NF1-HCT (ASO Program)

iNFixion Bioscience is developing an antisense oligonucleotide (ASO) therapy designed to address the root cause of Neurofibromatosis Type 1 (NF1). NF1 is a genetic disorder caused by mutations in one copy of the NF1 gene, leading to a deficiency in the neurofibromin protein, a condition known as haploinsufficiency. This deficiency results in uncontrolled cell growth, leading to tumors on nerves, neurocognitive issues, and other systemic manifestations. iNFixion's ASO program, part of their NF1 Haploinsufficiency Correction Therapy (NF1-HCT) approach, utilizes targeted oligonucleotides to boost the expression of functional neurofibromin from the remaining healthy allele. The goal is to restore neurofibromin levels to a normal physiological range to treat or prevent the progression of the disease. The program is currently in the preclinical stage of development.

Other names

NF1 Haploinsufficiency Correction Therapy (ASO)