Coming Together for the Nano-rare Patient

May 3, 2023


Two rival drug discovery titans come together for one reason – the patients

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

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John Maraganore is well known and widely admired throughout the biotechnology industry. At Alnylam, he led the conversion of siRNAs from compelling cell biology to a powerful new drug discovery platform, forever changing the scientific world.

John did his undergraduate studies and Ph.D. at his hometown university, the University of Chicago. When he started researching protein structure and function and enzymology his sophomore year, he fell in love with science and began to see much of his time dedicated to research. He credits his father for his keen interest in medicine, and his mother for his optimism, a trait that is absolutely critical for a CEO and in the world of drug discovery and development.

After observing the fascinating emergence of antisense technology at Ionis, John joined Alnylam with the belief that he could be involved with the creation of a whole new class of medicines, RNAi therapeutics. Over the years, John has helped bring three rare disease medicines to the market. He shares the same motivation for this development as Stan – helping patients.

When John and Stan first met each other there was immediate chemistry and respect. The two worked well together and eventually co-founded Regulus Therapeutics, focused on the discovery and development of innovative medicines targeting microRNAs. Although, over the years, they grew into fierce rivals with a few drugs that were in direct competition with each other. This eventually led to a falling out between the two and a halt in collaboration.

As Stan was working on n-Lorem, he knew that his organization’s aspirations of providing personalized medicine were bigger than his rivalry with John. So, Stan reached out to John through email and the rest was history. John had been watching the n-Lorem story from afar; graciously, he knew that the two must come together to help nano-rare families. John supports n-Lorem as the co-chair of the Advisory Council and has been a generous donor to aid efforts surrounding the discovery and development of personalized medicines for nano-rare patients. Together, n-Lorem and Alnylam can be thought of as ‘Corps of Discovery’, with the common goal of helping patients.

John Maraganore, Ph.D., is co-chair of the n-Lorem Advisory Council. He was the founding CEO of Alnylam where he built and led the company from early platform research on RNA interference (RNAi) through global approval and commercialization of four RNAi therapeutic medicines including ONPATTRO®, GIVLAARI®, and OXLUMO®, commercialized by Alnylam, and Leqvio®, commercialized by Novartis. He continues to serve on the Alnylam Scientific Advisory Board. Prior to Alnylam, Dr. Maraganore served as an officer and a member of the management team for Millennium Pharmaceuticals, Inc., most recently as senior vice president, strategic product development. Before Millennium, he served as director of molecular biology and director of market and business development at Biogen, Inc. At Biogen, Maraganore invented and led the discovery and development of ANGIOMAX® (bivalirudin) for injection. Prior to Biogen, Dr. Maraganore was a scientist at ZymoGenetics, Inc., and the Upjohn Company. Dr. Maraganore received his Ph.D. in biochemistry and molecular biology at the University of Chicago. He is a member of the board of Agios Pharmaceuticals, Beam Therapeutics, Kymera Therapeutics, ProKidney Corp, And Takeda Pharmaceuticals. He is on the board of the Biotechnology Innovation Organization (BIO), where he was chair from 2017-2019. Maraganore also serves as a venture partner with Atlas Ventures and Arch Ventures, and is an advisor with Blackstone Life Sciences and RTW Investments. If you want to learn more about Alnylam, visit You can follow John on Twitter @JMaraganore.


Hosted by: Dr. Stan Crooke.
Videographer: Jon Magnuson of Mighty One Productions.
Producers: Kim Butler, Colin Delaney, Kira Dineen, Jon Magnuson, Andrew Serrano and Amy Williford.

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To follow along with this interview, download the transcript below.


Listen to our next interview:

One Year Anniversary: It Takes a Rare Community

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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Join us on our Corps of Discovery of the mind and heart. Help us bring hope and potential help to nano-rare patients today. For free, for life.

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