the Rare Disease Club. “I was surprised to learn how much work Shannon and Rob put into building a community for families affected by TSC to be able to exchange information and receive mutual support from one another.

“Listening to Rob and Shannon share their patient journey has reaffirmed my passion to raise awareness about rare diseases in order to improve the lives of patients that suffer from them.”