Partnership leverages La Jolla Labs’ automated, high-throughput screening technology to identify optimal ASO candidates.
SAN DIEGO, Calif., Mar. 16, 2022, (Business Wire) — n-Lorem, a nonprofit foundation, has engaged La Jolla Labs, a RNA drug discovery company, to support n-Lorem’s discovery efforts to provide personalized experimental antisense oligonucleotide (ASO) medicines for nano-rare patients (1 to 30 patients worldwide) for free, for life.
“Our patients are at the heart of everything that we do at n-Lorem. When I founded n-Lorem, I recognized that the demand for treatment for nano-rare patients would be substantial because each patient needs an individualized approach that addresses their specific gene mutation. What I didn’t expect was that the demand would be so enormous, so early,” said Stanley T. Crooke, M.D., Ph.D., Founder, CEO and Chairman of n-Lorem Foundation. “La Jolla Labs is the ideal partner to help us meet this demand. Jeff and his team have significant experience in ASO technology and the expertise and tools to expedite our ASO discovery programs while maintaining our rigorous quality standards.”
This strategic collaboration focuses on leveraging La Jolla Labs’ high throughput screening technology to design and screen for RNA-targeting antisense oligonucleotide therapeutics.
“We are pleased to partner with n-Lorem for this critical initial step in bringing help to nano-rare patients. Unlike standard drug discovery and development efforts, which can take 12 or more years to reach a patient, the discovery work we are doing today could reach a nano-rare patient in less than two years,” said Jeff Milton, CEO at La Jolla Labs. “My team has extensive experience in ASO technology and we understand the importance of maintaining rigorous quality while also expediting the discovery process using our proprietary assays and automated screening technology.”
- Read: Annual Letter
- Watch: n-Lorem Foundation Delivers Hope and Treatment for Patient with Ultra-Rare Disease
- Watch: n-Lorem Foundation: Creating a Better Future for Ultra-Rare, One Patient at a Time
About La Jolla Labs
Founded in 2021 La Jolla Labs is focused on developing high throughput screening automation and software tools for RNA drug discovery. In addition to proprietary screening assays La Jolla Labs is developing hts.bio™, a cloud-based software tool for designing, executing, and analyzing large high-throughput experiments for genomic-based drug discovery. This subscription-based service is scheduled for release in late 2022.
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 40 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