Slide 1: Updated Results from the Phase 1 CARBON Trial of CTX110
Updated Results from the Phase 1 CARBON Trial of CTX110™ October 12, 2021 ® Exhibit 99.2
Slide 2: Forward-looking statements
The presentation and other related materials may contain a number of “forward-looking statements” within the meaning of the Private Securities Litigation Reform Act of 1995, as amended, including statements regarding CRISPR Therapeutics’ expectations about any or all of the following: (i) the safety
Slide 3: Presenters on Today's Call
Presenters on Today’s Call Samarth Kulkarni, PhD Chief Executive Officer Tony Ho, MD Executive Vice President, Head of Research & Development Ewelina Morawa, MD Vice President, Clinical Development
Slide 4: Building the Leading CRISPR Company
4 clinical programs; >450 employees; ~$2.5B cash balance Over 50 sickle cell and beta-thalassemia patients treated with CTX001™ showing a consistent, functionally curative profile Regulatory filings possible in the next 18-24 months with 30,000+ patients suitable for treatment in the U.S. and Europe
Slide 5: Allogeneic CAR-T Therapy Has Transformative Potential
Allogeneic CAR-T Therapy Has Transformative Potential Specificity, efficiency, and versatility of CRISPR gene editing facilitates consistent, multiplex editing to produce allogeneic cell therapies and enhance immune cell performance Before Patient Diagnosis After Patient Diagnosis Off-the-shelf: Imm
Slide 6: CTX110: Differentiated CRISPR-edited Allogeneic CAR-T Design
CTX110: Differentiated CRISPR-edited Allogeneic CAR-T Design Anti-CD19 CAR TCR disruption MHC I disruption Prevent GvHD via TCR disruption Improve consistency and safety by precise insertion of CAR construct into TRAC locus without using lentivirus or retrovirus Improve persistence in the allo setti
Slide 7: CARBON: Patient Flow
CARBON: Patient Flow ≥28 days of follow-up (included in data cut): 26 patients Enrolled: 30 patients Treated1: 29 patients just one patient enrolled but not treated Data as of August 26, 2021 23 patients infused 24 patients enrolled (1) Includes patients in the process of being treated as of the cut
Slide 8: CARBON: Trial Design
CARBON: Trial Design Key eligibility criteria Age ≥18 years Relapsed/refractory non-Hodgkin lymphoma, as evidenced by 2+ lines of prior therapy ECOG performance status 0 or 1 Adequate renal, liver, cardiac, and pulmonary organ function No prior allogeneic SCT or treatment with CAR-T therapy Primary
Slide 9: CARBON Only Enrolled Patients with Aggressive Disease
CARBON Only Enrolled Patients with Aggressive Disease including DLBCL NOS, high-grade lymphoma (e.g., triple hit), and transformed follicular lymphoma with significant baseline tumor volume Both relapsed and refractory patients, including – 31% of patients had progressed through 2 or more lines of t
Slide 10: CARBON: Baseline Patient Characteristics
CARBON: Baseline Patient Characteristics Cell dose (CAR+ T cells) DL1 30x106 N=3 DL2 100x106 N=3 DL3 300x106 N=6 DL3.5 450x106 N=6 DL4 600x106 N=8 Median age, years (range) 52 (50-61) 64 (58-74) 69 (62-74) 67.5 (25-74) 65.5 (55-75) Female 1 (33) 1 (33) 4 (67) 2 (33) 2 (25) Lymphoma subtypes Large B-
Slide 11: Dose-Dependent Responses with CTX110
Cell dose (CAR+ T cells) DL1 30x106 N=3 DL2 100x106 N=3 DL3 300x106 N=6 DL3.5 450x106 N=6 DL4 600x106 N=8 Overall response rate (ORR), N (%) 0 (0%) 1 (33%) 3 (50%) 4 (67%) 6 (75%) Complete response (CR) rate, N (%) 0 (0%) 1 (33%) 2 (33%) 3 (50%) 3 (38%) Dose-Dependent Responses with CTX110 DL2+ mITT
Slide 12: Durable Responses Observed with CTX110
Durable Responses Observed with CTX110 Dose level of re-dose indicated if different from initial dose level; Imaging per protocol occurs at M1, M3, and M6; CR: complete response; PR: partial response; SD: stable disease; PD: progressive disease (1) Patient had a localized tumor recurrence that was e
Slide 13: CTX110 Was Well Tolerated Across All Dose Levels
CTX110 Was Well Tolerated Across All Dose Levels DL1 (N=3) DL2 (N=3) DL3 (N=6) DL3.5 (N=6) DL4 (N=8) DL2+ (N=23) Gr 1-2 Gr 3+ Gr 1-2 Gr 3+ Gr 1-2 Gr 3+ Gr 1-2 Gr 3+ Gr 1-2 Gr 3+ Gr 1-2 Gr 3+ CRS1 1 (33) - 2 (67) - 2 (33) - 3 (50) - 6 (75) - 13 (57) - ICANS2 - - 1 (33) - - - - - - 1 (13) 1 (4) 1 (4)
Slide 14: CTX110 Case Study: On-going Complete Response Months After Single Infusion
CTX110 Case Study: On-going Complete Response 12 Months After Single Infusion Patient characteristics 62-year-old female diagnosed with DLBCL Relapsed following 2 prior lines of therapy, including autologous SCT Treated with CTX110 at DL3 (300x106 CAR+ T cells) Safety and efficacy data CR at Day 28
Slide 15: CTX110 Case Study: 18+ Months of Clinical Benefit in a Primary Refractory Patient
CTX110 Case Study: 18+ Months of Clinical Benefit After 2 Infusions in a Primary Refractory Patient Patient characteristics 58-year-old male with Stage IV DLBCL (NOS) Refractory to both prior lines of therapy (R-CHOP, R-GDP) Safety and efficacy data 1st infusion of CTX110: DL2 (100x106 CAR+ T cells)
Slide 16: Pharmacokinetic Profile Supports Consolidation Dose at Month 1
Pharmacokinetic Profile Supports Consolidation Dose at 1 Month in the peripheral blood aroundpost infusion with no evidence of accelerated clearance from anti-drug or anti-HLA antibodies In many patients, CTX110 levels in the peripheral blood drop LLOD Mean ± SEM N=15-21 at each timepoint1 (1) Sampl
Slide 17: Strong Rationale for Consolidation Dose of CTX110
Strong Rationale for Consolidation Dose of CTX110 30x106 N=3 100x106 N=3 300x106 N=6 450x106 N=6 600x106 N=8 Cell dose (CAR+ T cells) ORR, % CR rate, % Consolidation has the potential to create a 2nd round of antitumor activity with favorable “E:T” ratio to increase deep and durable responses (1) CA
Slide 18: Conclusions and Next Steps
Expand CARBON into a in Q1 2022 Broaden into Further scale manufacturing in our by advancing additional gene-edited allogeneic CAR-T programs to the clinic, including novel edits for increased potency Conclusions Rationale for consolidation & next steps Context Results with approved autologous CAR-T
Slide 19: Thank You to Patients and Their Families
Thank You to Patients and Their Families Thank you to and their families, investigators, and site staff CTX110 sites United States Emory University Atlanta, GA Mayo Clinic Jacksonville, FL Oregon Health and Science University Portland, OR Sarah Cannon Research Institute Nashville, TN Texas Transplan
Slide 20: CRISPR Therapeutics
CRISPR THERAPEUTICS CRISPR Therapeutics Wwww.crisprtx.com