CRISPR Therapeutics Reports Positive Top-Line Results from Its Phase 1 CARBON Trial of CTX110 in Relapsed or Refractory CD19+ B-cell Malignancies - Seite 3

(CR) rate, N (%) 0 (0%) 1 (33%) 2 (50%) 1 (100%)

• Complete response (CR) was achieved at Dose Levels 2, 3, and 4. At DL3, two out of four patients had a complete response. These two patients remain in CR.
• The four patients with CR had deep responses including the complete resolution of extranodal disease, normalization of all nodal disease to 1.5 cm or smaller, and a Deauville score of 2 or lower. Additionally, one of these patients who had 30% lymphoblasts in the bone marrow achieved complete clearance after CTX110 infusion.
• CR was achieved both in patients with diffuse large B-cell lymphoma and with transformed follicular lymphoma, as well as in patients who were primary refractory and who had relapsed after autologous stem cell transplant.
• At DL2 and above, CTX110 was detected at multiple time points in all patients, with peak expansion occurring at 1-2 weeks and cells detected as late as 180 days post-infusion​.

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About CTX110
CTX110, a wholly owned program of CRISPR Therapeutics, is a healthy donor-derived gene-edited allogeneic CAR-T investigative therapy targeting cluster of differentiation 19, or CD19. CTX110 is being investigated in the CARBON trial.

About CARBON
The ongoing Phase 1 single-arm, multi-center, open label clinical trial, CARBON, is designed to assess the safety and efficacy of several dose levels of CTX110 for the treatment of relapsed or refractory B-cell malignancies. CRISPR Therapeutics is the sponsor of the CARBON trial.

About CRISPR Therapeutics
CRISPR Therapeutics is a leading gene editing company focused on developing transformative gene-based medicines for serious diseases using its proprietary CRISPR/Cas9 platform. CRISPR/Cas9 is a revolutionary gene editing technology that allows for precise, directed changes to genomic DNA. CRISPR Therapeutics has established a portfolio of therapeutic programs across a broad range of disease areas including hemoglobinopathies, oncology, regenerative medicine and rare diseases. To accelerate and expand its efforts, CRISPR Therapeutics has established strategic partnerships with leading companies including Bayer, Vertex Pharmaceuticals and ViaCyte, Inc. CRISPR Therapeutics AG is headquartered in Zug, Switzerland, with its wholly-owned U.S. subsidiary, CRISPR Therapeutics, Inc., and R&D operations based in Cambridge, Massachusetts, and business offices in San Francisco, California and London, United Kingdom. For more information, please visit www.crisprtx.com.