n-Lorem Foundation Partners with Cytiva to Bring Individualized ASO Medicines to Patients with Nano-rare Diseases
Cytiva donates equipment totaling $415,000 to n-Lorem that will aid in the development and manufacturing of investigational ASO medicines for n-Lorem’s nano-rare patients
SAN DIEGO--(BUSINESS WIRE)--#ASO--n-Lorem, a nonprofit foundation, is collaborating with Cytiva, a global life sciences leader, in support of n-Lorem’s effort to discover and provide experimental antisense oligonucleotide (ASO) medicines to nano-rare disease patients (1 to 30 patients worldwide) for free, for life.
“On behalf of our patients and their families, we offer a heartfelt thank you to the leadership and employees of Cytiva for such a generous donation,” said Stanley T. Crooke, M.D., Ph.D., Founder, CEO and Chairman of n-Lorem Foundation. “Decreasing costs and increasing efficiency and speed at all points in the drug discovery, development and manufacturing processes translates to a significant reduction in per-patient costs, which makes our non-profit model feasible. Without partners, like Cytiva, who share our mission and commitment to helping as many nano-rare patients as we can, we would not be able to discover, develop and provide ASO medicines to nano-rare patients for free for life.”
“At Cytiva, our mission is to advance and accelerate therapeutics. n-Lorem is offering this type of personalized experimental ASO therapy to patients with few therapeutic options for free, for life,” said Dirk Voekel, Chief Innovation Officer from Cytiva. “As leaders in bringing speed, efficiency and capacity to research and manufacturing workflows, we are delighted to be able to support the manufacturing of transformative ASO medicines to nano-rare patients.”
- Watch: n-Lorem Foundation Delivers Hope and Treatment for Patient with Nano-Rare Disease
- Watch: n-Lorem Foundation: Creating a Better Future for Nano-Rare, One Patient at a Time
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.
Amy Williford, Ph.D.
Director of Communications