Coming Together for the Nano-rare Patient

Patient Empowerment Program: A Rare Disease Podcast

This episode is proudly sponsored by our partner, Alnylam Pharmaceuticals, the leading RNAi therapeutics company. When Stan Crooke ran Ionis and John Maraganore ran Alnylam, they were partners that turned into rivals — and not always friendly ones — as they persevered to pursue an entirely new therapeutic space; RNA-targeted drug discovery and development. Now, the pair of drug discovery titans have united once again in support of Dr. Crooke’s n-Lorem Foundation—working to provide personalized medicines to the rarest of rare disease patients (nano-rare) using the antisense oligonucleotide (ASO) technology Stan led the creation of at Ionis. In this episode, Stan has a conversation with Dr. John Maraganore about John’s past, their former rivalry, and the optimism shared between the two with respect to a better future for nano-rare patients.

On This Episode We Discuss:

- Being a dream merchant and driving a dream into a real therapeutic platform

- Founding of Regulus and the synergy between Ionis and Alnylam throughout the years

- What happens when two very competitive CEO-scientists have competing drugs

- Overcoming their differences

- Giving hope to a patient and their family with nano-rare diseases is powerful

- Saving the world one life at a time

- Bringing ASO and RNAi technologies together to collaboratively help nano-rare patients

- A sustainable non-profit model?

- Hope – a powerful thing to lose and an important thing to recover