SAN DIEGO, Calif., January 4, 2022 (Business Wire) — n-Lorem a non-profit Foundation, discovering and providing experimental antisense oligonucleotide (ASO) medicines to nano-rare disease patients (1 to 30 patients worldwide) for free, for life, today announced that it has appointed John Maraganore, Ph.D., Chair of its Advisory Council. In this role, John Maraganore will chair a group of n-Lorem advisors with a focus on exploring fundraising opportunities, raising awareness of n-Lorem and its mission.

“I am pleased to welcome John to the n-Lorem Advisory Council. John’s long-standing work in the field of RNA-targeted therapies and his financial expertise will be a substantial benefit to n-Lorem’s mission,” said Stanly T. Crooke, M.D., Ph.D., Founder, CEO and Chairman of n-Lorem Foundation. “John and I were both privileged to play leadership roles in the establishment of RNA-targeted therapeutics as a broadly useful, more efficient drug discovery platform. Adding John to the team will bring many strengths as we enhance our ability to achieve our mission of bringing RNA-targeted treatments to patients with nano-rare diseases for free for life.”

“I am honored to be joining the n-Lorem Advisory Council as its Chair, and look forward to working with like-minded advisors to support n-Lorem’s mission to bring experimental therapies to nano-rare patients,” said John Maraganore, Ph.D. “I feel that the patients served by n-Lorem are in desperate need of the best from all of us. I am honored to work again with Stan in achieving a high-quality and scalable approach for the nano-rare patient population. I am committed to do all that I can to enable n-Lorem to treat even more patients.”

“At n-Lorem, we are fortunate to have a group of inspired and motivated individuals from across the drug discovery and development spectrum, across patient support and advocacy and across the financial industry. John is a natural choice to lead this Advisory group, as he understands the power RNA-targeted technologies can bring to bear on treating nano-rare patients, and how vital it is that we continue to raise funds and increase awareness. These efforts will translate into more opportunities to discover, develop and provide potential therapies for free, for life for these patients,” concluded Dr. Crooke.

Learn more about n-Lorem’s mission at www.nlorem.org, and please consider giving to n-Lorem to bring hope, possibility and treatment options to these needy patients and families.

• Watch: n-Lorem Foundation Delivers Hope and Treatment for Patient with Ultra-Rare Disease
• Watch: n-Lorem Foundation: Creating a Better Future for Ultra-Rare, One Patient at a Time

About John Maraganore, Ph.D.

John Maraganore was the founding CEO of Alnylam from 2002-2021. At Alnylam, 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, and the Biotechnology Innovation Organization (BIO), where he was Chair from 2017-2019. Maraganore also serves as a Venture Partner with Arch Ventures.

About n-Lorem

n-Lorem Foundation is a non-profit organization established to apply the efficiency, versatility and specificity of antisense technology to charitably provide experimental antisense oligonucleotide (ASO) medicines to treat nano-rare patients diagnosed with diseases that are the result of a single genetic defect unique to only one or very few individuals. Nano-rare patients describe a very small group of patients (1-30 worldwide) who, because of their small numbers, have few if any treatment options. n-Lorem Foundation was created to provide hope to these nano-rare patients by developing individualized ASO medicines, which are short strands of modified DNA that can specifically target the transcripts of a defective gene to correct the abnormality. The advantage of experimental ASO medicines is that they can be developed rapidly, inexpensively and are highly specific. To date, n-Lorem has assisted in the development and treatment of 14 nano-rare patients and received over 100 applications for treatment with more than 40 nano-rare patients approved. n-Lorem was founded by Stanley T. Crooke, M.D., Ph.D., former chairman and CEO of Ionis Pharmaceuticals, who founded Ionis Pharmaceuticals in 1989 and, through his vision and leadership, established the company as the leader in RNA-targeted therapeutics. Follow us on Twitter, Facebook, LinkedIn and YouTube.

n-Lorem contact:

Amy Williford, Ph.D.
Director of Communications
[email protected]

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