RARE Daily

n-Lorem Partners with Cytiva to Bring Individualized ASO Medicines to Patients with Nano-rare Diseases

February 8, 2022

Rare disease nonprofit n-Lorem is collaborating with Cytiva, a research and biopharmaceutical manufacturer, in support of n-Lorem’s effort to discover and provide experimental antisense oligonucleotide medicines to nano-rare disease patients (1 to 30 patients worldwide) free for life.

Photo: Stanley Crooke, 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,” said Stanley Crooke, founder, CEO and chairman of n-Lorem Foundation. “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.”

Cytiva, a subsidiary of Danaher, is a global provider of technologies and services that advance and accelerate the development and manufacture of therapeutics, ranging from biological research to developing innovative vaccines, biologic drugs, and novel cell and gene therapies.

“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 at 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.”

n-Lorem Foundation was 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 more than 100 applications for treatment with more than 50 nano-rare patients approved.

Author: Rare Daily Staff

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