RARE Daily

St. Jude Enters Collaboration with n-Lorem to Support Kids with Extremely Rare Diseases

October 5, 2023

Rare Daily Staff

The nonprofit n-Lorem said it entered into a collaboration with St. Jude Children’s Research Hospital to accelerate the development of experimental antisense oligonucleotide 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 it said 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 Crooke, 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.”

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