SAN DIEGO--(BUSINESS WIRE)--n-Lorem a nonprofit 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 Alexion, AstraZeneca’s Rare Disease group, has made a donation of $1 million.
“At Alexion, we are committed to supporting patients and their families affected by rare diseases. With this donation we seek to support n-Lorem’s development of individualized medicines one nano-rare patient at a time,” said Wendy Erler, Vice President, Patient Advocacy, Alexion.
“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. We are pleased to add Alexion to our expanding network of donors committed to advancing healthcare, of which a growing number of biotechnology companies, including Ionis Pharmaceuticals, Biogen and Ultragenyx, and now Alexion play a particularly important role in our efforts to help these patients,” said Stanley T. Crooke, M.D., Ph.D., Founder, CEO and Chairman, n-Lorem Foundation. “Alexion’s dedication to science and innovation to deliver life-changing medicines complements n-Lorem’s unique charitable model and expanding network of treating physicians and tertiary care centers. 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.”
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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 50 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.