Crispr Therapeutics (CRSP) said Friday that its alpha-1 antitrypsin deficiency treatment program, called CTX460, showed "specific and durable effects" in preclinical studies.
Data demonstrated that a single dose of CTX460 resulted in significant, dose-dependent correction of liver deoxyribonucleic acid or DNA in both rat and mouse AATD models, as well as over 90% messenger ribonucleic acid or mRNA correction at a clinically relevant dose of 0.5 mg/kg in PiZ mice.
Additionally, the durability of gene editing with CTX460 was maintained in both rat and mouse models for up to seven and nine weeks, respectively, the company said.
Crispr Therapeutics said it plans to conduct a clinical trial with CTX460 in mid-2026.
Shares of the company were up 4% in recent Friday premarket activity.