Bridging genomic discovery to potentially life-saving therapies, the key to treating the untreatable
Antisense therapies (antisense oligonucleotides or ASOs) are designed to bind precisely with RNA, halting the process of creating a disease-causing protein — disrupting diseases and changing their course—from the rarest of conditions, to those that impact millions of people. ASO’s are short strands of modified DNA that can specifically target the transcripts of a defective gene to correct the abnormality. The advantage of an ASO medicine is that they can be developed rapidly, inexpensively, and are highly specific.
Antisense Drugs Target the Messenger, Not the Protein Product
We Can Do This!
The n-Lorem Foundation and its partners are ready to evaluate patients with rare genetic diseases who may benefit from being treated with a custom experimental ASO.