ASO-005-02

ASO-005-02 is an antisense oligonucleotide (ASO) therapeutic developed by Q-State Biosciences for the treatment of Spastic Paraplegia 49 (SPG49), also known as Hereditary Sensory and Autonomic Neuropathy type 9 (HSAN9). This ultra-rare neurological disorder is caused by mutations in the *TECPR2* gene, specifically the pathogenic frameshift mutation c.1319delT (p.Leu440Argfs*19) in exon 8. ASO-005-02 utilizes an exon-skipping strategy to bypass the premature stop codon, resulting in the expression of a functional, truncated protein variant (TECPR2ΔEx8). The drug features 2'-O-methyl modifications and phosphorothioate chemistry. Preclinical studies have demonstrated potency in patient-derived fibroblasts and broad CNS distribution with acceptable tolerability in non-human primates following intrathecal administration.

Other names

TECPR2 antisense oligonucleotideTECPR-2 antisense oligonucleotideTECPR 2 antisense oligonucleotideTECPR2 ASOTECPR-2 ASOTECPR 2 ASO