n-Lorem Foundation Collaborates to Support Children With Extremely Rare Diseases

This collaboration brings together two patient-centric, non-profit organizations that accept patients irrespective of their financial means

SAN DIEGO--(BUSINESS WIRE)--#ASO--n-Lorem, a nonprofit foundation, today announce a collaboration with St. Jude Children’s Research Hospital to accelerate the development of optimized experimental antisense oligonucleotide (ASO) medicines for pediatric patients with extremely rare genetic neurological disorders.

St. Jude and n-Lorem will initially develop two ASO treatments together through a collaboration designed to increase the capacity of the n-Lorem pipeline and to expand St. Jude’s recently formed genomic medicine program, which will initially focus on ASO therapies and expand to other therapeutic strategies to advance treatments for this underserved population. As part of the collaborative effort, n-Lorem is contributing its approach to ASO design and development which will help inform initial ASO programs at St. Jude.

“This collaboration is truly transformational and has the potential to expand the number of patients with extremely rare diseases that could benefit from an optimized personalized ASO medicine. By creating a model that is set apart by the rigorous processes n-Lorem has in place and leveraging the teams that created ASO technology to teach others, we are able to work with other high-quality institutions, like St. Jude, so that together, we can accelerate the development of therapies for more patients,” said Stanley T. Crooke, M.D., Ph.D., Founder, Chairman and CEO, n-Lorem Foundation. “St. Jude’s patient-centric focus offers a holistic approach to patient care that should serve as a model for other institutions.”

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 received over 225 applications for treatment with approximately 100 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.

To learn more about n-Lorem’s mission at www.nlorem.org to bring hope, possibility and treatment options to these patients and families in need.

n-Lorem Contact:
Amy Williford, Ph.D.
Sr. Director of Communications
amy.williford@nlorem.org