The Conrad Prebys Foundation to Support the Discovery and Development of Personalized ASO Medicines for Nano-rare Patients

n-Lorem awarded a $300,000 one-year grant to help address the needs of nano-rare patients.  

SAN DIEGO–(BUSINESS WIRE)–#ASO—n-Lorem, a nonprofit foundation, is the recipient of a $300,000 grant from The Conrad Prebys Foundation that will support n-Lorem’s discovery and development efforts to provide personalized experimental antisense oligonucleotide (ASO) medicines for nano-rare patients (1 to 30 patients worldwide) for free, for life.

“We are grateful to be a part of the San Diego community and to be working with the Foundation whose support may have a profound impact on the lives of several of our nano-rare patients. Nano-rare patients have a genetic mutation that is unique to them, and therefore they have few, if any therapeutic options. Today ASO technology and n-Lorem represent the only option for the treatment for many of these nano-rare patients, who are desperate and isolated,” said Stanley T. Crooke, M.D., Ph.D., Founder, CEO and Chairman of n-Lorem Foundation. “We have more than 40 nano-rare patients at n-Lorem, eight of whom are located in San Diego county. Out of this work, we hope to advance several programs from discovery into development. This work will lead us one step closer to providing a personalized ASO medicine for each nano-rare patient.”

The funding will support n-Lorem’s efforts to discover optimal ASO candidates for several nano-rare patients. Once identified, n-Lorem will conduct preclinical studies necessary to support clinical development for each optimized ASO.

“Through significant investments in medical research during his lifetime, Conrad Prebys helped pave the way for private philanthropy to play a meaningful role in the advancement of the field. The Conrad Prebys Foundation is proud to continue this legacy by supporting n-Lorem Foundation’s unique and vitally important work in rare genetic disease treatment,” said Erin Decker, Director of Grantmaking at The Conrad Prebys Foundation. “Beyond the significant communities of rare disease patients, n-Lorem’s research has the potential to accelerate important breakthroughs in the biomedical understanding of diseases in general for the benefit of all.”

Learn more about n-Lorem’s mission at www.nlorem.org, and please consider giving to n-Lorem to bring hope, possibility and treatment options to these needy patients and families.

• Read: Stanley Crooke’s Annual Letter
• 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

The Conrad Prebys Foundation

The Conrad Prebys Foundation was established by real estate entrepreneur Conrad Prebys to perpetuate his commitment to philanthropic endeavors. The Conrad Prebys Foundation inspires transformational change by funding projects in alignment with Conrad Prebys’ legacy interests and continuing his efforts that spark meaningful advancement in our communities. For more information, visit www.ConradPrebysFoundation.org.

About n-Lorem

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 130 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, YouTube and Instagram.

Contacts

Amy Williford, Ph.D.

Director of Communications

[email protected]