RARE Daily

n-Lorem Enters Partnership with Ultragenyx for Personalized Treatments for Patients with Ultra-Rare Conditions

April 5, 2021

Rare Daily Staff

n-Lorem Foundation, a nonprofit that provides free, lifetime supplies of individualized RNA-targeted medicines to patients living with ultra-rare diseases, said it entered into a partnership with Ultragenyx Pharmaceutical to bolster n-Lorem’s efforts.

Ultragenyx joins Ionis Pharmaceuticals, Biogen, Charles River Labs, Covance by Labcorp, and Korea Institute of Toxicology as industry partners supporting n-Lorem’s work.

Ultragenyx focuses on developing products for serious rare and ultra-rare genetic diseases, and the company’s President and CEO Emil Kakkis brings a wealth of 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 Kakkis will be critical as n-Lorem continues to scale-up its unique charitable treatment model to help patients with ultra-rare mutations, the foundation said.

“Today, we have the science to treat some of the rarest diseases in the world, and these patients deserve treatments,” said Kakkis, 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.”

Photo: Emil Kakkis, president and CEO of Ultragenyx

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