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

n-Lorem Foundation, a nonprofit organization that provides free, lifetime supplies of individualized RNA-targeted medicines to patients living with ultra-rare diseases, announced a new partnership with Ultragenyx Pharmaceutical, 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.”