Abiopharma

CRISPR Therapeutics (CRSP) Announces Clinical Trial Update CRISPR Therapeutics (CRSP) provided an update on its clinical development programs. Clinical Development Highlights:

• Collaboration: CRISPR THERAPEUTICS AG
  • targeting angiopoietin-related protein 3 (ANGPTL3), into Phase 1b clinical trials, prioritizing development in severe hypertriglyceridemia and refractory hypercholesterolemia.oCTX320, targeting LPA, has demonstrated reductions of up to 73% in the dose escalation phase of the clinical trial. In parallel, the Company is advancing a next-generation LPA program, CTX321, incorporating an updated guide RNA that demonstrates approximately two-fold greater potency in preclinical testing, while utilizing the same LNP delivery system. CTX321 is currently in IND/CTA-enabling studies.oIn addition to its clinical-stage programs, the Company continues to advance several preclinical in vivo gene editing candidates, including: ▪CTX460, targeting SERPINA1 for the treatment of alpha-1 antitrypsin deficiency (AATD), the first investigational candidate to emerge from the Company’s SyNTase editing platform; and▪CTX340, targeting angiotensinogen (AGT) for refractory hypertension, which is currently in IND/CTA-enabling studies. •Zugocabtagene geleucel (zugo-cel; formerly CTX112) continues to advance in both autoimmune disease and hematologic malignancies. oIn autoimmune disease, Phase 1 clinical trials are ongoing across multiple indications, including systemic lupus erythematosus (SLE), systemic sclerosis (SSc), and inflammatory myositis and a second Phase 1 trial in immune thrombocytopenia purpura (ITP) and warm autoimmune hemolytic anemia (wAIHA). The first patient with SLE, refractory to 9 prior therapies with a baseline Systemic Lupus Erythematosus Disease Activity Index 2000