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.
We cannot do
this alone
Together we are changing the world—
one patient at a time
We hope that you join us on this journey to discover, develop and provide individualized antisense medicines for free for life for nano-rare patients. The ultimate personalized medicine approach – for free, for life.
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