- Global Pharma News & Resources

n-Lorem Foundation Partners with Ultragenyx to Bolster Development of Personalized Medicines for Patients with Ultra-Rare Diseases

Foundation’s collaboration with premier rare disease company strengthens efforts to reach more patients in need

SAN DIEGO--(BUSINESS WIRE)--#ASOs--n-Lorem Foundation, a nonprofit organization that provides free, lifetime supplies of individualized RNA-targeted medicines to patients living with ultra-rare diseases, today announced a new partnership with Ultragenyx Pharmaceutical (NASDAQ:RARE), a biopharmaceutical company focused on the development and commercialization of novel products for serious rare and ultra-rare genetic diseases.

n-Lorem Foundation is the first and only foundation with a mission to provide potentially life-saving treatments to patients who have diseases caused by extremely rare mutations (1 to 30 patients worldwide) for free, for life, by coupling advanced genomic diagnostics with antisense oligonucleotides (ASOs) medicines. The new collaboration with Ultragenyx, one of the leading rare disease companies in the world, and its CEO and founder, Dr. Emil Kakkis, bolsters n-Lorem’s mission to bring immediate hope and rapid treatment to ultra-rare disease patients in need.

“On behalf of the patients we serve, I thank Dr. Emil Kakkis and Ultragenyx for their generous support, and I look forward to working with Emil and his team at Ultragenyx to bring therapies to patients with ultra-rare diseases for free, for life,” said Dr. Stanley T. Crooke, Founder, CEO and Chairman of n-Lorem Foundation. “Ultragenyx joins Ionis Pharmaceuticals, Biogen, Charles River Labs, Covance by Labcorp, and Korea Institute of Toxicology (KIT) as industry partners supporting our efforts at n-Lorem. We look forward to working with other health care companies in the future.”

Ultragenyx focuses on developing products for serious rare and ultra-rare genetic diseases, and Dr. Kakkis brings a wealth of valuable expertise through his work on policy and industry issues affecting rare disease treatment development. The additional resources, financial contribution and experience from both the company and Dr. Kakkis will be critical as n-Lorem continues to scale-up its unique charitable treatment model to help patients with ultra-rare mutations.

“Today, we have the science to treat some of the rarest diseases in the world, and these patients deserve treatments,” said Emil D. Kakkis, M.D., Ph.D., CEO and President of Ultragenyx Pharmaceutical. “We are proud to partner with n-Lorem, a first-of-its-kind organization that is using a proven technology to develop treatments for patients with these ultra-rare diseases for the first time.”

To learn how your organization can partner with n-Lorem and help answer the challenge of treating patients with ultra-rare diseases, contact Tracy Johnson, Executive Director ( Learn more about n-Lorem’s mission at, and please consider giving to n-Lorem to bring hope, possibility and treatment options to these desperate patients and families.

  • Watch: n-Lorem Foundation: Creating a Better Future for Ultra-Rare, One Patient at a Time
  • Watch: n-Lorem Foundation’s 1st year of Progress

About n-Lorem Foundation

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 patients with ultra-rare diseases (1 to 30 patients) that are the result of a single genetic defect unique to only one or very few individuals. ASOs 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. n-Lorem was founded by Stanley T. Crooke, M.D., Ph.D., former chairman and CEO, and current executive chairman of the board 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. To learn more about the n-Lorem Foundation, visit, and follow us on Twitter, Facebook, LinkedIn and YouTube.

About Ultragenyx Pharmaceutical Inc.

Ultragenyx is a biopharmaceutical company committed to bringing to patients novel products for the treatment of serious rare and ultra-rare genetic diseases. The company has built a diverse portfolio of approved therapies and product candidates aimed at addressing diseases with high unmet medical need and clear biology for treatment, for which there are typically no approved therapies treating the underlying disease. The company is led by a management team experienced in the development and commercialization of rare disease therapeutics. Ultragenyx’s strategy is predicated upon time- and cost-efficient drug development, with the goal of delivering safe and effective therapies to patients with the utmost urgency. For more information on Ultragenyx, please visit the company's website at:


n-Lorem contact:
Tracy Johnson, Executive Director

Media Contact:
Will Zasadny
Canale Communications

Ultragenyx Contact:
Joshua Higa

Editor Details

  • Company:
    • Businesswire
Last Updated: 05-Apr-2021