How Oligonucleotide Therapeutics May Change the Course of Rare Diseases

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How Oligonucleotide Therapeutics May Change the Course of Rare Diseases

Available On Demand

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Using antisense oligonucleotides (ASOs) to treat patients with ultra-rare diseases is an incredibly exciting area. But the road from drug design to treatment is a long one.

On this GEN Live, we’ll discuss where ASO research is now, the challenges it faces as it moves forward, and the innovative new ways that those challenges are being overcome to treat patients. Dr. Stan Crooke, a pioneer in the field, founder of Ionis Pharmaceuticals and the n-Lorem Foundation and Dr. Punit Seth, senior vice president, at Alnylam Pharmaceuticals will join us for a broad discussion on the topic.

A live Q&A will follow the discussion allowing you a chance to pose your questions to our expert panelists.

Presenters

Stan Crooke MD, PhD

Founder and CEO, n-Lorem Foundation
Founder and Former CEO, Ionis Pharmaceuticals

Punit Seth, PhD

SVP Research
Alnylam Pharmaceuticals