2015 Rare Disease Day at KGI to Explore the Use of Precision Medicine for the Treatment of Rare Disease

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Dr. Stanley Crooke, founder of Isis Pharmaceuticals and a pioneer in the field of antisense therapy, to give the keynote talk at Rare Disease Day 2015 at Keck Graduate Institute.

Dr. Stanley Crooke, founder and CEO of Isis Pharmaceuticals

Precision medicine is the new trend for gene therapies and antisense therapies. It involves utilizing very precise methods for replacing or inhibiting mutant genes known to cause a disease.

Precision medicine is a hot topic in health care and scientific research. President Obama spoke about it in his State of the Union Address on January 22, and the White House quickly followed up with details about the President’s Precision Medicine Initiative ―a $215 million investment that is aimed at accelerating biomedical discoveries and dramatically improving treatment options for a number of diseases, including those classified as “rare.”

In recognition of the potential of precision medicine to revolutionize the treatment of rare disease, the theme of Keck Graduate Institute’s 6th Annual Rare Disease Day on Friday, February 27, is “Precision Medicine for Rare Disease.” Rare Disease Day is an international advocacy day to bring widespread recognition of rare diseases as a global health challenge. Each year, KGI’s Center for Rare Disease Therapies recognizes the day with a speaker series, panel discussion and the showing of a documentary film on rare disease.

This year’s keynote speaker is Dr. Stanley Crooke, a pioneer in the field of precision medicine known as antisense therapy. Antisense gene therapy is a gene silencing technique. The therapy is called a gene silencing technique because, instead of repairing the gene that causes disease, it aims to “silence” the gene’s effect. As the founder, chairman and CEO of Isis Pharmaceuticals, Dr. Crooke has led the scientific development of antisense technology and engineered the creation of one of the largest and more advanced development pipelines in the biotechnology industry. Isis has achieved commercialization of the first two antisense drugs to reach the market, Vitravene and KYNAMRO®. KYNAMRO®, approved in January 2013, is the first systemically administered antisense drug to be approved and the first to be approved for lifelong treatment of a chronic rare disease, Homozygous Familial Hypercholesterolemia, disorder of high LDL (“bad”) cholesterol that is passed down through families.

“Precision medicine is the new trend for gene therapies and antisense therapies. It involves utilizing very precise methods for replacing or inhibiting mutant genes known to cause a disease,” said Dr. Ian Phillips, director of KGI’s Center for Rare Disease Therapies. “Having Dr. Crooke as a speaker at this year’s Rare Disease Day is an incredible opportunity for our students and members of our community to hear firsthand how Isis has been at the forefront of this research and technology.”

Chris Garabedian, CEO of Sarepta Pharmaceuticals, is also slated to speak at the event. He will talk about the development of antisense therapy to treat Duchenne muscular dystrophy, a rare disease affecting around 1 in 3,600 males, which results in muscle degeneration and eventual death. His talk will also cover medical countermeasures (MCMs) against the Ebola, Marburg and flu viruses, and against antibacterial resistance.

The Rare Disease Day event will also include the showing of the film “Silent Angels: The Rett Syndrome Story.” Narrated by actress Julia Roberts, the documentary film explores the lives of children (primarily girls) living with this rare disorder of the nervous system that leads to developmental reversals, especially in the areas of expressive language and hand use.

Rare Disease Day at KGI will also include a panel discussion with Dr. Tim Coté, the founder of Coté Orphan Consulting and a former director of the FDA’s Office of Orphan Product Development; Dr. Jon Bui, associate professor at UC San Diego School of Medicine’s Department of Neurosciences; Dr. Sukirti Bagal, director, US & Global Medical Affairs and Clinical Development, Pfizer; and Barbara Lavery, board member, Global Genes. Global Genes™ is one of the leading rare disease patient advocacy organizations in the world.

KGI’s 6th Annual Rare Disease Day will take place on Friday, February 27 at KGI’s campus in Claremont, CA. For more information contact Kelly Esperias at Kelly_Esperias(at)kgi(dot)edu or 909-607-9651.

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Paige Stein
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