RARE Daily

Alexion Donates $1 Million to n-Lorem to Develop and Provide Free Individualized Medicines to Nano-rare Patients

January 27, 2022

AstraZeneca’s Rare Disease group Alexion has made a $1 million donation to the nonprofit n-Lorem Foundation, which is dedicated to discovering and providing experimental antisense oligonucleotide medicines to nano-rare disease patients free for life.

Photo: Stanley Crooke, founder, CEO and chairman, n-Lorem Foundation

“Nano-rare patients have genetic diseases that often are unique to a single individual in the world. Today ASO technology and n-Lorem represent the only option for the treatment for many of these patients,” said Stanley Crooke, founder, CEO and chairman, n-Lorem Foundation.

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 50 nano-rare patients approved.

“At Alexion, we are committed to supporting patients and their families affected by rare diseases,” said Wendy Erler, vice president, Patient Advocacy, Alexion. “With this donation we seek to support n-Lorem’s development of individualized medicines one nano-rare patient at a time.”

Author: Rare Daily Staff

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