Slide 1: Top-Line Results from the Phase 1 CARBON Trial of CTX110
Top-Line Results from the Phase 1 CARBON Trial of CTX110™ October 21, 2020 ® 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 CRISPR Therapeutics Tony Ho, MD Executive Vice President, Head of Research & Development CRISPR Therapeutics Ewelina Morawa, MD Vice President, Clinical Development CRISPR Therapeutics Joseph McGuirk, DO Professor of Medicine a
Slide 4: We Have Made Tremendous Progress Over the Past 5 Years
Built the leading CRISPR company: 4 programs in the clinic; >300 employees; >$1B cash balance Demonstrated, for the first time, the power of CRISPR gene editing in rare diseases: initial data with CTX001™ supportive of a potential functional cure for sickle cell disease and beta thalassemia Advanced
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 Multiplex CRISPR gene editing in one step designed to: 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 lentivi
Slide 7: 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 8: CARBON: Patient Flow
CARBON: Patient Flow At each completed dose level, two lots of CTX110 manufactured from different healthy donors were used Given antitumor activity observed, additional patients enrolled at DL3 and DL4 added Data as of September 28, 2020 At least 28 days of follow-up (included in data cut): 11 patie
Slide 9: CARBON: Baseline Patient Characteristics
CARBON: Baseline Patient Characteristics Cell dose (CAR+ T cells) DL1 30x106 N=3 DL2 100x106 N=3 DL3 300x106 N=4 DL4 600x106 N=1 Median age, years (range) 52 (50-61) 64 (58-74) 64.5 (62-74) 72 Male 2 (66.7) 2 (66.7) 1 (25) 1 (100) Lymphoma subtypes Diffuse large B-cell lymphoma (DLBCL)1 1 (33.3) 3 (
Slide 10: Dose-Dependent Responses Observed with CTX110
Cell dose (CAR+ T cells) DL1 30x106 N=3 DL2 100x106 N=3 DL3 300x106 N=4 DL4 600x106 N=1 Overall response rate (ORR), N (%) 0 (0%) 1 (33%) 2 (50%) 1 (100%) Complete response (CR) rate, N (%) 0 (0%) 1 (33%) 2 (50%) 1 (100%) Data as of September 28, 2020 Dose-Dependent Responses Observed with CTX110 Be
Slide 11: Dose-Dependent Reduction in Tumor Size with CTX110
Dose-Dependent Reduction in Tumor Size with CTX110 Data as of September 28, 2020 DL1 DL2 DL3 DL4 660% PD* PD PD SD SD PD SD CR CR CR CR Patients * Patient subsequently failed autologous CAR-T Best tumor size reduction per 2014 Lugano criteria by independent central assessment Case study 1 Case study
Slide 12: Complete Responses with CTX110 Showed Durability at Month 3 and Beyond
Complete Responses with CTX110 Showed Durability at Month 3 and Beyond Data as of September 28, 2020 CR SD PD PR DL1 DL2 DL3 DL4 * Imaging per protocol occurs at M1, M3, and M6; * Patient died while in CR at Day 52 post CTX110 infusion following data cutoff
Slide 13: Acceptable Safety Profile with CTX110 at DL3 and Below
Acceptable Safety Profile with CTX110 at DL3 and Below N=10 Grade 1 Grade 2 Grade 3 Grade 4 Grade 5 Graft-versus-Host Disease (GvHD) 0 0 0 0 0 Cytokine Release Syndrome (CRS)1,2 1 (10%) 2 (20%) 0 0 0 ICANS1,3 0 1 (10%) 0 0 0 Infections 0 0 1 (10%) 0 0 (1) Per ASTCT criteria; other AEs graded per CTC
Slide 14: Patient Treated at DL4 (600x10^6 CAR+ T cells)
Patient characteristics: 72-year-old male with relapsed transformed follicular lymphoma following five prior lines of therapy, including autologous stem cell transplant Efficacy: Complete response on Day 25 post infusion of CTX110 Safety: Experienced Grade 2 CRS at Day 5 that resolved Admitted with
Slide 15: Emerging Pharmacokinetic Profile of CTX110
Emerging Pharmacokinetic Profile of CTX110 For patients in DL2 through DL4: CAR-T cells detected at multiple time points in all patients Redistribution phase observed from Day 1 to Day 3, followed by expansion Consistent peak expansion of CTX110 in the peripheral blood seen around 1-2 weeks post inf
Slide 16: CTX110 Case Study: Stable Disease with Remaining Tumor Negative for CD19
CTX110 Case Study: Stable Disease with Remaining Tumor Negative for CD19 Patient characteristics 62-year-old male with transformed follicular lymphoma Relapsed following two prior lines of therapy Treated with CTX110 at DL3 (300x106 CAR+ T cells) Safety and efficacy No fever, CRS, or ICANS Visible r
Slide 17: CTX110 Case Study: Complete Response Following Eradication of a Large Tumor Mass
CTX110 Case Study: Complete Response Following Eradication of a Large Tumor Mass Baseline D28 (CR) Patient characteristics 62-year-old female diagnosed with DLBCL Relapsed following two prior lines of therapy, including autologous SCT Treated with CTX110 at DL3 (300x106 CAR+ T cells) Safety and effi
Slide 18: Initial CTX110 Data Supports Our Approach
Initial CTX110 Data Supports Our Approach Early evidence of dose response Complete responses achieved in 4 patients (both DLBCL and tFL) Data in line with early autologous CAR-T trials Dose-dependent antitumor activity Acceptable safety profile at DL3 and below Initial experience demonstrates versat
Slide 19: Planned Next Steps for CTX110 and Our CRISPR-Edited CAR-T Pipeline
“Full steam ahead” on CTX110 Proceed into expansion cohort following selection of optimal dose Re-dosing now included as an option in all cohorts; one patient re-dosed with CTX110 at DL3 so far Continue rapid progress on CTX120 and CTX130 Dosing ongoing in trial of CTX120 in multiple myeloma Dosing
Slide 20: Our I/O Strategy and Pipeline
Our I/O Strategy and Pipeline Expand from hematologic cancers into solid tumors Validate allogeneic platform with proven targets Unlock the full potential of I/O cell therapy with CRISPR CTX120 Anti-BCMA allogeneic CAR-T CTX110 Anti-CD19 allogeneic CAR-T Enrolling Enrolling CTX130 in lymphomas Anti-
Slide 21: Thank You to Patients and Their Families
Thank You to Patients and Their Families Thank you to patients and their families, investigators, and site staff United States CTX110 sites Europe Australia University of Kansas Medical Center Westwood, KS Oregon Health and Science University Portland, OR Sarah Cannon Research Institute Nashville, T
Slide 22: CRISPR Therapeutics
CRISPR THERAPEUTICS, CRISPR Therapeutics www.crisprtx.com