Vertex partner CRISPR’s zugo-cel CAR-T hits 70% initial response, <$10k cost
CRISPR said its allogeneic CAR-T zugo-cel achieved nearly 70% initial complete response rate, with autologous-like expansion at top doses versus CTX110’s 40% benchmark. The company outlined autoimmune trial ambitions with cost of goods under $10,000, SLE remission data due H2 and noted its sickle cell/thalassemia commercial product with Vertex.
1. Efficacy and Persistence Gains
At the Citi summit, CRISPR highlighted zugo-cel’s targeted CAR insertion into the TCR locus and beta-2 microglobulin edit, driving immune evasion and nearly 70% initial complete response rate with autologous-like T-cell expansion versus CTX110’s 40%.
2. Autoimmune Development and Cost Efficiency
The company underscored a cost of goods under $10,000 for allogeneic CAR-T and reported nine-month off-therapy SLE remission in one patient, while recruiting for a rheumatology basket trial in lupus, myositis and scleroderma and initiating ITP/WAIHA studies.
3. Vertex Collaboration in Sickle Cell and Thalassemia
CRISPR noted its existing commercial sickle cell disease and thalassemia product with Vertex, leveraging its Framingham manufacturing facility to support U.S. launches and potential label expansions.
4. Regulatory and Commercial Strategy
CRISPR plans to engage regulators by year-end on single-arm versus randomized registrational trials, test combination therapy with pirtobrutinib in third-line oncology, and pursue global rollouts to enhance adoption in oncology and autoimmune settings.