NEW YORK – St. Jude Children's Research Hospital in Memphis, Tennessee, is collaborating with the nonprofit n-Lorem Foundation to develop antisense oligonucleotide (ASO) therapies for children with rare genetic neurological diseases, n-Lorem announced Thursday.
Carlsbad, California-based n-Lorem designs experimental ASO therapies for patients diagnosed with rare diseases caused by single genetic defects. The organization focuses on so-called "nano-rare" diseases, which it describes as conditions affecting 30 or fewer patients worldwide. It provides ASO therapies to patients for free and is funded by donations and philanthropy.
ASOs comprise short strands of modified DNA designed to target messenger RNA of a defective gene to correct the abnormality.
As part of the partnership, n-Lorem with St. Jude's genomic medicine program will develop two experimental ASO therapies. n-Lorem did not immediately respond to a request for comment on which conditions the initial two programs will be designed for or how the organizations will decide which conditions to focus on.
"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," n-Lorem Founder, Chairman, and CEO Stanley Crooke said in a statement. "Together, we can accelerate the development of therapies for more patients."
Crooke, who previously founded Ionis Pharmaceuticals, launched n-Lorem in 2020.