CRISPR Therapeutics(US:CRSP)2025-06-25 14:06CASGEVY & Hemoglobinopathies - CASGEVY is approved for severe sickle cell disease and beta thalassemia, enabled by CRISPR/Cas9 technology[7] - CRISPR Therapeutics estimates an addressable market of approximately 60,000 severe patients in approved territories for CASGEVY[17] - The company anticipates continued progress in the U S market for CASGEVY, addressing a significant unmet need and reducing expenditures of $3 billion annually in the U S for sickle cell disease[18] - The first Gulf Cooperation Council (GCC) patient was reimbursed at approximately $2 million for CASGEVY treatment, and NHS reimbursement was achieved for beta-thalassemia[18] CAR T Therapies - CTX112, an allogeneic CAR T therapy, showed an overall response rate (ORR) of 67% and a complete response rate (CR) of 50% in initial efficacy data from a Phase I clinical trial (N=12)[36] - New CTX112 data (N=25) indicates cell expansion comparable to autologous CAR T therapies, with mean Cmax ranging from 45,000 to 70,000 copies/μg at dose levels 3 and 4[39] - CTX112 demonstrated a 100% overall response rate (ORR) in 6 patients receiving the therapy post-T cell engager (TCE) therapy[42] In Vivo & Cardiovascular Disease - CTX320, targeting elevated Lp(a), has the potential to benefit over 60 million U S patients[9, 63] - In non-human primate (NHP) studies, a single dose of CTX320 resulted in approximately 70% editing of LPA and approximately 95% reduction in plasma Lp(a) sustained at 2 years[55] - CTX310, targeting ANGPTL3, demonstrated approximately 70% editing of ANGPTL3 and sustained reduction in plasma ANGPTL3 and triglycerides in NHPs out to 1 year[66] Financials & Pipeline - CRISPR Therapeutics had a strong balance sheet of approximately $1.9 billion[12] - The company has 5 clinical programs and 10 preclinical programs[6]